Gastrointestinal Stromal Tumor (GIST)
Conditions
Keywords
PDGFRA mutations, KIT mutations
Brief summary
The purpose of this Phase 1/2 master protocol study is to evaluate if DCC-3009 is safe, tolerable and works effectively in the treatment of GIST. The study will use a modular approach with each module being defined according to therapy: DCC-3009 alone or DCC-3009 in combination with other anticancer therapies. Each module will be conducted in 2 parts: Part 1 (Dose Escalation) and Part 2 (Dose Expansion). Participants will be treated in 28-day treatment cycles with an estimated duration of up to 2 years.
Interventions
DRUGDCC-3009
Administered orally
Sponsors
Deciphera Pharmaceuticals, LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Module A Part 1 (Escalation): * Any participant with histologically or cytologically confirmed advanced/unresectable or metastatic GIST with documented KIT or platelet-derived growth factor receptor alpha (PDGFRA) mutation, who has progressed on or was intolerant to at least 1 approved tyrosine kinase inhibitor (TKI) regimen in the advanced/metastatic setting * Have at least 1 measurable lesion as defined by mRECIST, v1.1 * Have Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 or 1 * Adequate organ function, bone marrow function, and electrolytes * All participants agree to comply with the contraception requirements * Have a life expectancy of more than 3 months
Exclusion criteria
* Received systemic anticancer therapy or radiotherapy within 14 days prior to first dose of study drug * Prior or concurrent malignancy that requires treatment or is expected to require treatment for active cancer * Has known active central nervous system (CNS) metastases or an active primary CNS cancer * History or presence of clinically relevant cardiovascular abnormalities * Major surgery within 28 days of the first dose of study drug * Had systemic arterial thrombotic or embolic events within 6 months prior to the first dose of study drug * Had venous thrombotic events (e.g., deep vein thrombosis) or venous thrombotic embolic events (e.g., pulmonary embolism) within 1 month prior to the first dose of study drug * Known allergy or hypersensitivity to any component of the study drug * Malabsorption syndrome or other illness that could affect oral absorption * Any other clinically significant comorbidities
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Dose-Limiting Toxicities (DLT) (Part 1 Escalation) | Cycle 1 (28 Days) | DLTs assessed for each dose level. |
| Objective Response Rate (ORR) (Part 2 Expansion) | Baseline to Progressive Disease (PD), Death due to Any Cause, or Start of New Antitumor Therapy (Estimated up to 24 months) | ORR is the percentage of participants with confirmed complete response (CR) or confirmed partial response (PR) based on Modified Response Evaluation Criteria in Solid Tumors (mRECIST), v1.1. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Objective Response Rate (ORR) (Part 1 Escalation) | Baseline to Progressive Disease (PD), Death due to Any Cause, or Start of New Antitumor Therapy (Estimated up to 24 months) | ORR is the percentage of participants with confirmed complete response (CR) or confirmed partial response (PR) based on mRECIST, v1.1. |
| Duration of Response (DOR) | First Recorded CR or PR until PD or Death (Estimated up to 24 months) | DOR for participants with confirmed CR or confirmed PR based on mRECIST, v1.1, defined as the time interval from the time that the measurement criteria are first met for CR or PR (whichever is first recorded) until the first date that the disease progression is objectively documented or death, whichever occurs first. |
| Progression-Free Survival (PFS) | Initiation of Treatment to PD or Death (Estimated up to 24 months) | PFS is the time from initiation of treatment until documented disease progression per mRECIST, v1.1, or death, whichever occurs first. |
| Overall Survival (OS) | Initiation of Treatment to Death from Any Cause (Estimated up to 24 months) | OS is the time from initiation of treatment to the date of death from any cause. |
| Pharmacokinetics (PK): Maximum observed plasma drug concentration (Cmax) | Estimated up to 24 months | Cmax |
Countries
United States
Contacts
CONTACTClinical Team
STUDY_DIRECTORClinical Team
Deciphera Pharmaceuticals, LLC
Outcome results
None listed