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5-Fluorouracil

Phase 3

Colorectal Cancer | Small molecule | Oncology |Roche Holding AG|Last Updated: Nov 28, 2025

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Trial Design

RandomizedCONTROLLED
Total Trials7
Total Enrollment7,955

FDA Designations

No designations recorded

Clinical trial landscape

5-Fluorouracil · 18 trials · 6 indications

Phase 3 8Phase 2 10
NCT01774786A Study of Pertuzumab in Combination With Trastuzumab and Chemotherapy in Participants With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Metastatic Gastroesophageal Junction or Gastric CancerGastric Cancer
COMPLETED780 Analytics
NCT01358877A Study of Pertuzumab in Addition to Chemotherapy and Trastuzumab as Adjuvant Therapy in Participants With Human Epidermal Growth Receptor 2 (HER2)-Positive Primary Breast CancerBreast Cancer
COMPLETED4,804 Analytics
NCT00950300A Study to Compare Subcutaneous (SC) Versus Intravenous (IV) Administration of Herceptin (Trastuzumab) in Women With Human Epidermal Growth Factor Receptor (HER) 2-Positive Early Breast CancerBreast Cancer
COMPLETED596 Analytics
NCT00548548A Study of Bevacizumab in Combination With Capecitabine and Cisplatin as First-line Therapy in Patients With Advanced Gastric CancerAdenocarcinoma
COMPLETED774 Analytics
NCT00112918Combination Chemotherapy With or Without Bevacizumab in Treating Patients Who Have Undergone Surgery for High Risk Stage II or Stage III Colon CancerColorectal Cancer
COMPLETED3,451 Analytics
NCT00069121A Study of Xeloda (Capecitabine) Plus Oxaliplatin in Patients With Colon CancerColorectal Cancer
COMPLETED1,886 Analytics
NCT02563054A Study of Capecitabine (Xeloda) in Participants With Advanced or Metastatic Gastric CancerGastric Cancer
COMPLETED316 Analytics
NCT00009737A Study of Xeloda (Capecitabine) Compared With 5-Fluorouracil in Combination With Low-Dose Leucovorin in Patients Who Have Undergone Surgery for Colon CancerColorectal Cancer
COMPLETED1,987 Analytics
PHASE3COMPLETED
A Study of Pertuzumab in Combination With Trastuzumab and Chemotherapy in Participants With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Metastatic Gastroesophageal Junction or Gastric Cancer
Gastric CancerUnlock trial analytics
PHASE3COMPLETED
A Study of Pertuzumab in Addition to Chemotherapy and Trastuzumab as Adjuvant Therapy in Participants With Human Epidermal Growth Receptor 2 (HER2)-Positive Primary Breast Cancer
Breast CancerUnlock trial analytics
PHASE3COMPLETED
A Study to Compare Subcutaneous (SC) Versus Intravenous (IV) Administration of Herceptin (Trastuzumab) in Women With Human Epidermal Growth Factor Receptor (HER) 2-Positive Early Breast Cancer
Breast CancerUnlock trial analytics
PHASE3COMPLETED
A Study of Bevacizumab in Combination With Capecitabine and Cisplatin as First-line Therapy in Patients With Advanced Gastric Cancer
AdenocarcinomaUnlock trial analytics
PHASE3COMPLETED
Combination Chemotherapy With or Without Bevacizumab in Treating Patients Who Have Undergone Surgery for High Risk Stage II or Stage III Colon Cancer
Colorectal CancerUnlock trial analytics
PHASE3COMPLETED
A Study of Xeloda (Capecitabine) Plus Oxaliplatin in Patients With Colon Cancer
Colorectal CancerUnlock trial analytics
PHASE3COMPLETED
A Study of Capecitabine (Xeloda) in Participants With Advanced or Metastatic Gastric Cancer
Gastric CancerUnlock trial analytics
PHASE3COMPLETED
A Study of Xeloda (Capecitabine) Compared With 5-Fluorouracil in Combination With Low-Dose Leucovorin in Patients Who Have Undergone Surgery for Colon Cancer
Colorectal CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall Survival
From Baseline until death from any cause (Median [full range] duration of follow-up in Pertuzumab vs. Placebo arms for Primary Analysis: 24.4 [0-42] months vs. 25.0 [0-41] months; Final Analysis: 46.1 [0-70] months vs. 44.4 [0-68] months)

Overall survival (OS) was defined as the time from randomization to death from any cause. For participants who were still alive on the date of clinical data cut-off for the OS analysis, the last date when the participant was known to be alive on, or prior to the clinical cut-off date, was used to determine the censoring date. Participants who did not have any post-baseline data (e.g., dosing records, imaging dates, visit dates) were censored at the date of randomization plus 1 day.

Percentage of Participants With Invasive Disease-Free Survival (IDFS) Event (Excluding Second Primary Non-Breast Cancer [SPNBC]), as Assessed Using Radiologic, Histologic Examinations or Laboratory Findings
Randomization to the first occurrence of IDFS event (excluding SPNBC) (until data cut-off date 19 December 2016; median [range] follow-up: 3.8 [0-4.9] years)

Percentage of participants with IDFS events (excluding SPNBC) is reported. IDFS event was defined as the first occurrence of one of the following events: Ipsilateral invasive breast tumor recurrence (that is \[i.e.\], an invasive breast cancer involving the same breast parenchyma as the original primary lesion); ipsilateral local-regional invasive breast cancer recurrence (i.e., an invasive breast cancer in the axilla, regional lymph nodes, chest wall, and/or skin of the ipsilateral breast); distant recurrence (i.e., evidence of breast cancer in any anatomic site - other than the two above mentioned sites); death attributable to any cause; contralateral invasive breast cancer. All SPNBCs and in situ carcinomas (including ductal carcinoma in situ \[DCIS\] and lobular carcinoma in situ \[LCIS\]) and non-melanoma skin cancer were excluded as an event.

Kaplan-Meier Estimate of the Percentage of Participants Who Were IDFS Event-Free (Excluding SPNBC) at 3 Years, as Assessed Using Radiologic, Histologic Examinations or Laboratory Findings
3 years

Kaplan-Meier estimate of the percentage of participants who were IDFS event-free (excluding SPNBC) at 3 years is reported. IDFS event was defined as the first occurrence of one of the following events: Ipsilateral invasive breast tumor recurrence (i.e., an invasive breast cancer involving the same breast parenchyma as the original primary lesion); ipsilateral local-regional invasive breast cancer recurrence (i.e., an invasive breast cancer in the axilla, regional lymph nodes, chest wall, and/or skin of the ipsilateral breast); distant recurrence (i.e., evidence of breast cancer in any anatomic site - other than the two above mentioned sites); death attributable to any cause; contralateral invasive breast cancer. All SPNBCs and in situ carcinomas (including DCIS and LCIS) and non-melanoma skin cancer were excluded as an event.

Observed Serum Trough Concentration (Ctrough) of Trastuzumab Prior to Surgery
Pre-dose (0 hours) on Day 1 of Cycle 8 (cycle length of 21 days)

Pre-dose samples were obtained prior to surgery (Cycle 8). The observed Ctrough was recorded, averaged among all participants, and expressed in micrograms per milliliter (μg/mL).

Percentage of Participants With Pathological Complete Response (pCR)
After surgery following eight cycles of Herceptin + chemotherapy (approximately 6 months from Baseline)

Participants were evaluated following eight cycles of treatment and after surgery to assess for pCR, defined as absence of neoplastic invasive cells in the breast according to pathologist examination. The percentage of participants with pCR was reported, and the 95% CI for one-sample binomial was constructed using the Pearson-Clopper method.

Disease-free Survival in Stage III Cancer Patients - Time to Event
From first patient randomized until the data cut-off date of 30 June 2010 (36 months after the last patient randomized).

Disease-free survival (DFS) was defined as the time from the date of randomization to the time of a recurrence, a new occurrence of colorectal cancer or death due to any cause, whichever occurred first. Patients without an event were censored at the last date the patient was known to be disease-free. Recurrence and new occurrence of colorectal cancer were based on tumor assessments made by the investigator. Patients with no tumor assessments after baseline but still alive at the time of the clinical cut-off were censored at day 1.

Disease-free Survival in Stage III Cancer Patients - Number of Events
From first patient randomized until the data cut-off date of 30 June 2010 (36 months after the last patient randomized).

A disease-free survival (DFS) event was composed of a recurrence, a new occurrence of colorectal cancer or death due to any cause. Recurrence and new occurrence of colorectal cancer were based on tumor assessments made by the investigator. Triggering events for DFS are reported; a patient can have both recurrence and a new occurrence of colon cancer.

Disease-Free Survival (DFS) [Number of Events]
Time from randomization date to date of first DFS event/date last known to be event-free. Median observation time for DFS was 74 months (range: 0-95 months).

Determination of an event was based on tumor assessments and survival follow-up assessments. A disease-free survival (DFS) event was defined as any recurrence of the original colon cancer or appearance of a new colon or rectal cancer (proven by cytology or histology, when possible) or death due to any cause, whichever was earliest. Participants who had not had any such event at the time of data analysis were censored at the last date they were known to be event-free. Participants with no tumor assessments after baseline were censored at Day 1. An isolated event of increased CEA, or unexplained clinical deterioration were not considered to be evidence of relapse without support of other objective measurements. The date of relapse was defined as the date of the definitive assessment by objective measurements.

Disease-Free Survival (DFS) [Time to Event]
Time from randomization date to date of first DFS event/date last known to be event-free. Median observation time for DFS was 74 months (range: 0-95 months).

Determination of an event was based on tumor assessments and survival follow-up assessments. A disease-free survival (DFS) event was defined as any recurrence of the original colon cancer or appearance of a new colon or rectal cancer (proven by cytology or histology, when possible) or death due to any cause, whichever was earliest. Participants who had not had any such event at the time of data analysis were censored at the last date they were known to be event-free. Participants with no tumor assessments after baseline were censored at Day 1. An isolated event of increased CEA, or unexplained clinical deterioration were not considered to be evidence of relapse without support of other objective measurements. The date of relapse was defined as the date of the definitive assessment by objective measurements. The median was estimated by the Kaplan-Meier method. The full range values include censored observations.

Time to disease progression
Up to approximately 7.3 years
Disease-free Survival
Approximately 3 years

Participants with disease-free survival were reported. Disease-free survival was assessed as the number of days between randomization and the first time at which relapse, a new occurrence of colon cancer, or death was recorded, or the last time at which a participant was known to be disease free (censoring time).

Percentage of Participants With at Least One Event of New York Heart Association (NYHA) Class III or IV Heart Failure During the Neoadjuvant Treatment Period
From day of first dose of pertuzumab or trastuzumab until the end of the neoadjuvant treatment period (as defined in the description; up to 25 weeks)

Symptomatic left ventricular systolic dysfunction (otherwise referred to as heart failure) is a serious adverse event. The NYHA Functional Classification System for Heart Failure considers the patient's symptoms: Class III: Marked limitation of physical activity; Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV: Unable to carry on any physical activity without discomfort; Symptoms of heart failure at rest; If any physical activity is undertaken, discomfort increases. The 95% CIs were calculated with the Clopper-Pearson method. Results included events with onset from first dose of pertuzumab/trastuzumab prior to surgery through the day before the first dose of any study drug after surgery. If participant withdrew without entering adjuvant period, results included all events with onset from first dose of pertuzumab/trastuzumab through 42 days after last dose of any study drug or on the day of target surgery whichever was later.

Percentage of Participants With at Least One Left Ventricular Ejection Fraction (LVEF) Significant Decline, Defined as a Drop in LVEF of at Least 10 Percentage Points From Baseline and to Below 50%, During the Neoadjuvant Treatment Period
From day of first dose of pertuzumab or trastuzumab until the end of the neoadjuvant treatment period (as defined in the description; up to 25 weeks)

LVEF significant decline was defined as the decline in LVEF of \>/= 10%-points from baseline to an LVEF of \< 50%. A Confirmed LVEF Significant Decline was defined as at least two consecutive readings of significant declines in LVEF. A Single LVEF Significant Decline was defined as only one reading of a significant decline (no consecutive readings) in LVEF. The category of 'At Least one LVEF Significant Decline Event' was defined as the total of confirmed and single LVEF significant declines. The 95% confidence intervals (CIs) were calculated using the Clopper-Pearson method. Results include events with onset from the first dose of pertuzumab or trastuzumab prior to surgery through the day before the first dose of any study drug after surgery. If participant withdrew without entering adjuvant period, results included all events with onset from first dose of pertuzumab or trastuzumab through 42 days after last dose of any study drug or on the day of target surgery whichever is later.

Progression-Free Survival (PFS) in All Randomized Participants and Participants With PTEN Loss Tumors at Primary Analysis
Screening, at the end of Cycle 4 (cycle = 14 days) and every fourth cycle thereafter until disease progression or death, whichever occurred first, assessed up to approximately 1.75 years

PFS was defined as the time from randomization to the first occurrence of disease progression (as determined using RECIST Version 1.1 and assessed by the investigator), or death from any cause on study. Progressive disease (PD): At least a 20% increase in the sum of diameters of target lesions, and the sum must also demonstrate an absolute increase of at least 5 mm or progression of non-target lesions. Death on study was defined as death from any cause within 30 days of the last dose of study treatment regimen. Kaplan-Meier estimates were used for evaluation.

Progression-free Survival (PFS) According to Modified RECIST v1.1 Criteria
approximately 2 year
Progression-Free Survival Based on Response Evaluation Criteria in Solid Tumors (RECIST) Version (v) 1.1 as Determined by the Investigator
Screening up to disease progression or death due to any cause, whichever occurs first (assessed every 8-9 weeks after Cycle 1 Day 1 up to approximately 27 months overall)
Objective Response Rate (ORR) in the Intent-to-treat (ITT) Analysis Set
Up to 11 cycles of treatment (up to Week 22)

ORR was defined as the percentage of participants with shrinkage (partial response \[PR\]) or disappearance of cancer (complete response \[CR\]). Tumor response was evaluated according to the Response Evaluation Criteria In Solid Tumors (RECIST v1.1). The same method of tumor measurement and assessment had to be used to characterize each lesion throughout the study. Tumor assessment consisted of computerized tomography (CT) scan (abdomen + pelvis + chest) or contrast-enhanced magnetic resonance imaging (CE-MRI) (abdomen + pelvis) + non CE-CT (chest) according to the choice of the center. CR, Disappearance of all target lesions; PR, \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Objective Response Rate (ORR) in the Per-protocol Analysis Set (PPAS)
Up to 11 cycles of treatment (up to Week 22)

ORR was defined as the percentage of participants with shrinkage (PR) or disappearance of cancer (CR). Tumor response was evaluated according to the RECIST v1.1. The same method of tumor measurement and assessment had to be used to characterize each lesion throughout the study. Tumor assessment consisted of CT scan (abdomen + pelvis + chest) or CE-MRI (abdomen + pelvis) + non CE-CT (chest) according to the choice of the center. CR, Disappearance of all target lesions; PR, \>=30% decrease in the sum of the longest diameter of target lesions; OR = CR + PR.

Progression-Free Survival (PFS) According to Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1
From Baseline until death or disease progression; assessed every 6 weeks (maximum up to 45 months overall)

Tumor assessments were performed according to RECIST Version 1.1. Disease progression was defined as greater than or equal to (≥) 20 percent (%) increase in sum of largest diameters (LD) of target lesions in reference to the smallest sum of LD on study, in addition to an absolute increase ≥5 millimeters (mm). Disease progression was further defined as the last documented progression determined by the Investigator no later than 1 day before initiation of second-line therapy. Death on study included death from any cause occurring no later than 3 months after the last component of study treatment. PFS was defined as the time from randomization to death or disease progression, whichever occurred first. The median duration of PFS was estimated by Kaplan-Meier analysis and expressed in months. The 95% confidence interval (CI) was computed using the method of Brookmeyer and Crowley.

PFS According to RECIST Version 1.1 in Participants With High ERCC-1 Levels
From Baseline until death or disease progression; assessed every 6 weeks (maximum up to 45 months overall)

Tumor assessments were performed according to RECIST Version 1.1. Disease progression was defined as ≥20% increase in sum of LD of target lesions in reference to the smallest sum of LD on study, in addition to an absolute increase ≥5 mm. Disease progression was further defined as the last documented progression determined by the Investigator no later than 1 day before initiation of second-line therapy. Death on study included death from any cause occurring no later than 3 months after the last component of study treatment. PFS was defined as the time from randomization to death or disease progression, whichever occurred first. The median duration of PFS was estimated by Kaplan-Meier analysis and expressed in months. The 95% CI was computed using the method of Brookmeyer and Crowley.

PFS According to RECIST Version 1.1 in Participants With Low ERCC-1 Levels
From Baseline until death or disease progression; assessed every 6 weeks (maximum up to 45 months overall)

Tumor assessments were performed according to RECIST Version 1.1. Disease progression was defined as ≥20% increase in sum of LD of target lesions in reference to the smallest sum of LD on study, in addition to an absolute increase ≥5 mm. Disease progression was further defined as the last documented progression determined by the Investigator no later than 1 day before initiation of second-line therapy. Death on study included death from any cause occurring no later than 3 months after the last component of study treatment. PFS was defined as the time from randomization to death or disease progression, whichever occurred first. The median duration of PFS was estimated by Kaplan-Meier analysis and expressed in months. The 95% CI was computed using the method of Brookmeyer and Crowley.

PFS According to RECIST Version 1.1 in Participants With High ERCC-1 Levels Versus Participants With Low ERCC-1 Levels
From Baseline until death or disease progression; assessed every 6 weeks (maximum up to 45 months overall)

Tumor assessments were performed according to RECIST Version 1.1. Disease progression was defined as ≥20% increase in sum of LD of target lesions in reference to the smallest sum of LD on study, in addition to an absolute increase ≥5 mm. Disease progression was further defined as the last documented progression determined by the Investigator no later than 1 day before initiation of second-line therapy. Death on study included death from any cause occurring no later than 3 months after the last component of study treatment. PFS was defined as the time from randomization to death or disease progression, whichever occurred first. The median duration of PFS was estimated by Kaplan-Meier analysis and expressed in months. The 95% CI was computed using the method of Brookmeyer and Crowley.

PFS According to RECIST Version 1.1 in Participants With High Vascular Endothelial Growth Factor (VEGF)-A Levels Versus Participants With Low VEGF-A Levels
From Baseline until death or disease progression; assessed every 6 weeks (maximum up to 45 months overall)

Tumor assessments were performed according to RECIST Version 1.1. Disease progression was defined as ≥20% increase in sum of LD of target lesions in reference to the smallest sum of LD on study, in addition to an absolute increase ≥5 mm. Disease progression was further defined as the last documented progression determined by the Investigator no later than 1 day before initiation of second-line therapy. Death on study included death from any cause occurring no later than 3 months after the last component of study treatment. PFS was defined as the time from randomization to death or disease progression, whichever occurred first. The median duration of PFS was estimated by Kaplan-Meier analysis and expressed in months. The 95% CI was computed using the method of Brookmeyer and Crowley.

Percentage of Participants With Pathological Complete Response Following Principle Investigator Review
Up to 7.5 years

Pathological complete response was defined as absence of invasive neoplastic cells at microscopic examination of the tumor remnants after surgery following primary systemic therapy.

Percentage of Participants With Tumor Sterilization Defined by ypT0-N0
After surgery (Arm A: approximately 28-31 weeks after initiation of treatment; Arm B: approximately 13-15 weeks after initiation of treatment)

Tumor sterilization was defined as the absence of residual tumor cells in the resected specimen including lymph nodes (ypT0-N0). The rate of sterilization of the tumoral specimen was assessed after surgery on the surgical specimen by local review. Analyses were performed for participants who have been operated as defined by the protocol (within the study and TME technique) and for all participants who have been operated. Reported is the percentage of participants with tumor sterilization.

Percentage of participants with pathological complete tumor response Assessed by Expert Blinded Independent Review according to the Sataloff classification\n
Day 120 +/- 7 days

Secondary Endpoints

Progression-Free Survival, as Determined by the Investigator According to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) Criteria
Baseline to death or progressive disease (PD), whichever occurred first (Median [full range] duration of follow-up in Pertuzumab vs. Placebo arms for Primary Analysis: 24.9 [0-41] vs. 21.3 [0-39] months; Final Analysis: 50.4 [0-70] vs. 47.4 [0-66] months)
Primary Analysis of the Percentage of Participants With Overall Objective Response, as Determined by the Investigator According to RECIST v1.1 Criteria
Baseline up to death or progressive disease, whichever occurred first (Median [full range] duration of follow-up in Pertuzumab vs. Placebo arms for Primary Analysis: 24.9 [0-41] months vs. 21.3 [0-39] months)
Final Analysis of the Percentage of Participants With Overall Objective Response, as Determined by the Investigator According to RECIST v1.1 Criteria
Baseline up to death or progressive disease, whichever occurred first (Median [full range] duration of follow-up in Pertuzumab vs. Placebo arms for Final Analysis: 50.4 [0-70] months vs. 47.4 [0-66] months)
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Pertuzumab + Trastuzumab + ChemotherapyEXPERIMENTALParticipants will receive pertuzumab in combination with trastuzumab and chemotherapy (cisplatin and fluoropyrimidine \[capecitabine or 5-fluorouracil\]) for the first 6 treatment cycles (cycle length = 21 days). Thereafter, participants will continue to receive pertuzumab and trastuzumab until disease progression, occurrence of unacceptable toxicity, or withdrawal from the study for another reason.
Placebo + Trastuzumab + ChemotherapyPLACEBO_COMPARATORParticipants will receive placebo in combination with trastuzumab and chemotherapy (cisplatin and fluoropyrimidine \[capecitabine or 5-fluorouracil\]) for the first 6 treatment cycles (cycle length = 21 days). Thereafter, participants will continue to receive placebo and trastuzumab until disease progression, occurrence of unacceptable toxicity, or withdrawal from the study for another reason.
Herceptin IV + ChemotherapyACTIVE_COMPARATORParticipants will receive Herceptin via IV infusion for 8 cycles prior to surgery and an additional 10 cycles after surgery. Docetaxel will be co-administered during Cycles 1 to 4; chemotherapy during Cycles 5 to 8 will include 5-fluorouracil, cyclophosphamide, and epirubicin. Herceptin IV will be given on Day 1 of each 21-day cycle, as 8 milligrams per kilogram (mg/kg) for a loading dose during Cycle 1 and as 6 mg/kg during subsequent cycles.
Herceptin SC + ChemotherapyEXPERIMENTALParticipants will receive Herceptin via SC injection for 8 cycles prior to surgery and an additional 10 cycles after surgery. Docetaxel will be co-administered during Cycles 1 to 4; chemotherapy during Cycles 5 to 8 will include 5-fluorouracil, cyclophosphamide, and epirubicin. Herceptin SC will be given on Day 1 of each 21-day cycle, as a 600-milligram (mg) fixed dose.
BevacizumabEXPERIMENTALParticipants received intravenous (IV) bevacizumab 7.5 mg/kg every 3 weeks, oral capecitabine 1,000 mg/m˄2 twice daily for 14 days every 3 weeks, or 5-fluorouracil (5-FU) at a dose of 800 mg/m˄2/day as a continuous IV infusion over the first 5 days of every 3 week cycle, and cisplatin 80 mg/m˄2 as an IV infusion every 3 weeks for a maximum of 6 cycles. Bevacizumab and capecitabine/5-FU were administered until disease progression or unacceptable toxicity.
PlaceboPLACEBO_COMPARATORParticipants received intravenous (IV) placebo infusion every 3 weeks, oral capecitabine 1,000 mg/m˄2 twice daily for 14 days every 3 weeks, or 5-fluorouracil (5-FU) at a dose of 800 mg/m˄2/day as a continuous IV infusion over the first 5 days of every 3 week cycle, and cisplatin 80 mg/m˄2 as an IV infusion every 3 weeks for a maximum of 6 cycles. The placebo and capecitabine/5-FU were administered until disease progression or unacceptable toxicity.
FOLFOX4ACTIVE_COMPARATORWeeks 1-24: Oxaliplatin was administered as an 85 mg/m\^2 intravenous infusion over 2 hours concomitantly with leucovorin as a 200 mg/m\^2 infusion over 2 hours, followed by 5-FU, given as a 400 mg/m\^2 bolus injection, and then as a 600 mg/m\^2 continuous infusion over 22 hours. Leucovorin 200 mg/m\^2 (alone), followed by 5-FU 400 mg/m\^2 bolus injection, and 5-FU 600 mg/m\^2 continuous infusion were repeated on day 2. Cycle length was 2 weeks and cycles were repeated every second week for a total of 12 cycles (24 weeks). Weeks 25-48: Observation only.
FOLFOX4 + BvEXPERIMENTALWeeks 1-24: Bevacizumab 5 mg/kg was administered as an intravenous infusion over 30 - 90 minutes followed by oxaliplatin, administered as an 85 mg/m\^2 intravenous infusion over 2 hours (on day 1 only) concomitantly with leucovorin, as a 200 mg/m\^2 infusion over 2 hours, followed by 5-FU, given as a 400 mg/m\^2 bolus injection, and then as a 600 mg/m\^2 continuous infusion over 22 hours. Leucovorin 200 mg/m\^2 (alone), followed by 5-FU 400 mg/m\^2 bolus injection, and 5-FU 600 mg/m\^2 continuous infusion are repeated on day 2. Cycle length is 2 weeks and cycles were repeated every second week for a total of 12 cycles (24 weeks). Weeks 25-48: Bevacizumab 7.5 mg/kg was administered as an intravenous infusion over 30 minutes. Cycle length was 3 weeks. Cycles were repeated every 3 weeks for a total of 8 cycles (24 weeks).
XELOX+BvEXPERIMENTALWeeks 1-24: Bevacizumab 7.5 mg/kg was administered as an intravenous infusion over 30 - 90 minutes followed by oxaliplatin administered as a 130 mg/m\^2 intravenous infusion over 2 hours (day 1 every 3 weeks) in combination with capecitabine, which was administered orally at a dose of 1000 mg/m\^2 twice daily (equivalent to a total daily dose of 2000 mg/m\^2), with first dose the evening of day 1 and last dose the morning of day 15, given as intermittent treatment (3-week cycles consisting of 2 weeks of treatment followed by 1 week without treatment), for a total of 8 cycles (24 weeks). Weeks 25-48: Bevacizumab 7.5 mg/kg was administered as an intravenous infusion over 30 minutes. Cycle length was 3 weeks. Cycles were repeated every 3 weeks for a total of 8 cycles (24 weeks).
5-Fluorouracil/Leucovorin (5-FU/LV)ACTIVE_COMPARATORParticipants were given one of two regimens (each participating center prespecified which regimen they would use for all patients at that center): i) Mayo Clinic regimen group: LV 20 mg/m\^2 IV bolus injection + 5-FU 425 mg/m\^2 IV bolus injection daily on Days 1-5 of a four-week cycle, for a total of six cycles (24 weeks), or; ii) Roswell Park regimen group: LV 500 mg/m\^2 by two-hour IV infusion + 5-FU 500 mg/m\^2 IV bolus injection one hour after the start of the LV infusion on Day 1 of Weeks 1 to 6 of each eight-week cycle, for a total of four cycles (32 weeks).
Capecitabine in Combination with Oxaliplatin (XELOX)EXPERIMENTALCapecitabine was administered as an oral twice daily outpatient intermittent treatment (3-week cycles consisting of two weeks of treatment followed by one week without treatment) combined with intravenous (IV) oxaliplatin on Day 1 of each cycle. Capecitabine was administered orally at a dose of 1000 mg/m\^2 twice-daily (equivalent to a total daily dose of 2000 mg/m\^2) with the first dose given during the evening of Day 1 and last dose given during the morning of Day 15. Oxaliplatin was administered as a 130 mg/m\^2 IV infusion over two hours on Day 1 of each cycle. The XELOX combination was administered for a total of eight cycles (24 weeks).
5-Fluorouracil + CisplatinACTIVE_COMPARATORParticipants will receive 5-FU in combination with cisplatin upto disease progression.
Capecitabine + CisplatinEXPERIMENTALParticipants will receive capecitabine in combination with cisplatin upto disease progression.
CapecitabineEXPERIMENTALParticipants received capecitabine 1250 milligram per square meter (mg/m \^ 2) orally, twice a day, for 14 days, followed by a 7-day rest period without treatment, as an intermittent therapy in a 3-week cycle for 8 cycles (24 weeks).
5-Fluorouracil + LeucovorinACTIVE_COMPARATORParticipants received leucovorin 20 mg/m \^ 2 followed by 5-fluorouracil at 425 mg/m \^ 2, by rapid intravenous injection, daily, from Days 1 to 5 of the first week in each 4-week cycle for 6 cycles (24 weeks).
Cohort A: ddAC, Paclitaxel, Pertuzumab, TrastuzumabEXPERIMENTALParticipants received neoadjuvant treatment with dose-dense doxorubicin and cyclophosphamide (ddAC), with administration of doxorubicin 60 milligrams per square meter (mg/m\^2) intravenously (IV) once every 2 weeks (q2w) and cyclophosphamide 600mg/m\^2 IV q2w for 4 cycles, followed by paclitaxel 80mg/m\^2 IV once weekly (qw) for 12 weeks. Pertuzumab (840 milligrams \[mg\] IV loading dose then 420mg IV q3w) and trastuzumab (8 milligrams per kilogram \[mg/kg\] IV loading dose then 4mg/kg IV q3w) were administered along with paclitaxel for 4 cycles (8 cycles of chemotherapy in total prior to surgery). Following surgery, participants received adjuvant treatment with pertuzumab and trastuzumab IV q3w (up to 13 cycles), for a total of 17 cycles of pertuzumab and trastuzumab therapy during the study. Radiotherapy and adjuvant hormonal therapy were also given as clinically indicated. Following treatment completion/discontinuation, participants were followed for safety and efficacy for up to 5 years.
Cohort B: FEC, Docetaxel, Pertuzumab, TrastuzumabEXPERIMENTALParticipants received neoadjuvant treatment with 5-fluorouracil, epirubicin, and cyclophosphamide (FEC), with administration of 5-fluorouracil 500mg/m\^2 intravenously (IV) q3w, epirubicin 100mg/m\^2 IV q3w, and cyclophosphamide 600mg/m\^2 IV q3w for 4 cycles, followed by docetaxel (with starting dose of 75mg/m\^2 in Cycle 5, then 100mg/m\^2 for Cycles 6-8) q3w for 4 cycles. Pertuzumab (840 mg IV loading dose then 420mg IV q3w) and trastuzumab (8 mg/kg IV loading dose then 4mg/kg IV q3w) were given along with doectaxel for 4 cycles (8 cycles of chemotherapy in total prior to surgery). Following surgery, participants received adjuvant treatment with pertuzumab and trastuzumab IV q3w (up to 13 cycles), for a total of 17 cycles of pertuzumab and trastuzumab therapy during the study. Radiotherapy and adjuvant hormonal therapy were also given as clinically indicated. Following treatment completion/discontinuation, participants were followed for safety and efficacy for up to 5 years.
Ipatasertib + mFOLFOX6EXPERIMENTALParticipants will receive oral ipatasertib on Days 1 to 7 of each 14-day cycle in combination with mFOLFOX6, administered on Day 1 of each cycle.
Placebo + mFOLFOX6PLACEBO_COMPARATORParticipants will receive the placebo equivalent to ipatasertib on Days 1 to 7 of each 14-day cycle in combination with mFOLFOX6, administered on Day 1 of each cycle.
FOLFIRI + CetuximabACTIVE_COMPARATOR -
FOLFIRI + MEHD7945AEXPERIMENTAL -
MEGF0444A + mFOLFOX-6 + BevacizumabEXPERIMENTALParticipants will receive MEGF0444A + mFOLFOX-6 (oxaliplatin, folinic acid, and 5-fluorouracil) regimen + bevacizumab. Participants will receive oxaliplatin for up to 8 cycles (Cycle = 14 days) and 5-fluorouracil, folinic acid, bevacizumab and MEGF0444A will be administered until disease progression or unacceptable toxicity for a maximum of up to 52 cycles.
Placebo + mFOLFOX-6 + BevacizumabPLACEBO_COMPARATORParticipants will receive MEGF0444A matching placebo + mFOLFOX-6 regimen + bevacizumab. Participants will receive oxaliplatin for up to 8 cycles and 5-fluorouracil, folinic acid, bevacizumab and placebo will be administered until disease progression or unacceptable toxicity for a maximum of up to 52 cycles.
Bevacizumab + mFOLFOX-6EXPERIMENTALParticipants will receive combination therapy of bevacizumab 5 mg/kg IV dose and mFOLFOX-6 (Levofolinic acid, 5-FU and oxaliplatin) on Day 1 of every 2 weeks' cycle for 5 cycles (Cycle 1-5), followed by 1 cycle (Cycle 6) of mFOLFOX6 alone (preoperative treatment phase). After 3 weeks of preoperative treatment phase, participants satisfying the surgical criteria for hepatic resectability will undergo a liver metastasectomy. Thereafter participants will receive combination therapy of mFOLFOX-6 + bevacizumab for another 6 cycles (Cycle 7-12); (post-operative treatment phase) followed by bevacizumab alone for 52 weeks (26 cycles) (maintenance therapy).
Bevacizumab + mFOLFOX6EXPERIMENTALParticipants will receive bevacizumab plus mFOLFOX6 by intravenous (IV) infusion on Day 1 of each 2-week cycle until disease progression or unacceptable toxicity. Participants may be transitioned to oral capecitabine in the event of unacceptable toxicity to oxaliplatin, with bevacizumab continued in 3-week cycles.
Bevacizumab + FOLFIRIEXPERIMENTALParticipants will receive bevacizumab plus FOLFIRI by IV infusion on Day 1 of each 2-week cycle until disease progression or unacceptable toxicity. Participants may be transitioned to oral capecitabine in the event of unacceptable toxicity to irinotecan, with bevacizumab continued in 3-week cycles.
Arm A (Bevacizumab, Induction Chemotherapy, Chemoradiotherapy)EXPERIMENTALIn this arm, participants will undergo 3 phases of treatment. During the Phase 1, participants will receive induction chemotherapy with 6 two-week cycles of bevacizumab + Folfox-4 (5-FU + oxaliplatin + folinic acid) for 12 weeks followed by a treatment-free interval of 3 to 4 weeks. The Phase 2 will include 7 weeks of bevacizumab + chemoradiotherapy (intravenous \[IV\] infusion of bevacizumab alone, 2 weeks before administration of the first cycle of chemoradiotherapy, then 5 one-week cycles of chemoradiotherapy \[5-FU + radiotherapy\], with administration of bevacizumab every two weeks \[Cycles 1, 3 and 5\]) followed by a treatment-free interval of 6 to 8 weeks. The Phase 3 will be surgery involving a radical rectal excision using the total mesorectal excision (TME) technique.
Arm B (Bevacizumab, Chemoradiotherapy)EXPERIMENTALIn this arm, participants will receive the Phase 2 and Phase 3 treatments only. The phase 2 will include 7 weeks of bevacizumab + chemoradiotherapy (IV infusion of bevacizumab alone, 2 weeks before administration of the first cycle of chemoradiotherapy, then 5 one-week cycles of chemoradiotherapy \[5-FU + radiotherapy\], with administration of bevacizumab every two weeks \[Cycles 1, 3 and 5\]) followed by a treatment-free interval of 6 to 8 weeks. The phase 3 will be surgery involving a radical rectal excision using the TME technique.
Single ArmEXPERIMENTAL -
5-Fluorouracil + Epirubicin + CyclophosphamideACTIVE_COMPARATORNeoadjuvant treatment (Period 1): Participants will receive 4 cycles (cycle length = 21 days) of 5-FU + epirubicin + cyclophosphamide. Surgery will be performed 5 (+/- 1) weeks after the last cycle. Adjuvant treatment (Period 2): Participants will receive 4 cycles (cycle length = 21 days) of docetaxel.
Capecitabine + Epirubicin + CyclophosphamideEXPERIMENTALNeoadjuvant treatment (Period 1): Participants will receive 4 cycles (cycle length = 21 days) of capecitabine + epirubicin + cyclophosphamide. Surgery will be performed 5 (+/- 1) weeks after the last cycle. Adjuvant treatment (Period 2): Participants will receive 4 cycles (cycle length = 21 days) of docetaxel.

Interventions

NameTypeDescription
5-FluorouracilDRUGParticipants will receive 5-fluorouracil 800 milligrams per meter square (mg/m\^2)/24 hour IV infusion for 120 hours (Days 1-5) q3w for 6 cycles.
CapecitabineDRUGParticipants will receive capecitabine 1000 mg/m\^2 orally twice daily, evening of Day 1 to morning of Day 15 (28 doses) q3w for 6 cycles.
CisplatinDRUGParticipants will receive cisplatin 80 mg/m\^2 IV q3w for 6 cycles.
PertuzumabDRUGParticipants will receive pertuzumab 840 mg IV q3w until disease progression, occurrence of unacceptable toxicity, or withdrawal from the study for another reason.
PlaceboDRUGParticipants will receive placebo (matched to pertuzumab) IV q3w until disease progression, occurrence of unacceptable toxicity, or withdrawal from the study for another reason.
TrastuzumabDRUGParticipants will receive 8 mg/kg IV initial dose on Day 1, followed by 6 mg/kg IV q3w until disease progression, occurrence of unacceptable toxicity, or withdrawal from the study for another reason.
CarboplatinDRUGCarboplatin will be administered as per the schedule specified in the respective arm.
CyclophosphamideDRUGCyclophosphamide will be administered as per the schedule specified in the respective arm.
DocetaxelDRUGDocetaxel will be administered as per the schedule specified in the respective arm.
DoxorubicinDRUGDoxorubicin will be administered as per the schedule specified in the respective arm.
EpirubicinDRUGEpirubicin will be administered as per the schedule specified in the respective arm.
PaclitaxelDRUGPaclitaxel will be administered as per the schedule specified in the respective arm.
Herceptin IV [trastuzumab]DRUGHerceptin will be administered as 8 mg/kg (loading dose during Cycle 1) and 6 mg/kg (subsequent cycles) via IV infusion on Day 1 of each 21-day cycle for a total of 18 cycles.
Herceptin SC [trastuzumab]DRUGHerceptin will be administered as fixed dose 600 mg SC on Day 1 of each 21-day cycle for a total of 18 cycles.
BevacizumabDRUGIntravenous bevacizumab 7.5 mg/kg once every 3 weeks, given until disease progression or unmanageable toxicity.
5-Fluorouracil (5-FU)DRUGAdministered as either a bolus injection or continuous intravenous infusion over 22 hours.
Leucovorin calciumDRUGAdministered as a 200 mg/m\^2 infusion over 2 hours.
OxaliplatinDRUGAdministered as an intravenous infusion over 2 hours.
Leucovorin (LV)DRUGAdministered by one of two regimens, as specified in the arm description.
LeucovorinDRUG20mg/m2 iv daily from day 1 to day 5 every 28 days.
Capecitabine [Xeloda]DRUG1250mg/m2 po bid on days 1-14 every 21 days.
IpatasertibDRUGParticipants will receive ipatasertib, 600 milligrams (mg) orally once daily on Days 1 to 7 of each 14-day cycle until disease progression or unacceptable toxicity.
CetuximabDRUGCetuximab 400 mg/m\^2 intravenous infusion as a loading dose on Day 1 Cycle 1, followed by 250 mg/m\^2 intravenous infusion weekly until documented disease progression or unacceptable toxicity.
IrinotecanDRUGStandard Irinotecan chemotherapy (180 milligram per square meter \[mg/m\^2\] administered as intravenous infusion over 60 +/- 30 minutes) or according to local standard-of-care prescribing information's, every 2 weeks until documented disease progression or unacceptable toxicity.
MEHD7945ADRUGMEHD7945A 1100 milligram (mg) intravenous infusion every 2 weeks until documented disease progression or unacceptable toxicity.
Folinic acidDRUGParticipants will receive folinic acid 400 mg/m\^2 IV infusion over 2 hours on Day 1 of every cycle until disease progression, unacceptable toxicity or at the maximum of 52 cycles, whichever occurs first.
MEGF0444ADRUGParticipants will be administered MEGF0444A at a fixed dose of 400 milligrams (mg) IV on Day 1 of Cycle 1, followed by subsequent doses of 400 mg every 14 days until disease progression or unacceptable toxicity, for a maximum of 52 cycles, whichever occurs first.
Levofolinic acidDRUGParticipants will receive levofolinic acid 200 mg/m\^2 IV infusion over 2 hours on Day 1 of each 2 weeks' cycle up to 12 cycles.
EpidoxorubicinDRUG90 mg/m\^2 as an i.v. infusion over 1 hour every 3 weeks for 4 cycles.
Preoperative RadiotherapyRADIATIONRadiotherapy will be delivered in fraction of 1.8 gray per day (Gy/day), 5 days a week for 5 weeks, i.e., a total dose of 45 Gy will be administered in 25 fractions over a period of 33 days.
SurgeryPROCEDURERadical rectal excision based on the TME technique.
bevacizumab [Avastin]DRUG5 mg/kg intravenously every 2 weeks, 4 cycles
Radiation therapyRADIATIONTotal dose of 45 Gy over 38 days
Mesorectal excisionPROCEDURE6-8 weeks after completion of neoadjuvant treatment
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites172

Inclusion Criteria: * Histologically confirmed metastatic adenocarcinoma of the stomach or GEJ * Measurable or evaluable non-measurable disease as assessed by the investigator according to RECIST v1.1 criteria * Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 * Life expectancy g...

Countries:United StatesAustraliaAustriaBelgiumBrazilBulgariaCanadaChinaCroatiaEl SalvadorFinlandGermanyGuatemalaHungaryItalyJapanKazakhstanMalaysiaMexicoNetherlandsNorth MacedoniaPanamaPeruPolandRomaniaRussiaSouth KoreaSpainSwitzerlandTaiwanThailandTurkey (Türkiye)ArgentinaChileColombiaCzechiaDenmarkFranceHong KongIrelandIsraelNew ZealandPhilippinesSloveniaSouth AfricaSwedenUkraineUnited KingdomEstoniaSlovakiaGreecePortugalSingaporeBelizeUruguayNorway
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Frequently asked questions about 5-Fluorouracil

What is 5-Fluorouracil used for?

5-Fluorouracil is an oncology small molecule being studied for gastric cancer, rectal cancer, adenocarcinoma, metastatic colorectal cancer, colorectal cancer, and breast cancer. It is currently in Phase 3 clinical development and is being developed by Roche Holding AG (RHHBY).

Who makes 5-Fluorouracil?

5-Fluorouracil is being developed by Roche Holding AG, which trades under the ticker RHHBY. The drug is an oncology small molecule in Phase 3 clinical development for multiple cancer indications including gastric, rectal, and breast cancer.

What phase is 5-Fluorouracil in?

5-Fluorouracil is in Phase 3 clinical development. It is an investigational oncology small molecule being studied for gastric cancer, rectal cancer, adenocarcinoma, metastatic colorectal cancer, colorectal cancer, and breast cancer. It has not been reported as FDA approved.

What clinical trials is 5-Fluorouracil in?

5-Fluorouracil has been studied in seven completed clinical trials with a total enrollment of 7,955 participants. Notable trials include NCT00548548 in adenocarcinoma, NCT00559845 in breast cancer, NCT02132949 in breast cancer, and NCT02846428 in breast cancer. All trials are completed.

Is 5-Fluorouracil the same as capecitabine?

5-Fluorouracil is distinct from capecitabine, though both are used in combination in clinical trials. For example, NCT00548548 studies capecitabine with other agents, and NCT02846428 evaluates capecitabine alone. 5-Fluorouracil itself is the drug being developed by Roche.