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Kinase Inhibitor

BLU-263 for Systemic Mastocytosis

Phase 2 & 3

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

Patients must have symptoms consistent with mast cell activation (despite BSC) in at least two organ systems characterized by cutaneous flushing, tachycardia, syncope, hypotension, diarrhea, nausea, vomiting and gastro-intestinal cramping) and serum blood tryptase (sBT) levels above 8 ng/mL OR Severe (Ring and Messmer grading ≥ II, recurrent anaphylaxis, including but not limited to hymenoptera venom, drug or food, regardless of sBT levels.

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial is testing a new drug, BLU-263, to see if it can help people with a mastocytosis, a disease where too many mast cells build up in the body. The trial will compare the new drug to a placebo to see if it is effective and safe.

Who is the study for?

This trial is for patients with indolent systemic mastocytosis (ISM) who still have symptoms despite best supportive care. They should have a blood tryptase level above 8 ng/mL or severe reactions to allergens, an ECOG Performance Status of 0-2, and not controlled symptoms after trying at least two therapies like antihistamines or corticosteroids. People can't join if they've had certain heart issues, other myeloproliferative disorders, recent cancer treatments outside of specific exceptions, or previous treatment with targeted KIT inhibitors.Check my eligibility

What is being tested?

The HARBOR study is testing the effectiveness and safety of BLU-263 compared to a placebo in managing ISM symptoms. Participants will receive either BLU-263 plus best supportive care or a placebo plus best supportive care in a randomized and double-blind setup. After completing initial phases, all participants may receive BLU-263 openly.See study design

What are the potential side effects?

While the side effects of BLU-263 are not detailed here, typical drug-related side effects could include digestive issues, skin reactions, headaches, fatigue and potential allergic responses. The exact profile will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

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

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I experience symptoms like flushing, rapid heartbeat, fainting, low blood pressure, diarrhea, nausea, vomiting, or stomach cramps in at least two parts of my body and have high tryptase levels or severe reactions to things like insect stings, medications, or foods.

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

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My tests show I have KIT D816V or CD25+ Mast cells.

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I can take care of myself and am up and about more than half of my waking hours.

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

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 Dose (RD) in patients with ISM

Part 2: Proportion of responders, defined as ≥30% reduction in ISM-Symptom in Assessment Form (ISM-SAF) Total Symptom Score (TSS)

Part 3: Long-term safety and tolerability of BLU-263 as assessed by the number of adverse events and serious adverse events

+1 more

Secondary outcome measures

Part 1: Mean change in Indolent Systemic Mastocytosis-Symptom Assessment Form (ISM-SAF) Total Symptom Score (TSS)

Part 1: Mean change in Indolent Systemic Mastocytosis-Symptom Assessment Form (ISM-SAF) individual symptom scores

Part 1: Mean change in measures of mast cell burden

+18 more

Other outcome measures

Mast cell

Trial Design

9Treatment groups

Experimental Treatment

Placebo Group

Group I: PK Groups (Dose 2 or Dose 3)Experimental Treatment1 Intervention

Patients will receive best supportive care (BSC) and Dose 2 or Dose 3 of BLU-263 tablets. BSC will be determined on a per patient basis. BLU-263 will be administered orally for the duration of participation in the study.

Group II: (Part M) BLU-263 RD + BSCExperimental Treatment1 Intervention

Patients will receive best supportive care (BSC) and the recommended dose (RD) of BLU-263 tablets. BSC will be determined on a per patient basis. BLU-263 will be administered orally, once daily for the duration of participation in the study.

Group III: (Part 3) BLU-263 RD + BSCExperimental Treatment1 Intervention

Patients will receive best supportive care (BSC) and the recommended dose (RD) of BLU-263 tablet in an open-label fashion for up to 5 years.

Group IV: (Part 2) BLU-263 RD + BSCExperimental Treatment1 Intervention

Group V: (Part 1) BLU-263 Dose 3 + BSCExperimental Treatment1 Intervention

Patients will receive best supportive care (BSC) and Dose 3 of BLU-263 tablets. BSC will be determined on a per patient basis. BLU-263 will be administered orally, once daily until completion of Part 1.

Group VI: (Part 1) BLU-263 Dose 2 + BSCExperimental Treatment1 Intervention

Patients will receive best supportive care (BSC) and Dose 2 of BLU-263 tablets. BSC will be determined on a per patient basis. BLU-263 will be administered orally, once daily until completion of Part 1.

Group VII: (Part 1) BLU-263 Dose 1 + BSCExperimental Treatment1 Intervention

Patients will receive best supportive care (BSC) and Dose 1 of BLU-263 tablets. BSC will be determined on a per patient basis. BLU-263 will be administered orally, once daily until completion of Part 1.

Group VIII: (Part 2) Placebo + BSCPlacebo Group1 Intervention

Patients will receive best supportive care (BSC) and matching placebo tablets. BSC will be determined on a per patient basis. Placebo will be administered orally, once daily once daily for approximately 24 weeks

Group IX: (Part 1) Placebo + BSCPlacebo Group1 Intervention

Patients will receive best supportive care (BSC) and matching placebo tablets. BSC will be determined on a per patient basis. Placebo will be administered orally, once daily until completion of Part 1

0 / 5Eligibility criteria met