Actively Recruiting
Phase 2 Study of Avapritinib for CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors
Led by M.D. Anderson Cancer Center · Updated on 2026-03-02
50
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating avapritinib, an oral drug, for patients with malignant solid tumors that have specific genetic mutations in CKIT or PDGFRA. These tumors are locally advanced or have spread to other parts of the body (metastatic). The study aims to assess the response rate, duration of response, disease control, safety, and overall survival in patients with these mutations. It also explores treatment effects on brain or central nervous system metastases and analyzes genetic markers related to clinical outcomes. Participants receive avapritinib by mouth once daily in 28-day cycles, continuing as long as the disease does not progress or unacceptable side effects occur. The study includes patients with various solid tumors, including melanoma, breast, lung, gastroesophageal, colorectal cancers, sarcoma, and primary central nervous system tumors. After treatment ends, participants are followed up 30 days later and then every eight weeks to monitor health and treatment effects. During the trial, patients undergo regular assessments including imaging scans based on RECIST or RANO criteria to measure tumor response. Safety is monitored using standardized criteria for adverse events. Researchers also collect tissue samples to confirm genetic mutations and may perform biopsies as required. The primary outcome is the overall response rate up to 50 months, with secondary outcomes including duration of response, disease control rate, and incidence of side effects. Participants are expected to comply with scheduled visits and examinations throughout the study duration.
CONDITIONS
Brief Title
Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient provides written informed consent for the study.
- Male or female aged 18 years or older; adolescents 12 years and older allowed with assent and parental consent.
- Has a locally advanced or metastatic solid tumor with progression after standard therapy, no meaningful benefit from standard therapy, no available standard therapy, or declined standard therapy.
- Includes melanoma, breast, lung, gastroesophageal, colorectal cancers, sarcoma, solid tumors NOS, and primary CNS tumors (excluding GIST).
- Measurable disease by RECIST v1.1 or RANO criteria.
- Documented pathogenic CKIT or PDGFRA activating mutation confirmed by certified genetic testing.
- Available archival tissue for mutation testing.
- Adequate organ and marrow function within 7 days before treatment.
- Cardiac ejection fraction greater than 45%.
- ECOG performance status 0-2.
- Life expectancy of at least 3 months.
- Willing and able to comply with study protocol including visits and exams.
- Willing to undergo biopsy if required.
- Females must be postmenopausal, surgically sterile, or have a negative pregnancy test.
- Females of childbearing potential must use effective contraception or abstain during and 6 weeks after treatment.
- Males with female partners of reproductive potential must use contraception or abstain during and 30 days after treatment.
You will not qualify if you...
- Diagnosis of gastrointestinal stromal tumor (GIST).
- Resistance to tyrosine kinase inhibitors with CKIT mutations V654A or T670I.
- Presence of meningeal carcinomatosis, spinal cord compression, or symptomatic/unstable brain metastases.
- History of serious congestive heart failure or cardiac arrhythmias requiring treatment.
- QT interval over 470 msec.
- Participation in another investigational study within 2 weeks before treatment.
- Recent anticancer treatment or major surgery within 2 weeks before treatment.
- Symptomatic non-healing wounds, ulcers, gastrointestinal perforation, or bone fractures.
- History of psychotic or depressive disorder unless stable and controlled for 12 months.
- Use of strong CYP3A4 inhibitors or inducers without required washout.
- Pregnancy or breastfeeding.
- Unable to swallow or retain oral medications.
- Gastrointestinal conditions affecting drug absorption.
- Additional progressing malignancies requiring active treatment except certain skin or cervical cancers.
- Any condition or abnormality that may interfere with study participation.
- Prior intracerebral agent or bevacizumab treatment (Cohort 3 only).
- Prior treatment for low-grade glioma (Cohort 3 only).
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive avapritinib orally once daily in repeated 28-day cycles until disease progression or unacceptable toxicity.
Visits occur every 28 days for treatment cycles
Duration - Up to approximately 50 months
Participants are followed up after treatment completion to monitor safety and survival outcomes.
1 visit at 30 days after treatment, then visits every 8 weeks
Trial Site Locations
Total: 1 location
1
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
J
Jordi R Rodon, MD, PHD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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