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Continued treatment with pelabresib for patients from previous studies

An Open-Label, Multicenter, Extension Study for Patients Previously Enrolled in Studies With Pelabresib

Phase 3 Interventional Novartis · NCT06401356

This study is testing whether continued treatment with pelabresib can help people who have already tried it in earlier studies stay healthy and live longer without leukemia.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment50 (estimated)
Ages18 Years and up
SexAll
SponsorNovartis Industry-sponsored
Locations15 sites (Los Angeles, California and 14 other locations)
Trial IDNCT06401356 on ClinicalTrials.gov

What this trial studies

This study provides ongoing access to pelabresib for patients who have previously participated in studies involving this treatment. It aims to gather additional safety and efficacy data while monitoring overall survival and leukemia-free survival rates. Patients who received pelabresib or placebo in earlier studies will be followed up to assess their health outcomes. The study will also collect information on adverse events related to the treatment.

Who should consider this trial

Good fit: Ideal candidates are patients who have previously received pelabresib and are experiencing clinical benefits from the treatment.

Not a fit: Patients who discontinued pelabresib treatment for reasons unrelated to participation in this study will not benefit from ongoing treatment.

Why it matters

Potential benefit: If successful, this study could offer patients continued access to a potentially beneficial treatment while providing valuable data on its long-term effects.

How similar studies have performed: Other studies involving pelabresib have shown promising results, indicating that this approach may build on existing knowledge rather than being entirely novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Eligibility for Ongoing Pelabresib Treatment

   * Able to provide signed informed consent, agreeing to all protocol and ICF requirements.
   * At least 18 years old and legally able to consent in the study's jurisdiction.
   * Previously enrolled and currently receiving pelabresib in a parent study.
   * Demonstrating clinical benefit from pelabresib, as judged by the investigator.
   * Willing and able to follow all study visits, treatments, and procedures.
   * Agree to avoid pregnancy or fathering children:

     * Men: Must use highly effective contraception (≥99% effective) and avoid sperm donation from eligibility check through 94 days post-treatment.
     * Women of childbearing potential (WOCBP): Must test negative for pregnancy at eligibility, use highly effective contraception through 184 days post-treatment, undergo regular pregnancy testing, and avoid breastfeeding and oocyte donation during this period.
     * Women not of childbearing potential (surgically sterile or postmenopausal ≥12 months without other cause) are eligible.

   Note: Women with amenorrhea due to chemo/radiotherapy are considered WOCBP and must use contraception.
2. Eligibility for Survival Follow-up

   * Provide signed informed consent, agreeing to all protocol and ICF requirements.
   * Are at least 18 years old and legally able to consent.
   * Were previously enrolled in a pelabresib clinical study.
   * Are willing and able to comply with follow-up procedures.

Exclusion Criteria:

1. Eligibility for Ongoing Pelabresib Treatment

   * Legally institutionalized or under judicial protection.
   * Enrolled in another interventional clinical trial (excluding the parent study).
   * History of hypersensitivity to pelabresib, its excipients, or similar drugs.
   * Significant gastrointestinal issues (e.g., active IBD, unresolved nausea/vomiting/diarrhea \> Grade 1) that may affect drug absorption.
   * Any medical condition deemed unsuitable by the investigator.
   * Uncontrolled illness or condition that may compromise safety or protocol compliance.
   * Received systemic anticancer or investigational treatment (excluding parent study drug or hormonal therapy) within 2 weeks or 5 half-lives before first dose. (Hydroxyurea/anagrelide allowed up to 24 hours prior.)
   * Received hematopoietic growth factors or androgenic steroids within 4 weeks before first dose.
   * Used strong CYP3A4 inhibitors/inducers (e.g., St. John's wort) within 2 weeks before first dose. Use during treatment is prohibited.
   * Female participants who are pregnant, breastfeeding, or not using required contraception.
   * Male participants who do not agree to use contraception or refrain from sperm donation as specified.
   * Unwilling or unable to comply with the study protocol.
2. Eligibility for Survival Follow-up • They are legally institutionalized or under judicial protection.

Where this trial is running

Los Angeles, California and 14 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
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Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.