← Back to Search

Tyrosine Kinase Inhibitor

Avapritinib for Pediatric Solid Cancers

Phase 1 & 2
Waitlist Available
Research Sponsored by Blueprint Medicines Corporation
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant has confirmed diagnosis of DMG-H3K27a that has failed standard therapy or for which no standard therapy that may convey clinical benefit exists.
Participant has confirmed diagnosis of R/R solid tumor, including CNS tumors, with a mutation in PDGFRA and/or KIT that has progressed despite standard therapy and no alternative treatment option is available.
Must not have
Participant has platelet count < 75 × 10^9/L (< 100 × 10^9/L if a CNS tumor) with no platelet transfusion within 14 days prior to the measurement.
Participant has Total bilirubin > 1.5 × ULN for age; and in presence of Gilbert's syndrome, total bilirubin > 3 × ULN or direct bilirubin > 1.5 × ULN.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug, avapritinib, for kids with cancer who have run out of treatment options. The trial will first test safety and how the body handles the drug (Part 1), then move on to testing how well the drug works against the cancer (Part 2).

Who is the study for?
This trial is for children and teenagers aged 2 to less than 18 with advanced solid tumors, including brain tumors that have specific genetic changes (mutations in KIT or PDGFRA) or a type of glioma called DMG-H3K27a. They should have tried standard treatments without success and not be eligible for other curative options. Participants need to be stable on certain medications, able to perform daily activities at least minimally, and agree to use contraception.
What is being tested?
The study tests avapritinib, a medication targeting cancers with certain genetic mutations. It's an open-label trial where all participants receive the drug. The first part confirms the dose and assesses safety; the second part looks at how effective it is at a recommended dose while continuing safety assessments.
What are the potential side effects?
While specific side effects are not listed here, common ones associated with cancer drugs like avapritinib may include nausea, vomiting, diarrhea, fatigue, muscle pain, liver problems (elevated enzymes), blood count changes leading to increased infection risk or bleeding tendencies.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My diagnosis is DMG-H3K27a and standard treatments have not worked or are not available.
Select...
My cancer has a PDGFRA or KIT mutation and has not responded to standard treatments.
Select...
I can do most activities but need help with some.
Select...
I am between 2 and 17 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My platelet count is low and I haven't had a transfusion in the last 14 days.
Select...
My bilirubin levels are higher than normal, possibly due to Gilbert's syndrome.
Select...
I have a serious heart condition that is not under control.
Select...
I am willing and able to follow the study's schedule and procedures.
Select...
My kidney function test shows creatinine levels higher than normal.
Select...
I have been treated with avapritinib before.
Select...
I am a woman able to have children and will not use effective birth control.
Select...
I have not taken strong CYP3A inhibitors or inducers in the last 28 days.
Select...
I am at risk for bleeding in my brain.
Select...
I have not had major surgery within the last 14 days.
Select...
I cannot swallow pills or the tiny tablets in sprinkle capsules.
Select...
My seizure disorder is not well controlled despite medication.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2021 Phase 3 trial • 476 Patients • NCT03465722
28%
Periorbital oedema
23%
Oedema peripheral
19%
Lacrimation increased
18%
White blood cell count decreased
15%
Eyelid oedema
14%
Hair colour changes
14%
Aspartate aminotransferase increased
13%
Dizziness
13%
Memory impairment
12%
Headache
11%
Anaemia
10%
Alopecia
10%
Weight decreased
9%
Dyspepsia
9%
Blood creatinine increased
8%
Weight increased
8%
Dysgeusia
8%
Platelet count decreased
7%
Gastrooesophageal reflux disease
7%
Cough
6%
Hypocalcaemia
6%
Alanine aminotransferase increased
5%
Abdominal pain
5%
Hypomagnesaemia
5%
Conjunctivitis
5%
Pruritus
5%
Anxiety
5%
Hypoalbuminaemia
4%
Arthralgia
4%
Dry skin
3%
Dry mouth
3%
Stomatitis
3%
Muscle spasms
3%
Myalgia
3%
Dysphonia
3%
Rash maculo-papular
3%
Blood alkaline phosphatase increased
3%
Gastrointestinal haemorrhage
2%
Pyrexia
2%
Ascites
2%
Sepsis
2%
Acute kidney injury
2%
Palmar-plantar erythrodysaesthesia syndrome
2%
Vomiting
2%
Diarrhoea
2%
Gastric haemorrhage
1%
Multi-organ failure
1%
Fatigue
1%
Hypoglycaemia
1%
Disease progression
1%
Face oedema
1%
General physical health deterioration
1%
Bacteraemia
1%
Pneumonia
1%
Tumour haemorrhage
1%
Cognitive disorder
1%
Haemorrhage intracranial
1%
Dyspnoea
1%
Pleural effusion
1%
Mucosal inflammation
1%
Gastrointestinal fistula
1%
Intestinal obstruction
1%
Intestinal perforation
1%
Abdominal pain upper
1%
Lower gastrointestinal haemorrhage
1%
Nausea
1%
Rectal haemorrhage
1%
Jaundice
1%
Portal hypertension
1%
Hypokalaemia
1%
Transient ischaemic attack
1%
Pulmonary oedema
1%
Generalised oedema
1%
Hepatic haemorrhage
1%
Hypophosphataemia
1%
Intra-abdominal haemorrhage
1%
Abdominal distension
1%
Cardiac failure congestive
1%
Back pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Avapritinib
Regorafinib

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: avapritinibExperimental Treatment1 Intervention
Avapritinib tablets for oral administration. Avapritinib will be dosed daily for 28 day cycles.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
avapritinib
2018
Completed Phase 3
~480

Find a Location

Who is running the clinical trial?

Blueprint Medicines CorporationLead Sponsor
29 Previous Clinical Trials
6,168 Total Patients Enrolled

Media Library

Avapritinib (Tyrosine Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04773782 — Phase 1 & 2
Brain Tumor Research Study Groups: avapritinib
Brain Tumor Clinical Trial 2023: Avapritinib Highlights & Side Effects. Trial Name: NCT04773782 — Phase 1 & 2
Avapritinib (Tyrosine Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04773782 — Phase 1 & 2
~2 spots leftby Feb 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top