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

Join the HARBOR study for indolent systemic mastocytosis research.

Recruiting
18 years and older
All
Phase 2/3

This study is testing a medicine called elenestinib (BLU-263) to see if it helps people with a condition called indolent systemic mastocytosis (ISM). ISM is a condition where certain cells in the body, called mast cells, grow too much and cause symptoms like redness or itching. Elenestinib is being compared to a placebo (a pill with no medicine) to see if it works better. The study is in two main parts, and people in the study will get either elenestinib or a placebo, along with their regular treatment. In the last part, everyone gets elenestinib to see how well it works. People who already tried a similar medicine can join a special part of the study.

  • The study will have three parts, and you may receive treatment for a long time.
  • Requirements include stable symptom management before joining.
  • Some people may not be able to join if they have other health issues.
Study details
    Indolent Systemic Mastocytosis
    Smoldering Systemic Mastocytosis

NCT04910685

Blueprint Medicines Corporation

25 April 2026

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Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

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The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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