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Tyrosine Kinase Inhibitor

Avapritinib for Systemic Mastocytosis

Phase 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
Patient must have a diagnosis of aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) or mast cell leukemia (MCL) based on World Health Organization diagnostic criteria. Before enrollment, the Study Steering Committee must confirm the diagnosis of AdvSM (based on Central Pathology Laboratory assessment of bone marrow).
Patient must have Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 3.
Must not have
Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 or creatinine > 1.5 × ULN.
Patient has received any cytoreductive therapy (including midostaurin and other TKIs, hydroxyurea, azacitidine) or an investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon and any antibody therapy (eg, brentuximab vedotin) less than 28 days before obtaining screening BM biopsy for this study.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up approximately 4 years after the first subjected enrolled
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug, avapritinib, for patients with advanced mastocytosis, which is a rare disease where mast cells accumulate in the body.

Who is the study for?
This trial is for adults with advanced systemic mastocytosis, including aggressive forms and mast cell leukemia. Participants must have a serum tryptase level of at least 20 ng/mL and an ECOG performance status of 0-3. They should not have had recent cytoreductive therapy or certain other treatments, nor should they have severe kidney issues, seizure disorders, brain cancer, low platelet counts, a history of intracranial bleeding, abnormal heart rhythm or liver enzymes due to causes other than mastocytosis.
What is being tested?
The PATHFINDER study is testing the effectiveness and safety of Avapritinib (BLU-285), which targets specific mutations in tyrosine kinase involved in systemic mastocytosis. It's an open-label Phase 2 trial where all participants receive the drug without being compared to another treatment or placebo.
What are the potential side effects?
Potential side effects from Avapritinib may include nausea, vomiting, swelling around the eyes or on the skin (periorbital edema), fatigue, muscle pain (myalgia), cognitive changes like memory problems or confusion (cognitive effects), hair color changes and fluid retention.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with a severe form of mastocytosis confirmed by a specialized committee.
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I can care for myself but may not be able to do active work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My kidney function is significantly reduced.
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I haven't taken any cancer-reducing drugs or experimental treatments within the last 14 days, or specific drugs like cladribine or antibody therapies within the last 28 days.
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I have epilepsy or need medication to prevent seizures.
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My platelet count is below 50,000 or I need platelet transfusions.
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I have a brain tumor or cancer that has spread to my brain.
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I have been treated with avapritinib before.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~approximately 4 years after the first subjected enrolled
This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 4 years after the first subjected enrolled for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Area Under Curve (0 to Tau) for Avapritinib
Change in EORTC QLQ-C30
Change in PGIS
+12 more

Side effects data

From 2021 Phase 1 trial • 250 Patients • NCT02508532
100%
Decreased appetite
67%
Weight decreased
67%
Blood bilirubin increased
67%
Cough
67%
Dizziness
67%
Dysgeusia
33%
Memory impairment
33%
Face oedema
33%
Peripheral sensory neuropathy
33%
Anemia
33%
Hypoxia
33%
Constipation
33%
Mood altered
33%
Periorbital oedema
33%
Influenza like illness
33%
Insomnia
33%
Rash
33%
Dry skin
33%
Hypertension
33%
Neutropenia
33%
Lacrimation increased
33%
Dyspepsia
33%
Dry mouth
33%
Hypokalaemia
33%
Back pain
33%
Myalgia
33%
Hair colour changes
33%
Alopecia
33%
Dermatitis acneiform
33%
Contusion
33%
Leukopenia
33%
Hypophosphataemia
33%
Anxiety
100%
80%
60%
40%
20%
0%
Study treatment Arm
Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD
Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 or 400 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD

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 will be administered as an immediate release tablet, orally, continuously, in 28-day cycles
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Avapritinib
2022
Completed Phase 1
~400

Find a Location

Who is running the clinical trial?

Blueprint Medicines CorporationLead Sponsor
29 Previous Clinical Trials
6,105 Total Patients Enrolled

Media Library

Avapritinib (Tyrosine Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03580655 — Phase 2
Systemic Mastocytosis Research Study Groups: Avapritinib
Systemic Mastocytosis Clinical Trial 2023: Avapritinib Highlights & Side Effects. Trial Name: NCT03580655 — Phase 2
Avapritinib (Tyrosine Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03580655 — Phase 2
~17 spots leftby Jan 2026
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