Solid Tumor, Unspecified, ChildPhase 1/2NCT04773782
A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
A Phase 1/2 study testing avapritinib in people with Solid Tumor, Unspecified, Child and Relapsed Solid Neoplasm, run by Blueprint Medicines Corporation at 26 sites in 9 countries.
Trial summary
This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to < 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA and/or KIT mutation (including non-synonymous point mutations, insertions, and deletions) or… Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Solid Tumor, Unspecified, Child, Relapsed Solid Neoplasm, CNS Tumor
- What is being tested?
- avapritinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 26 sitesin 9 countries
- Study dates
- 24 Feb 2022to 21 Nov 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participant must be 2 to < 18 years of age at the time of signing the informed consent.
- Diagnosis
- Participant has confirmed diagnosis of R/R solid tumor, including CNS tumors, with a mutation (including non-synonymous point mutations, insertions, and deletions) in PDGFRA and/or KIT (confirmed by local mutational testing of tumor sample) that has progressed despite standard therapy and no alternative treatment option is available. Participant with R/R solid tumors with only PDGFRA and/or KIT amplifications may be included with approval from the Sponsor.
- OR
- Participant has confirmed diagnosis of DMG-H3K27a (confirmed by local testing of tumor sample) that has failed standard therapy or for which no standard therapy that may convey clinical benefit exists, as judged by the investigator.
- Participants with CNS disease should be on a stable (≤ 10% change) or decreasing dose of corticosteroids for at least 7 days prior to first dose of avapritinib, with no plans for dose escalation.
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- Disease extent: a. Part 1: All participants must have at least 1 measurable lesion as defined by RECIST v1.1 or Response Assessment in Neuro-Oncology (RANO) (for CNS tumors). If radiation therapy has been administered, at least 1 measurable lesion must not have been irradiated, or must have clearly progressed since being irradiated as per RANO and must be ≥ 12 weeks from radiation to any target lesion.
- Part 2: All participants must have at least 1 measurable lesion as defined by RECIST v1.1 or RANO (for CNS tumors). For Participants with DMG-H3K27a or PDGFRA and/or KIT mutant/amplified solid tumors, including CNS tumors that have progressed despite prior therapy, who have received radiation therapy, at least 1 measurable lesion must not have been irradiated, or must have clearly progressed since being irradiated as per RANO and must be ≥ 12 weeks from radiation to any target lesion. For up to 5 Participants with newly diagnosed DMG-H3K27a where there is no standard therapy that may convey clinical benefit exists as judged by the investigator, progression of disease of a measurable lesion after irradiation is not required.
- A Lansky (< 16 years of age) or Karnofsky (≥ 16 years of age) score of at least 50. If the Participant is unable to walk due to paralysis, but is mobile in a wheelchair, the participant is considered ambulatory for the purpose of assessing their performance status.
- Participant agrees to utilize contraception consistent with local regulations.
- Male participants: Are vasectomized, or agree to use condoms, as defined in Section 5.4.2, from the start of Screening until 6 weeks after the last dose of study treatment, or practice true abstinence (when this is in line with the preferred and usual lifestyle of the Participant, see Section 5.4.2), or have a female partner who is NOT of childbearing potential.
- Female participants: Agree to use effective contraception, as defined in Section 5.4.2, from the start of Screening until 6 weeks after the last dose of study treatment and have a male partner who uses a condom, or practice true abstinence (when this is in line with the preferred and usual lifestyle of the Participant), or have a male partner who is vasectomized with confirmed azoospermia.
- Participant can give written informed consent/assent before any study-specific Screening procedures (if feasible). Parental/legal guardian consent will be determined by local, regional, and/or national guidelines.
Who cannot join
- Participant has any of the following within 14 days before the first dose of study treatment:
- 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.
- Absolute neutrophil count (ANC) < 1.0 × 10^9/L.
- Hemoglobin < 8.0 g/dL with no RBC transfusion ≤ 7 days prior to the measurement.
- AST or ALT > 3 × the ULN for age; except in Participants with tumor involvement of the liver who must not have AST and ALT > 5 × ULN for age.
- Total bilirubin > 1.5 × ULN for age; and in presence of Gilbert's syndrome, total bilirubin > 3 × ULN or direct bilirubin > 1.5 × ULN.
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- Serum creatinine > 1.5 × ULN for age.
- International normalized ratio or prothrombin time (PT) > ULN (> 1.5 × ULN if on prophylactic reversible anticoagulants).
- Participant has a QTcF > 470 msec. Participant has a familial or personal history of prolonged QT syndrome or Torsades de pointes.
- Participant has clinically significant, uncontrolled cardiovascular disease including congestive heart failure Grade III or IV according to the New York Heart Association classification; myocardial infarction or unstable angina within the previous 6 months, uncontrolled hypertension (> 95th percentile for age), or clinically significant, uncontrolled arrhythmias, including bradyarrhythmias that may cause QT prolongation (eg, Type II second-degree heart block or third-degree heart block).
- Participant received the following systemic antineoplastic therapies:
- Temozolomide within 4 weeks prior to the first dose of study drug
- Nitrosurea within 6 weeks prior to the first dose of study drug
- Any other systemic antineoplastic therapy (including experimental therapy) within 5 half-lives or 28 days prior to the first dose of study drug, whichever is shorter.
- Focal external beam radiotherapy, including stereotactic radiosurgery, within 6 weeks prior to the first dose of avapritinib to either target or nontarget lesions. Systemic radiopharmaceuticals, including nonstereotactic radiosurgery, within 2 weeks of the first dose of avapritinib (within 6 weeks for Participants with CNS tumors). Craniospinal irradiation within 6 weeks prior to the first dose of avapritinib.
- All AEs related to other antineoplastic therapies (eg, systemic antineoplastics, radiotherapy) must have resolved to Grade ≤ 1 (Grade ≤ 2 for peripheral neuropathy and/or ototoxicity) prior to the first dose of avapritinib.
- Participant has previously received treatment with avapritinib.
- Participant received autologous stem cell transplant following myeloablative therapy or chimeric antigen receptor T cell therapy within 3 months prior to the first dose of avapritinib or prior allogeneic stem cell transplant within 1 year and no evidence of Grade 1 or greater graft-versus-host disease and no immunosuppressants for graft-versus-host disease (steroids for primary malignancy being permitted). Participants who received stem cell reinfusion following nonmyeloablative therapy are eligible once they meet the peripheral blood count criteria in Exclusion Criterion #1.
- Participant requires ongoing treatment or has received treatment within 28 days before the start of avapritinib administration with drugs or foods that are strong CYP3A inhibitors or inducers.
- Participant has had a major surgical procedure within 14 days of the first dose of study treatment (procedures such as central venous catheter placement, tumor needle biopsy, and feeding tube placement are not considered major surgical procedures).
- Participant has a history of another primary malignancy that has been diagnosed or required therapy within 3 years before the first dose of avapritinib. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.
- Female subjects of childbearing potential who are unwilling, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of informed consent and for at least 6 weeks after the last dose of study treatment. Male subjects who are unwilling, if not surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of informed consent and for at least 6 weeks after the last dose of study treatment.
- Participant is pregnant, as documented by a serum β-hCG pregnancy test consistent with pregnancy obtained at Screening and within 72 hours before the first dose of study treatment. Participants with β-hCG values that are within the range for pregnancy but are not pregnant (false-positives) may be enrolled with written consent of the Sponsor after pregnancy has been ruled out. Female subjects of nonchildbearing potential (premenarchal, bilateral tubal ligation, bilateral oophorectomy, or hysterectomy) do not require a serum β-hCG test.
- Participant is breastfeeding.
- Participant has prior or ongoing clinically significant illness, medical condition, surgical history, physical finding, or laboratory abnormality that, in the Investigator's opinion, could affect the safety of the Participant; alter the absorption, distribution, metabolism, or excretion of the study drug; or impair the assessment of study results.
- History of thrombosis requiring treatment within the past 6 months. This exclusion does not apply to catheter-related thrombosis if the catheter has been removed and did not require any other treatment in the previous 3 months.
- Participants who require anticoagulants, with the exception of stable doses of prophylactic reversible anticoagulants.
- Participants who are unable to swallow tablets (in Part 1) or minitablets (in Part 2) within the sprinkle capsules.
- Participants with a known risk of intracranial bleeding, such as a brain aneurysm that has not been removed or repaired, or a history of intracranial bleeding within the past year, or radiographic evidence of hemorrhage on Screening MRI. Exceptions are: Participants with primary CNS tumors (provided they have not had CNS bleeding within 2 weeks of the first dose of avapritinib) or Participants with punctate hemorrhages < 3 mm.
- History of a seizure disorder that is not well controlled on current antiepileptic medications.
- Participant is unwilling or unable to comply with scheduled visits, treatment administration plan, laboratory tests, or other study procedures and study restrictions.
Trial locations and contacts
26 sites in 9 countries.
26 sites in 9 countries
United States15 sites
University of Michigan
Ann Arbor, Michigan, 48109
Children's Hospital Colorado
Aurora, Colorado, 80045
The Johns Hopkins Hospital
Baltimore, Maryland, 21287
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Medical Center
Dallas, Texas, 75235
Duke University Medical Center
Durham, North Carolina, 27710
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Columbia University Medical Center
New York, New York, 10032
Phoenix Children's Hospital
Phoenix, Arizona, 85016
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
University of California San Francisco, Benioff Children's Hospital
San Francisco, California, 94518
Washington University School of Medicine
St Louis, Missouri, 63110
Australia2 sites
Royal Children's Hospital
Parkville, Victoria, 3052
Sydney Children's Hospital, Kids Cancer Center
Sydney, New South Wales, 2031
Germany2 sites
Universitaetsmedizin Göttingen
Göttingen, Lower Saxony, 37075
Hopp Children's Cancer Center
Heidelberg
South Korea2 sites
Asan Medical Center
Seoul, 05505
Samsung Medial Center
Seoul, 06351
Italy1 site
Dipartimento di Oncologia Medica ed Ematologia - S.C. Pediatria Oncologica
Milan, 20133
United Kingdom1 site
Great Ormond Street Hospital For Children
London, WC1N 3JH
About this study
Official title: A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to < 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA and/or KIT mutation (including non-synonymous point mutations, insertions, and deletions) or amplification, or DMG-H3K27a who have no available curative treatment options. This is a single-arm trial in which all participants will receive avapritinib. The study consists of 2 parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the Part 2 recommended dose (Part 2).
- Being tested
- avapritinib
- Keywords
- KIT, PDGFRA, Relapsed/Refractory Solid Tumor, Glioma, H3K27M, DMG-H3K27a
- Medical subject headings
- Central Nervous System Neoplasms, Recurrence, Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
avapritinibDrug
Also called BLU-285
oral administration
| Study group | Role | What they receive |
|---|---|---|
| avapritinib | Experimental | Avapritinib tablets for oral administration. Avapritinib will be dosed daily for 28 day cycles. Receives: Drug: avapritinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of recommended Part 2 dose (Part 1)
Measured: up to 8 months
Objective Response Rate (Part 2)
Measured: up to 36 months
Rate and severity of adverse events (Part 1)
Measured: up to 8 months
Secondary outcomes (13)
Objective Response Rate (Part 1 and Part 2)
Measured: up to 42 months
Rate and severity of adverse events (Part 1 and Part 2)
Measured: up to 42 months
Palatability assessments as measured by the 5-point Hedonic scale (Part 1 and Part 2)
Measured: up to 42 months
Duration of Response (Part 1 and Part 2)
Measured: up to 42 months
Progression-free survival (Part 1 and Part 2)
Measured: up to 42 months
Disease control rate (Part 1 and Part 2)
Measured: up to 42 months
Time to response (Part 1 and Part 2)
Measured: up to 42 months
Cmax (Part 1 and Part 2)
Measured: up to 36 months
AUC(0-24) (Part 1 and Part 2)
Measured: up to 36 months
Tmax (Part 1)
Measured: up to 36 months
T 1/2 (Part 1)
Measured: up to 36 months
Ctrough (Part 2)
Measured: up to 36 months
Change from baseline in levels of KIT and PDGFRA mutant allele fractions in peripheral blood (Part 1 and Part 2)
Measured: up to 42 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2021
- Study start24 Feb 2022
- Primary completion21 Nov 2025
- Study completion21 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Solid Tumor, Unspecified, ChildStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04773782
- Sponsor's protocol ID
- BLU-285-3101
- Status last verified
- Jan 2026
- First submitted
- 9 Feb 2021
- Last updated
- 13 Jan 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
- ClinicalTrials.gov ID
- NCT04773782
- Acronym / sponsor ID
- BLU-285-3101
- Keywords
- KIT, PDGFRA, Relapsed/Refractory Solid Tumor, Glioma, H3K27M, DMG-H3K27a
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Study start
- 24 Feb 2022 (actual)
- Primary completion
- 21 Nov 2025 (actual)
- Study completion
- 21 Nov 2025 (actual)
- First posted
- 26 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to < 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA and/or KIT mutation (including non-synonymous point mutations, insertions, and deletions) or amplification, or DMG-H3K27a who have no available curative treatment options. This is a single-arm trial in which all participants will receive avapritinib. The study consists of 2 parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the Part 2 recommended dose (Part 2).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| avapritinib | Experimental | Drug: avapritinibAvapritinib tablets for oral administration. Avapritinib will be dosed daily for 28 day cycles. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (13 listed)
- Participant must be 2 to < 18 years of age at the time of signing the informed consent.
- Diagnosis
- OR
- Participants with CNS disease should be on a stable (≤ 10% change) or decreasing dose of corticosteroids for at least 7 days prior to first dose of avapritinib, with no plans for dose escalation.
- Disease extent: a. Part 1: All participants must have at least 1 measurable lesion as defined by RECIST v1.1 or Response Assessment in Neuro-Oncology (RANO) (for CNS tumors). If radiation therapy has been administered, at least 1 measurable lesion must not have been irradiated, or must have clearly progressed since being irradiated as per RANO and must be ≥ 12 weeks from radiation to any target lesion.
Key exclusion criteria (31 listed)
- Participant has any of the following within 14 days before the first dose of study treatment:
- Participant has a QTcF > 470 msec. Participant has a familial or personal history of prolonged QT syndrome or Torsades de pointes.
- Participant has clinically significant, uncontrolled cardiovascular disease including congestive heart failure Grade III or IV according to the New York Heart Association classification; myocardial infarction or unstable angina within the previous 6 months, uncontrolled hypertension (> 95th percentile for age), or clinically significant, uncontrolled arrhythmias, including bradyarrhythmias that may cause QT prolongation (eg, Type II second-degree heart block or third-degree heart block).
- Participant received the following systemic antineoplastic therapies:
- Participant has previously received treatment with avapritinib.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| avapritinibAlso: BLU-285oral administration | Drug | avapritinib |
Primary outcome
Determination of recommended Part 2 dose (Part 1)
Time frame: up to 8 months
Objective Response Rate (Part 2)
Time frame: up to 36 months
Rate and severity of adverse events (Part 1)
Time frame: up to 8 months
13 secondary outcomes
Objective Response Rate (Part 1 and Part 2)
Time frame: up to 42 months
Rate and severity of adverse events (Part 1 and Part 2)
Time frame: up to 42 months
Palatability assessments as measured by the 5-point Hedonic scale (Part 1 and Part 2)
Time frame: up to 42 months
Duration of Response (Part 1 and Part 2)
Time frame: up to 42 months
Progression-free survival (Part 1 and Part 2)
Time frame: up to 42 months
Disease control rate (Part 1 and Part 2)
Time frame: up to 42 months
Time to response (Part 1 and Part 2)
Time frame: up to 42 months
Cmax (Part 1 and Part 2)
Time frame: up to 36 months
AUC(0-24) (Part 1 and Part 2)
Time frame: up to 36 months
Tmax (Part 1)
Time frame: up to 36 months
T 1/2 (Part 1)
Time frame: up to 36 months
Ctrough (Part 2)
Time frame: up to 36 months
Change from baseline in levels of KIT and PDGFRA mutant allele fractions in peripheral blood (Part 1 and Part 2)
Time frame: up to 42 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
26 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing avapritinib in people with Solid Tumor, Unspecified, Child and Relapsed Solid Neoplasm, run by Blueprint Medicines Corporation at 26 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Australia, Germany, South Korea, Austria and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determination of recommended Part 2 dose (Part 1) (time frame: up to 8 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.