(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Conditions

• Indolent Systemic Mastocytosis
• Smoldering Systemic Mastocytosis

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Elenestinib — DRUG
  Elenestinib oral tablet
• Placebo — DRUG
  Placebo oral tablet

Study Details

This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants with ISM.

Key Dates

Start date

Nov 30, 2021

Status verified

May 2026

Primary completion

Sep 30, 2032

Completion

Sep 30, 2032

Study Design

Enrollment

534 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: (Part 1) Elenestinib Dose 1 + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
• Experimental: (Part 1) Elenestinib Dose 2 + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
• Experimental: (Part 1) Elenestinib Dose 3 + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
• Placebo Comparator: (Part 1) Placebo + SDT
  Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily until completion of Part 1.
• Experimental: (Part 2) Elenestinib Dose 1 + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for approximately 48 weeks.
• Placebo Comparator: (Part 2) Placebo + SDT
  Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily for approximately 48 weeks.
• Experimental: (Part 3) Elenestinib + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
• Experimental: (Part S) Elenestinib Dose 1 + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
• Experimental: (Part K) Elenestinib Dose 1 + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
• Experimental: (PK groups) Elenestinib + SDT
  Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.

Primary Outcome Measure

Part 1: Number of participants with Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to 12 weeks ]

Central Contacts

• Blueprint Medicines
  617-714-6707
  [email protected]

Locations (14)

Find similar trials in Birmingham, AL

Related Studies

• An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
  Recruiting · Blueprint Medicines Corporation · Chestnut Hill, Massachusetts