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Tyrosine Kinase Inhibitor

Avapritinib for Systemic Mastocytosis

Phase 1
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
Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) and at least 1 C-finding attributable to systemic mastocytosis (SM). The AHN must be myeloid, with the following exceptions that are excluded: Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS) that is very high- or high-risk as defined by the International prognostic scoring system for myelodysplastic syndromes (IPSS-R) and Philadelphia chromosome positive malignancies.
Mast cell leukemia (MCL).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 8, 24, 40, 68 and every 24 weeks until patient terminates from the study (approximately 24 months)
Awards & highlights

Study Summary

This trial is testing a new drug, avapritinib, for safety, how well it works, and how it is metabolized in patients with advanced mastocytosis or other relapsed or refractory myeloid malignancies. The study has two parts, dose escalation and expansion.

Eligible Conditions
  • Systemic Mastocytosis
  • Hematologic Non-Mast Cell Disease
  • Mast Cell Cancer
  • Myeloid Malignancies

Eligibility Criteria

Inclusion Criteria

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

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~8, 24, 40, 68 and every 24 weeks until patient terminates from the study (approximately 24 months)
This trial's timeline: 3 weeks for screening, Varies for treatment, and 8, 24, 40, 68 and every 24 weeks until patient terminates from the study (approximately 24 months) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Maximum tolerated dose (MTD) of avapritinib (also known as BLU-285)
Number of patients with adverse and serious adverse events and changes in physical findings, vital signs, clinical laboratory results and ECG findings
Recommended Phase 2 dose (RP2D) of avapritinib
Secondary outcome measures
Change in liver volume by imaging
Change in spleen volume by imaging
Changes in levels of serum tryptase and levels of V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog (KIT) D816V allele burden in blood
+7 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%
Memory impairment
33%
Hypoxia
33%
Peripheral sensory neuropathy
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%
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

Trial Design

1Treatment groups
Experimental Treatment
Group I: Avapritinib (also known as BLU-285)Experimental 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
2015
Completed Phase 1
~340

Find a Location

Who is running the clinical trial?

Blueprint Medicines CorporationLead Sponsor
28 Previous Clinical Trials
6,042 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~10 spots leftby Mar 2025