GIST With D842V Mutated PDGFRA Gene
Conditions
Brief summary
This is a multicenter, randomized, double-blinded, placebo-controlled, trial of oral crenolanib versus oral placebo in combination with best supportive care in subjects with advanced or metastatic GIST with a D842V mutation in the PDGFRA gene. Approximately 120 subjects will be randomized in a 2:1 ratio to receive either crenolanib 100 mg or matching placebo orally (PO) 3 times daily (TID) in combination with best supportive care.
Interventions
DRUGCrenolanib
DRUGPlacebo
Sponsors
Centre Leon Berard
Fox Chase Cancer Center
Arog Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Histologically or cytologically-confirmed advanced or metastatic GIST with a D842V mutation in the PDGFRA gene as determined by central laboratory testing 2. Measurable disease as per modified RECIST 1.1 • A lesion in an area that was previously treated with local therapy (e.g. radiation, surgery, or cryotherapy) can be considered measurable disease as long as there is objective evidence of progression of the lesion prior to randomization 3. Subjects (male or female) ≥ 18 years of age 4. Female subjects with reproductive potential must have negative serum or urine pregnancy test 5. Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
Exclusion criteria
1. Severe liver disease (e.g. cirrhosis, non-alcoholic steatohepatitis, sclerosing cholangitis) 2. Known, active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV) 3. Female subject who is pregnant or breastfeeding, or planning to become pregnant within 30 days after ending treatment 4. Systemic anti-cancer treatment (e.g. chemotherapy, tyrosine kinase inhibitors, immunotherapy, or investigational agents) or investigational device within 3 weeks or 5 half-lives (if the drug's half-life in subject is known) prior to randomization, whichever is shorter
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Progression-free survival (PFS) will be measured from the date of randomization to the date of the first objective radiological disease progression according to centralized committee assessment using modified RECIST version 1.1 or death. | 3 years |
Secondary
| Measure | Time frame |
|---|---|
| Overall survival (OS) will be measured from the date of randomization to the date of death from any cause. OS will be estimated using the Kaplan-Meier method. | 3 years |
Countries
France, Germany, Italy, Norway, Poland, Spain, United States
Outcome results
None listed