NCT06401356 · Novartis Pharmaceuticals
An Extension Study for Patients Previously Enrolled in Studies With Pelabresib
What this study is about
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.
View original scientific description
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.
Interventions
DRUG
Pelabresib
Small molecule inhibitor of bromodomain and extraterminal (BET) protein
Primary outcome measures
Treatment-emergent adverse events (TEAEs) and serious TEAEs
Time frame: 5 years
To evaluate long-term safety in patients who are receiving pelabresib
Survival Follow-up
Time frame: 5 years
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.
Duration of Response (DoR)
Time frame: 5 years
DOR defined as the time from the date of first response to the date of confirmed disease progression
Progression-free survival (PFS)
Time frame: 5 years
PFS defined as the time from first dose to confirmed disease progression or death, whichever occurs first.
Leukemia-free survival (LFS)
Time frame: 5 years
LFS defined as the time from first dose to the date of leukemic transformation or death, whichever occurs first.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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.
- Are willing and able to comply with follow-up procedures.
Exclusion criteria
- 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.
Where
- Los Angeles, California
- Jacksonville, Florida
- Chicago, Illinois
- Ann Arbor, Michigan
- New York, New York
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 11, 2026 · Source of record for eligibility and locations