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BCNU

Phase 1

Pancreatic Adenocarcinoma Metastatic | Small molecule | Oncology |Myriad Genetics, Inc.|Last Updated: Jan 29, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04150042SHARON: A Clinical Trial for Metastatic Cancer Using Chemotherapy and Patients' Own Stem CellsPHASE1 RECRUITING 24Jan 13, 2021Dec 1, 2028Jan 29, 20262 United States
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Study Endpoints
Primary Endpoints
Rate of Sinusoidal obstruction syndrome
30 days after treatment

Sinusoidal obstruction syndrome diagnosis and grading will use the European Society for Blood and Marrow Transplantation's Revised Diagnosis and Severity Criteria for Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease in Adult Patients as published in 2016. Gradings are from mild to very severe (multi-organ dysfunction/multi-organ failure).

Rate of Idiopathic or Non-Infective Pulmonary Toxicity ≥ Grade 3
3 months after the last treatment

The American Thoracic Society Committee on Idiopathic Pneumonia Syndrome definition will be employed.

Rate of Presumptive Oxalate Nephropathy
Within 48 hours of vitamin C treatment

Oxalate nephropathy will be presumed if there is acute kidney injury or increased creatinine, grade 3 or higher by the criteria of CTCAE Version 5.0 within 48 h of the administration of vitamin C, in the absence of a clear alternative explanation (an example of an alternative explanation is tumor lysis syndrome).

Rate of Cytokine Release Syndrome ≥ Grade 3
Within 48 hours of each vitamin C treatment

Cytokine release syndrome will be assessed by the criteria of CTCAE Version 5.0. Elevation of plasma cytokine levels consistent with the diagnosis of cytokine release syndrome must be present.

Rate of Mucositis ≥ Grade 3
Day 7 after each treatment

Mucositis will be assessed using the WHO Mucositis Scale. Grading is from 0 (no symptoms) to 4 (no possible alimentation).

Rate of Delayed Engraftment of Neutrophils
Day 21 after each treatment

Neutrophil engraftment is defined as an absolute neutrophil count ≥ 500/microliter for 3 days, with the date of engraftment being the first of those 3 days. Delayed engraftment is engraftment that occurs after 21 days but within 30 days.

Rate of Failed Engraftment of Neutrophils
Day 30 after each treatment

Neutrophil engraftment is defined as an absolute neutrophil count ≥ 500/microliter for 3 days, with the date of engraftment being the first of those 3 days. Failure to engraft within 30 days will be considered an engraftment failure.

Rate of Delayed Engraftment of Platelets
Day 30 after each treatment

Platelet engraftment is defined as a platelet count ≥ 20,000/microliter for 3 days, with the date of engraftment being the first of those 3 days. Delayed engraftment is engraftment that occurs after 30 days.

Overall incidence rate of adverse events
Until 12 months after the second stem cell treatment

Adverse event is defined any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related.

Overall incidence rate of serious adverse events
Until 12 months after the second stem cell treatment

An adverse event is considered serious if, in the view of either the investigator or Sponsor, it results in any of the following outcomes: * Death. * A life-threatening adverse event. * Inpatient hospitalization or prolongation of existing hospitalization. * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly or birth defect. Important medical events that may not result in death, be life-threatening, or require hospitalization may be considered serious when, based upon appropriate medical judgment, they may jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the outcomes listed in this definition.

Overall incidence rate of Grade 3-5 adverse events
Until 12 months after the second stem cell treatment

Grading will be measured using Common Terminology Criteria for Adverse Events version 5.0

Secondary Endpoints
Objective response according to RECIST version 1.1
1 month after the first stem cell treatment
Objective response rate in metastatic lesions
1 month after the first stem cell treatment
Overall Survival
Until 12 months after the second stem cell treatment
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Chemotherapy/stem cell treatmentEXPERIMENTAL -
Interventions
NameTypeDescription
MelphalanDRUGIntravenous melphalan (to be given in conjunction with the other listed drugs).
BCNUDRUGIntravenous BCNU (to be given in conjunction with the other listed drugs).
Vitamin B12BDRUGIntravenous vitamin B12b (to be given in conjunction with the other listed drugs).
Vitamin CDRUGIntravenous vitamin C (to be given in conjunction with the other listed drugs).
Autologous Hematopoietic Stem CellsDEVICEAfter each cycle of chemotherapy, participants will receive an autologous hematopoietic stem cell infusion.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria * Age ≥ 18 years. * Pancreatic or breast cancer, as described below. * Stage IV (based on AJCC staging guidelines) at the time of enrollment. a. Note that potential subjects with stage IV cancer that have had a complete response from prior chemotherapy are still potentially el...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04150042primaryCompletionDate: changed
LOWMay 24, 2026NCT04150042studyFirstPostDate: changed