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Tyrosine Kinase Inhibitor
Avapritinib for Pediatric Solid Cancers
Phase 1 & 2
Recruiting
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 42 months
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
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.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 42 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 42 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Determination of recommended Part 2 dose (Part 1)
Objective Response Rate (Part 2)
Rate and severity of adverse events (Part 1)
Secondary outcome measures
AUC(0-24) (Part 1 and Part 2)
Change from baseline in levels of KIT and PDGFRA mutant allele fractions in peripheral blood (Part 1 and Part 2)
Cmax (Part 1 and Part 2)
+10 moreSide effects data
From 2021 Phase 3 trial • 476 Patients • NCT0346572228%
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%
Ascites
2%
Pyrexia
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
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
6,172 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My platelet count is low and I haven't had a transfusion in the last 14 days.My brain disease treatment with steroids has been stable or reduced for a week.My diagnosis is DMG-H3K27a and standard treatments have not worked or are not available.My cancer has a PDGFRA or KIT mutation and has not responded to standard treatments.I am a male and either vasectomized, will use condoms, practice abstinence, or my partner cannot become pregnant.I can do most activities but need help with some.My liver enzymes are high, but not over five times the normal limit if my cancer affects my liver.My bilirubin levels are higher than normal, possibly due to Gilbert's syndrome.I have a serious heart condition that is not under control.I am willing and able to follow the study's schedule and procedures.I have been treated for a blood clot in the last 6 months.My hemoglobin level is below 8.0 g/dL and I haven't had a blood transfusion in the last week.I have received cancer treatment within specific time limits.My kidney function test shows creatinine levels higher than normal.I have been treated with avapritinib before.I am a woman able to have children and will not use effective birth control.I have not taken strong CYP3A inhibitors or inducers in the last 28 days.I have had another type of cancer before.I have a significant ongoing or past medical condition.I need blood thinners, but there might be exceptions.I am at risk for bleeding in my brain.I've had a stem cell transplant or CAR T cell therapy recently.I have not had major surgery within the last 14 days.I cannot swallow pills or the tiny tablets in sprinkle capsules.My seizure disorder is not well controlled despite medication.I am between 2 and 17 years old.I am using effective birth control or my partner is sterile.
Research Study Groups:
This trial has the following groups:- Group 1: avapritinib
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many people are currently involved in this experiment?
"37 patients that fit the inclusion criteria must enroll in this study. Multiple centres are available for patient participation, such as Dana Farber Cancer Institute located in Boston, Massachusetts and Columbia University Medical Center situated in New york, New York."
Answered by AI
How many different research facilities are conducting this experiment?
"There are 26 participating centres in total, with 13 of them actively recruiting patients at the moment. These locations include the Dana Farber Cancer Institute in Boston, Columbia University Medical Center in New york, and Children's Hospital Colorado in Aurora."
Answered by AI
Is this research the first to be conducted on this topic?
"Avapritinib clinical trials are ongoing in 37 cities and 18 countries. The first study began in 2018, involving 103 individuals and sponsored by Blueprint Medicines Corporation. To date, 3 avapritinib trials have completed Phase 2 drug approval stages."
Answered by AI
Are there any other drugs that have been trialed alongside avapritinib in the past?
"avapritinib was first researched in 2018 at Universitätsmedizin Mannheim III. Medizinische Klinik. So far, there have been three completed clinical trials with seven more currently underway across the United States of America, many centred in Boston, Massachusetts."
Answered by AI
Does this experimental treatment program accept patients who are over 20 years old?
"Children aged 2 to 17 can enroll in this particular trial, according to the inclusion criteria. Out of 3201 total trials, 321 are for patients under 18 and 2280 are for senior citizens."
Answered by AI
What are the prerequisites for joining this clinical trial?
"The prerequisites for this particular clinical trial are that the patient has relapsed solid neoplasm and is between 2 to 17 years old. There is a total of 37 slots available for participants."
Answered by AI
Can new patients join this clinical trial?
"From what is published on clinicaltrials.gov, it appears that the trial is searching for participants. The listing shows that the 37 patients are needed at 13 different sites and the original posting was on February 24th, 2022 with the most recent update being November 11th, 2022."
Answered by AI
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