Recent Updates
Recently added Catalysts

Nilotinib, Imatinib

Phase 3

Gastrointestinal Stromal Tumors | Small molecule | Oncology |Novartis AG|Last Updated: Aug 19, 2024

Target and mechanism

Molecular targetABL1, BCR
Target classInhibitor
ModalitySmall molecule

Also known as Nilotinib, Nilotinib 100mg

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedACTIVE_CONTROLLEDDMC
Total Trials4
Total Enrollment402

FDA Designations

No designations recorded

Clinical trial landscape

Nilotinib, Imatinib · 4 trials · 1 indication

Phase 3 2Phase 2 1Phase 1 1
NCT01289028Efficacy of Nilotinib in Adult Patients With Gastrointestinal Stromal Tumors Resistant to Imatinib and Sunitinib.Gastrointestinal Stromal Tumors
COMPLETED125 Analytics
NCT00471328Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and SunitinibGastrointestinal Stromal Tumors
COMPLETED248 Analytics
PHASE3COMPLETED
Efficacy of Nilotinib in Adult Patients With Gastrointestinal Stromal Tumors Resistant to Imatinib and Sunitinib.
Gastrointestinal Stromal TumorsUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib
Gastrointestinal Stromal TumorsUnlock trial analytics

Study Endpoints

Primary Endpoints

Percent of Patients Achieving Stable Disease (SD)
During the first 4 months

Neither sufficient shrinkage to qualify for Partial Response nor sufficient increase to qualify for Progression Disease, taking as reference the smallest sum of the longest diameter since the treatment started.

Percent of Patients Achieving Partial Response (PR)
during the first 4 months

The primary efficacy variable was defined as the proportion of patients with a best overall response of CR by Week 16/Month 4 based on local assessment according to RECIST (Version 1.0). This is an at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum longest diameter.

Percent of Patients Achieving Complete Response (CR)
during the first 4 months

Complete response (CR) is the Disappearance of all target lesions.

Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008)
Up to 16 months

Progression-free survival (PFS) is the time from date of randomization to the date of first documented progression or death due to any cause. If a participant has not had an event, progression-free survival is censored at the time of last adequate tumor assessment. Progression is defined according to Modified Response Evaluation Criteria in Solid Tumors (modified RECIST) criteria as at least a 20% increase in the sum of the longest diameter of target lesions, worsening of the non-target lesions or the appearance of one or more new lesions.

Progression-free Survival (PFS) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set
Up to 34 months

PFS is defined as the time from the first date of cross-over to nilotinib therapy from the control arm to the date of the first observation of documented disease progression. Tumor assessment was based on the local investigator's measurement using Modified Response Evaluation Criteria in Solid Tumors (modified RECIST) criteria. Progression is defined according to modified RECIST criteria as at least a 20% increase in the sum of the longest diameter of target lesions, worsening of the non-target lesions or the appearance of one or more new lesions.

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse events were reported from enrolment until 30 days after last dose of study treatment, up to approximately 10.3 years.

Number of participants with adverse events (AEs) (any AE regardless of seriousness), serious adverse events (SAEs), Fatal SAEs and treatment related AEs.

Safety and tolerability of nilotinib either as single agent or in combination with Imatinib
AE monitored continuously from day 1 cycle 1 to the study completion visit; safety laboratory and cardiac safety monitored every 3 treatment cycles (every 3 months) from day 1 cycle 1 to the study completion visit

Assessed by AE evaluation, including safety laboratory and cardiac safety (ECG, cardiac enzymes, ECHO) measures.

Secondary Endpoints

Analysis of Time to Overall Response (CR or PR) According to RECIST Using Kaplan-Meier Method for ITT Population
24 weeks and 52 weeks
Time to Overall Response (CR or PR): Per Protocol Population
24 weeks and 52 weeks
Time to Tumor Progression
during the first 4 months
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
NilotinibEXPERIMENTALnilotinib 400 mg twice daily (bid).
Control/cross-over to NilotinibACTIVE_COMPARATORIn core study phase, patients in this arm received Best Supportive Care (BSC) with or without imatinib or sunitinib at the last tolerated dose or at the investigator's choice until documented disease progression followed by cross-over to nilotinib arm. Patients entering the extension study on this control arm were permitted to cross over to nilotinib arm only upon documented disease progression.
Monotherapy: AMN107EXPERIMENTALinitial dose of imatinib (dose level 1) was 400 mg bid was administered orally on a continuous daily schedule and was not escalated during the study
Combination Therapy: AMN107 + ImatinibACTIVE_COMPARATORsix possible doses of Nilotinib (100 mg once daily (qd), 200 mg qd, 400 mg qd, 200 mg bid, 300 mg bid, and 400 mg bid). four possible doses of Imatinib (0 mg, 400 mg qd, 600 mg qd, and 400 mg bid).the initial dose of nilotinib (dose level 1) was 200 mg qd and could have been escalated up to 400 mg bid

Interventions

NameTypeDescription
NilotinibDRUG -
Best Supportive Care (BSC) +/- imatinib or sunitinibOTHERCan include pain medication, localized radiotherapy, nutritional support, and/or oxygen therapy and blood transfusions. Imatinib or sunitinib can be administered at the last tolerated dose and regimen or at the Investigator's choice.
Nilotinib, ImatinibDRUGNilotinib 50 mg and 200 mg gelatin capsules and Imatinib 100 mg and 400 mg film-coated tablets in bottles.. Medication labels for each study drug complied with the legal requirements of each country, were printed in the local language.
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites19

Inclusion Criteria: * Histologically confirmed diagnosis of GIST that is unresectable and/or metastatic and therefore not amenable to surgery or combined modality with curative intent. * Radiologically confirmed disease progression during imatinib therapy at a dose of at least 400 mg daily and/or r...

Countries:GermanyItalyUnited StatesAustraliaAustriaCanadaCzechiaFranceNetherlandsPolandSouth KoreaSpainSwitzerlandJapan
Unlock Eligibility Criteria

Frequently asked questions about Nilotinib, Imatinib

What is Nilotinib used for?

Nilotinib is used for Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Chronic Myelogenous Leukemia, Myelogenous Leukemia, Chronic, Gastrointestinal Stromal Tumor (GIST), Chronic Myeloid Leukemia, and Glioma. It is an investigational small molecule in Phase 3 clinical development for these oncology indications.

What does Nilotinib target?

Nilotinib is a kinase inhibitor, belonging to the -tinib class of targeted therapies. It works by inhibiting specific kinases involved in cancer cell growth and survival, particularly those implicated in Philadelphia Chromosome Positive leukemias. The drug is being studied for its activity against these molecular targets in various oncology settings.

Who makes Nilotinib?

Nilotinib is developed by Novartis AG, a global pharmaceutical company listed on the stock exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the safety and efficacy of Nilotinib across multiple cancer indications, including chronic myeloid leukemia and gastrointestinal stromal tumors.

What phase is Nilotinib in?

Nilotinib is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. The drug is being studied in multiple oncology indications, with a total of 10 clinical trials conducted, of which 9 are completed and 1 is active but not recruiting.

What clinical trials is Nilotinib in?

Nilotinib has been studied in several clinical trials. Notable ones include NCT01220648, a Phase 1 trial in pretreated Ph+ CML-CP patients; NCT01223898, a Phase 1 drug interaction study; NCT02253277, a Phase 1 combination trial with ruxolitinib; and NCT03906292, an active Phase 2 trial of frontline asciminib combination in chronic phase CML.

Is Nilotinib the same as Nilotinib 300 mg or Nilotinib 100mg?

Yes, Nilotinib 300 mg and Nilotinib 100mg refer to different dosage strengths of the same drug, Nilotinib. These are alternative names used to specify the particular dose formulation being administered in clinical studies or prescriptions. The active pharmaceutical ingredient is identical across these dosage forms.