An Extension Study for Patients Previously Enrolled in Studies with Pelabresib

Phase 3

Recruiting

• Conditions: Hematologic Malignancy; Solid Tumor; Advanced Malignancies
• Interventions: Drug: Pelabresib

• Registration Number: NCT06401356
• Lead Sponsor: Constellation Pharmaceuticals

Brief Summary

The purpose of this study is:

1. To provide continued pelabresib treatment to patients who have previously received pelabresib in a parent study and continue collecting safety and efficacy information

2. To evaluate the overall survival and leukemia-free survival of patients who discontinued placebo or pelabresib treatment in pelabresib parent studies

Detailed Description

The purpose of this study is to provide continued access to pelabresib treatment for patients who previously received pelabresib in a parent study and to continue collecting information on safety and efficacy.

By collecting efficacy and safety information, the study team will monitor the efficacy of pelabresib with regard to disease status and survival, as well as information on AEs, including SAEs and AEs of special interest (AESIs),

Additionally, survival follow-up data will be collected from patients who continued treatment with pelabresib in this extension study, and may also be collected from patients who discontinued treatment with placebo or pelabresib in pelabresib parent studies.

During the survival follow-up, the team will collect information on the leukaemia-free survival status and overall survival (lifespan) of the patient during and after the end of treatment.

If a patient discontinued pelabresib treatment in the parent study for any other reason than participation in this study, they will not receive further pelabresib treatment in this extension study, but they can enter the study for survival-follow up only.

Study Timeline

• Study First Submitted: 2024-05-02
• Study First Submitted QC: 2024-05-02
• Study First Posted: 2024-05-06
• Study Start: 2024-08-13
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-16
• Last Update Posted: 2024-12-17
• Primary Completion: 2029-06-30
• Study Completion: 2029-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Eligibility for Ongoing Pelabresib Treatment

1. Capable of giving signed informed consent
2. Receiving clinical benefit from treatment with pelabresib in the parent study
3. Willingness and ability to comply with scheduled visits, treatment plans, and any other study procedures

Eligibility for Survival Follow-up (Overall Survival and Leukemia-Free Survival)

1. Capable of giving signed informed consent Patient must be at least 18 years of age and the legal age of consent in the jurisdiction in which the study is taking place at the time of signing the ICF
2. Patient has been enrolled in a clinical study with pelabresib
3. Willingness and ability to comply with ongoing follow-up as indicated in this protocol

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Exclusion Criteria

Eligibility for Ongoing Pelabresib Treatment

1. Patients who are legally institutionalized or under judicial protection
2. Any medical condition that, in the investigator's opinion, makes the patient unsuitable for the study
3. Patient is able to access pelabresib outside the study
4. Patient with an uncontrolled intercurrent illness or any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol
5. Female patients who are pregnant or breastfeeding

Eligibility for Survival Follow-up (Overall Survival and Leukemia-Free Survival)

1. Patients who are legally institutionalized or under judicial protection

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Pelabresib	Pelabresib	This is a multicenter, open-label extension study for patients previously enrolled in studies with pelabresib. The study does not define separate treatment arms. The study provides continued pelabresib treatment for patients who previously received pelabresib in a parent study. Patients 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.

Primary Outcome Measures

Name	Time	Method
Survival	5 years	Overall survival and leukemia-free survival
Safety: TEAEs and Serious TEAEs	5 years	Occurrence and severity of treatment-emergent adverse events (TEAEs) and serious TEAEs among patients who are receiving pelabresib
Time to event endpoints	5 years	Time to event endpoints (eg, duration of response, progression-free survival) in patients who are receiving or received pelabresib

Trial Locations

Locations (15)

Hematologic Malignancy/Stem (Department of Medicine, Division of Hematology/Oncology); 🇺🇸 Los Angeles, California, United States

Mayo Clinic (Cancer Clinical Research Office); 🇺🇸 Jacksonville, Florida, United States

Northwestern Memorial Hospital (Oncology); 🇺🇸 Chicago, Illinois, United States

UMHS - University of Michigan Medical Center (Radiation Oncology); 🇺🇸 Ann Arbor, Michigan, United States

Mount Sinai Hospital - Oncology; 🇺🇸 New York, New York, United States

Weill Cornell Medicine - New York Presbyterian Hospital (Oncology); 🇺🇸 New York, New York, United States

ZNA Cadix-Hematology; 🇧🇪 Antwerpen, Belgium

Az St-Jan Brugge-Oostende A.V.; 🇧🇪 Brugge, Belgium

AOU Careggi (Department of Experimental and Clinical Medicine); 🇮🇹 Firenze, Italy

Azienda Ospedaliero - Universitaria Maggiore della Carità (SCDU Ematologia); 🇮🇹 Novara, Italy

AO Ospedale di Circolo, PO Varese (Ematologia); 🇮🇹 Varese, Italy

Amsterdam UMC location Vumc (Hematology); 🇳🇱 Amsterdam, Netherlands

Cardiff and Vale University Health Board - University Hospital Wales (Hematology); 🇬🇧 Cardiff, United Kingdom

Beatson West of Scotland Cancer Centre (Hematology); 🇬🇧 Glasgow, United Kingdom

Guys and St Thomas' Hospital - Haematology; 🇬🇧 London, United Kingdom