An Extension Study for Patients Previously Enrolled in Studies With Pelabresib

NCT06401356 | Recruiting Phase 3 FDA Drug

• 3 other identifiers: CDAK539A12001BCNST0610C2102023-508950-24-00
• Study Type: interventional
• Target: 50
• Locations: 5 countries
• Sites: 15

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

Outcome Measures

Primary Outcomes (5)

• Treatment-emergent adverse events (TEAEs) and serious TEAEs

  To evaluate long-term safety in patients who are receiving pelabresib

  5 years

• Survival Follow-up

  Survival Follow-up consists of Survival and Leukemia-Free Survival Follow-up. All participants in the extension study will be followed up for Survival, while participants with hematological malignancies will be followed up for Survival and Leukemia-Free Survival. In addition, participants who will not receive pelabresib treatment may enter this extension protocol to be only followed up for Survival.

  5 years

• Duration of Response (DoR)

  DOR defined as the time from the date of first response to the date of confirmed disease progression

  5 years

• Progression-free survival (PFS)

  PFS defined as the time from first dose to confirmed disease progression or death, whichever occurs first.

  5 years

• Leukemia-free survival (LFS)

  LFS defined as the time from first dose to the date of leukemic transformation or death, whichever occurs first.

  5 years

Study Arms (1)

Pelabresib

EXPERIMENTAL

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.

Drug: Pelabresib

Interventions

Pelabresib DRUG

Small molecule inhibitor of bromodomain and extraterminal (BET) protein

Also known as: CPI-0610

Pelabresib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• Eligibility for Survival Follow-up • They are legally institutionalized or under judicial protection.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (15)

Hematologic Malignancy/Stem (Department of Medicine, Division of Hematology/Oncology)

Los Angeles, California, 90095-8344, United States

RECRUITING

Mayo Clinic (Cancer Clinical Research Office)

Jacksonville, Florida, 32224-1865, United States

RECRUITING

Northwestern Memorial Hospital (Oncology)

Chicago, Illinois, 60611-5957, United States

RECRUITING

UMHS - University of Michigan Medical Center (Radiation Oncology)

Ann Arbor, Michigan, 48109-5000, United States

RECRUITING

Mount Sinai Hospital - Oncology

New York, New York, 10029, United States

RECRUITING

Weill Cornell Medicine - New York Presbyterian Hospital (Oncology)

New York, New York, 10065, United States

RECRUITING

ZNA Cadix-Hematology

Antwerp, 2020, Belgium

RECRUITING

Az St-Jan Brugge-Oostende A.V.

Bruges, 8000, Belgium

RECRUITING

AOU Careggi (Department of Experimental and Clinical Medicine)

Florence, 50141, Italy

RECRUITING

Azienda Ospedaliero - Universitaria Maggiore della Carità (SCDU Ematologia)

Novara, 28100, Italy

ENROLLING BY INVITATION

AO Ospedale di Circolo, PO Varese (Ematologia)

Varese, 21100, Italy

RECRUITING

Amsterdam UMC location Vumc (Hematology)

Amsterdam, 1081 HV, Netherlands

RECRUITING

Cardiff and Vale University Health Board - University Hospital Wales (Hematology)

Cardiff, CF14 4XW, United Kingdom

RECRUITING

Beatson West of Scotland Cancer Centre (Hematology)

Glasgow, G12 0YN, United Kingdom

RECRUITING

Guys and St Thomas' Hospital - Haematology

London, SE1 9RT, United Kingdom

RECRUITING

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

CPI-0610

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

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

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 2, 2024
• First Posted: May 6, 2024
• Study Start: August 13, 2024
• Primary Completion (Estimated): June 2, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: June 22, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share

Locations

NL (1); US (6); BE (2); IT (3); GB (3)