Recruiting nowPhase 2
What this trial is trying to do
The researchers are testing whether avapritinib, a drug that blocks KIT or PDGFRA enzymes involved in cell growth, can shrink or control locally advanced or metastatic solid tumours carrying mutations in one of those genes. The trial includes several tumour types rather than colorectal cancer alone.
The idea is that blocking the altered enzyme may slow or reduce tumour growth when standard options are limited. The main measure is overall response rate—the proportion of participants whose tumours shrink by a specified amount—not how long people live or how long the cancer stays controlled.
Worth knowing: This is a small, non-randomised phase 2 trial with no comparison group, so tumour responses cannot be separated reliably from what might have happened with other treatment.
Written from the trial's own registry entry to explain what the researchers are testing and why. Nothing here is a result — this trial has not reported one — and it is not a view on whether the treatment works or on whether it would suit you.
| Registry number | NCT04771520 |
|---|---|
| Recruitment | Recruiting now |
| Phase | Phase 2 |
| Design | open label |
| Taking part | 50 people (planned) |
| Who can join | aged 18 Years and over |
| Run by | M.D. Anderson Cancer Center |
| Started | |
| Main results expected | |
| Record last updated |
What is being tested
- Drug Avapritinib
What it is measuring
Overall response rate (ORR)
This is the trial's main question — the one it is designed and sized to answer. Anything else it reports is a secondary finding, and secondary findings are far more likely to be chance.
Phase 2. Tests whether the treatment works well enough to be worth a larger trial. Usually too small to prove it changes survival.
How the researchers describe it
This phase II trial studies the effect of avapritinib in treating malignant solid tumors that have a genetic change (mutation) in CKIT or PDGFRA and have spread to nearby tissue or lymph nodes (locally advanced) or other places in the body (metastatic). Avapritinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Avapritinib may help to control the growth of malignant solid tumors.
Written by the trial’s sponsor, quoted from its ClinicalTrials.gov record.
What this trial is looking for
- Any genotype
- Metastatic
- Locally advanced
- Not yet treated
Read automatically from the criteria below, to make the list searchable. It is a summary of what the text mentions, not a decision about whether you qualify — and where the two disagree, the criteria are right and this is wrong.
Who the trial is looking for
Inclusion Criteria
1. The patient (or legally acceptable representative if applicable) provides written informed consent for the study.
2. Male or female ≥18 years of age on the day of informed consent signing. Adolescent patients aged 12 years and older are allowed with signed assent and parental consent according to institutional guidelines and requirements.
3. Cohorts 1 and 2: Patient has a locally advanced or metastatic solid tumor and has progressed on appropriate standard therapy, has not shown clinically meaningful benefit to appropriate standard therapy, has no available standard therapy, or has declined appropriate standard therapy.
- NOTE: Specific solid tumor types include but are not limited to melanoma, breast cancer, lung cancer, gastroesophageal cancer, colorectal cancer, sarcoma, solid tumors NOS, and primary CNS tumors. Patients with any other recurrent solid tumor type with the exception of gastrointestinal stromal tumor (GIST) will be eligible.
4. Cohort 3: Patient has newly diagnosed IDH wild-type, MGMT-unmethylated glioblastoma. Patients must have received prior treatment with radiation and concurrent temozolomide per standard of care.27 Patients must have completed radiation and concurrent temozolomide 3-8 weeks prior to study treatment initiation.
5. Measurable disease per the RECIST v1.1 or RANO criteria, as appropriate, for Cohorts 1 and 2.
This is an extract. Whether you qualify is decided by the trial team against the full criteria, not by reading this page. Read the full eligibility criteria
Where it is running
Running at 1 site. Listed in: United States.
Individual hospitals, and whether each is currently open, are listed on the registry record. Sites open and close throughout a trial.
Worth asking your oncology team
Being listed here is not a recommendation, and no one page can tell you whether a trial is right for you. These are the questions it raises.
- Is this trial open at a hospital I could realistically travel to?
- Given my stage, my previous treatment and my tumour's molecular profile, would I be eligible?
- What would I be giving up by joining — is the comparison arm the treatment I would otherwise be having?
- What is already known about the safety of what is being tested?
The source
Registry record NCT04771520, registered by M.D. Anderson Cancer Center on ClinicalTrials.gov, a public database run by the US National Library of Medicine. The details on this page come from that record and are only ever as current as the sponsor has kept it.
This is a listing of a registered clinical trial, reproduced for information. It is not a recommendation, this site has no connection to the trial or its sponsor, and being listed here says nothing about whether the treatment works. Talk to your own oncology team before pursuing any trial.
This article summarises published research for general information. It is not medical advice, and it is not a substitute for a conversation with your own oncology team, who know your case. Do not start, stop, or change any treatment or supplement on the basis of what you read here.