Recruiting
Phase 3

Pelabresib

Sponsor:

Novartis Pharmaceuticals

Code:

NCT06401356

Conditions

Hematologic Malignancy

Solid Tumor

Advanced Malignancies

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Pelabresib

Study Details

Brief summary:

The purpose of this study is to evaluate the long-term safety and the clinical benefit of pelabresib in patients with hematological and/or solid tumor indications or advanced malignancies. Additionally, participants previously enrolled in studies with pelabresib who received placebo or participants who discontinued pelabresib (for any other reason than participating in this extension study), may be enrolled in this extension study to evaluate the survival and leukemia-free survival (for patients with hematological malignancies) or only the Survival Follow-up (for all the other patients).

Conditions

Hematologic Malignancy

Solid Tumor

Advanced Malignancies

Study ID

NCT06401356

Start date

Aug 13, 2024

Status verified date

Jun, 2026

Completion date

Jun 30, 2027

Anticipated

Primary completion date

Jun 2, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

50 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Pelabresib

All eligible participants will receive continued treatment with pelabresib as administered in the relevant parent study.

Participants previously enrolled in studies with pelabresib who discontinued treatment with placebo or pelabresib may be enrolled in this extension study for the purpose of survival follow-up.

Interventions

Pelabresib

Small molecule inhibitor of bromodomain and extraterminal (BET) protein

Primary outcome measure

  • Treatment-emergent adverse events (TEAEs) and serious TEAEs [ Time Frame: 5 years ]
  • Survival Follow-up [ Time Frame: 5 years ]
  • Duration of Response (DoR) [ Time Frame: 5 years ]
  • Progression-free survival (PFS) [ Time Frame: 5 years ]
  • Leukemia-free survival (LFS) [ Time Frame: 5 years ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111novartis.email@novartis.com

Locations

University of California LA

Recruiting

Los Angeles, California, United States, 90095

Contacts

Nicole Alimi NP

310-825-5513nalimi@mednet.ucla.edu

Principal Investigator:

Gary Schiller

Mayo Clinic Jacksonville

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Tina Kothare

904-953-6153Kothare.Tina@mayo.edu

Principal Investigator:

Candido Rivera

Northwestern University

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Gabby Perea

312-695-1371gabrielle.perea@northwestern.edu

Principal Investigator:

Brady L Stein

Uni Of Michigan Health System

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Hayley Demchak

hdemchak@med.umich.edu

Principal Investigator:

Moshe Talpaz

New York Presbyterian Hospital

Recruiting

New York, New York, United States, 10021

Contacts

Penina Stewart

212-746-2932pes4006@med.cornell.edu

Principal Investigator:

Joseph Scandura

Mt Sinai Medical Center

Recruiting

New York, New York, United States, 10029-6574

Contacts

Carly Sullivan

347-714-3285carly.sullivan@mssm.edu

Principal Investigator:

Marina Kremyanskaya

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Jun 11, 2026

Last verified

Jun, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-06-11.