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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

For patients receiving corticosteroids, the dose must be ≤ 20 mg/d prednisone or equivalent, and the dose must be stable for ≥ 14 days.

Patient must have SM, confirmed by Central Pathology Review of BM biopsy, and central review of B- and C-findings by WHO diagnostic criteria.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial is testing a new drug, avapritinib, for people with a rare blood cancer called indolent systemic mastocytosis. The study will compare how well the new drug works compared to a placebo, and will also look at the side effects of the new drug.

Who is the study for?

This trial is for patients with indolent systemic mastocytosis (ISM) who haven't found relief from their symptoms through standard care. They must have a specific symptom score, confirmed ISM by pathology review, and be able to perform daily activities with minimal assistance. People can't join if they've had certain treatments recently, other SM subtypes, prior avapritinib use, or specific heart rhythm issues.Check my eligibility

What is being tested?

The study tests Avapritinib's effectiveness and safety against a placebo in ISM patients whose symptoms persist despite best supportive care. It's randomized and double-blind: neither doctors nor participants know who gets the real drug or placebo until the study ends. Everyone eventually receives Avapritinib during part three of the trial.See study design

What are the potential side effects?

While not specified here, side effects of Avapritinib may include nausea, vomiting, swelling around eyes or mouth; skin rash; shortness of breath; feeling dizzy or faint; rapid heartbeat; weight gain due to fluid retention.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am on a stable dose of corticosteroids, not exceeding 20 mg/d prednisone or its equivalent, for at least 14 days.

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My systemic mastocytosis was confirmed by a specialized lab review.

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My symptoms are moderate to severe based on a recent symptom score.

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I am able to care for myself and perform daily activities.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

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

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Part 1: Recommended Phase 2 dose (RP2D) in patients with ISM

Part 2: Mean change in ISM Symptom Assessment Form (ISM-SAF) total symptom score (TSS) as compared to placebo

Part 3: Number of Participants with Adverse Events

Secondary outcome measures

Part 2: Proportion of patients with a ≥50% reduction in bone marrow mast cells or no aggregates for patients with aggregates at Baseline

Part 2: Proportion of patients with a ≥50% reduction in peripheral blood V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) allele fraction or undetectable for patients with detectable mutation at Baseline

Part 2: Proportion of patients with a ≥50% reduction in serum tryptase

+13 more

Side effects data

From 2021 Phase 1 trial • 250 Patients • NCT02508532

100%

Decreased appetite

67%

Cough

67%

Weight decreased

67%

Dizziness

67%

Blood bilirubin increased

67%

Dysgeusia

33%

Peripheral sensory neuropathy

33%

Hypoxia

33%

Memory impairment

33%

Constipation

33%

Mood altered

33%

Periorbital oedema

33%

Influenza like illness

33%

Insomnia

33%

Anemia

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%

Face oedema

33%

Dermatitis acneiform

33%

Contusion

33%

Leukopenia

33%

Hypophosphataemia

33%

Anxiety

100%

80%

60%

40%

20%

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

Trial Design

7Treatment groups

Experimental Treatment

Placebo Group

Group I: (Part 3) Avapritinib RP2D + BSCExperimental Treatment1 Intervention