Hematologic MalignancyPhase 3NCT06401356
An Extension Study for Patients Previously Enrolled in Studies With Pelabresib
A Phase 3 study testing Pelabresib in people with Hematologic Malignancy and Solid Tumor, run by Novartis Pharmaceuticals at 15 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Malignancy, Solid Tumor, Advanced Malignancies
- What is being tested?
- Pelabresib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 15 sitesin 5 countries
- Study dates
- 13 Aug 2024to 30 Jun 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
15 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
15 sites in 5 countries
United States6 sites, 6 recruiting
- Recruiting
Uni Of Michigan Health System
Ann Arbor, Michigan, 48109
Hayley Demchak, hdemchak@med.umich.edu
Moshe Talpaz
- Recruiting
Northwestern University
Chicago, Illinois, 60611
Gabby Perea, 312-695-1371, gabrielle.perea@northwestern.edu
Brady L Stein
- Recruiting
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224
Tina Kothare, 904-953-6153, Kothare.Tina@mayo.edu
Candido Rivera
- Recruiting
University of California LA
Los Angeles, California, 90095
Nicole Alimi NP, 310-825-5513, nalimi@mednet.ucla.edu
Gary Schiller
- Recruiting
New York Presbyterian Hospital
New York, New York, 10021
Penina Stewart, 212-746-2932, pes4006@med.cornell.edu
Joseph Scandura
- Recruiting
Mt Sinai Medical Center
New York, New York, 10029-6574
Carly Sullivan, 347-714-3285, carly.sullivan@mssm.edu
Marina Kremyanskaya
Italy3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Florence, FI, 50134
- Recruiting
Novartis Investigative Site
Novara, 28100
- Recruiting
Novartis Investigative Site
Varese, VA, 21100
United Kingdom3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Cardiff, CF14 4XW
- Recruiting
Novartis Investigative Site
Glasgow, G12 0YN
- Recruiting
Novartis Investigative Site
London, SE1 7EH
Belgium2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Antwerp, 2030
- Recruiting
Novartis Investigative Site
Bruges, 8000
Netherlands1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Amsterdam, 1081
About this study
Official title: An Open-Label, Multicenter, Extension Study for Patients Previously Enrolled in Studies With Pelabresib
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).
Read the detailed description
The study will collect and monitor ongoing efficacy information with regard to disease and survival status, as well as safety information with regard to AEs, including SAEs and AEs of special interest (AESI).
Patients are eligible to continue pelabresib treatment as long as the regimen is tolerated, and the patient does not meet any of the discontinuation criteria.
There will be an eligibility check period of up to 28 days for this extension study. Patients who fulfill the inclusion and exclusion criteria are eligible for the study. After the patient willingly provides consent, eligibility will be assessed by the investigator and treatment with pelabresib can be started at the same dose and dosing schedule that was applied in the parent study in the last cycle prior to EOT unless there is need for dose adjustment due to toxicity.
A patient is considered to have completed the study after completion of all visits as defined in the Schedule of Assessments (SoA) as per protocol, including the Safety Follow-up visit and Survival Follow-up.
The Safety Follow-up period is the interval between the EOT visit and the scheduled Safety Follow-up visit, which should occur 30 days (±3 days) after the last dose of pelabresib. For patients who switch from investigational pelabresib to commercial pelabresib treatment, the Safety Follow-up period is the time from the last dose of investigational pelabresib until the first dose of commercial pelabresib. The treatment start date with commercial pelabresib will be considered the date of "early Safety Follow-up termination." All patients will be followed for AEs and SAEs for 30 days (±3 days) following the last dose of pelabresib on the extension study or until the initiation of a new anticancer therapy, whichever occurs first.
Reasonable efforts should be made to have the patient return for the Safety Follow-up visit and review any AEs that may occur during this period. If the patient cannot return to the site for the Safety Follow-up visit, then the patient should be contacted by telephone for assessment of AEs.
The end of study will occur when all parent studies have been closed with no further patients expected to be enrolled into the extension study, and the last patient in the extension study has permanently discontinued study treatment (investigational pelabresib) and has completed the Safety Follow-up visit or is designated as lost to follow-up, died, or has withdrawn consent, whichever comes first. The sponsor reserves the right to close a study site or terminate the study at any time for any reason at the sole discretion of the sponsor.
- Conditions
- Hematologic Malignancy, Solid Tumor, Advanced Malignancies
- Being tested
- Pelabresib
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PelabresibDrug
Also called CPI-0610
Small molecule inhibitor of bromodomain and extraterminal (BET) protein
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Pelabresib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent adverse events (TEAEs) and serious TEAEs
To evaluate long-term safety in patients who are receiving pelabresib
Measured: 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.
Measured: 5 years
Duration of Response (DoR)
DOR defined as the time from the date of first response to the date of confirmed disease progression
Measured: 5 years
Progression-free survival (PFS)
PFS defined as the time from first dose to confirmed disease progression or death, whichever occurs first.
Measured: 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.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2024
- Study start13 Aug 2024
- Primary completion2 Jun 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic MalignancyStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06401356
- Sponsor's protocol ID
- CDAK539A12001B
- Other IDs
- CNST0610C210, 2023-508950-24-00, 2023
- Status last verified
- Jun 2026
- First submitted
- 2 May 2024
- Last updated
- 11 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multicenter, Extension Study for Patients Previously Enrolled in Studies With Pelabresib
- ClinicalTrials.gov ID
- NCT06401356
- Acronym / sponsor ID
- CDAK539A12001B
- Conditions
- Hematologic Malignancy, Solid Tumor, Advanced Malignancies
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 13 Aug 2024 (actual)
- Primary completion
- 2 Jun 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 6 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
The study will collect and monitor ongoing efficacy information with regard to disease and survival status, as well as safety information with regard to AEs, including SAEs and AEs of special interest (AESI).
Patients are eligible to continue pelabresib treatment as long as the regimen is tolerated, and the patient does not meet any of the discontinuation criteria.
There will be an eligibility check period of up to 28 days for this extension study. Patients who fulfill the inclusion and exclusion criteria are eligible for the study. After the patient willingly provides consent, eligibility will be assessed by the investigator and treatment with pelabresib can be started at the same dose and dosing schedule that was applied in the parent study in the last cycle prior to EOT unless there is need for dose adjustment due to toxicity.
A patient is considered to have completed the study after completion of all visits as defined in the Schedule of Assessments (SoA) as per protocol, including the Safety Follow-up visit and Survival Follow-up.
The Safety Follow-up period is the interval between the EOT visit and the scheduled Safety Follow-up visit, which should occur 30 days (±3 days) after the last dose of pelabresib. For patients who switch from investigational pelabresib to commercial pelabresib treatment, the Safety Follow-up period is the time from the last dose of investigational pelabresib until the first dose of commercial pelabresib. The treatment start date with commercial pelabresib will be considered the date of "early Safety Follow-up termination." All patients will be followed for AEs and SAEs for 30 days (±3 days) following the last dose of pelabresib on the extension study or until the initiation of a new anticancer therapy, whichever occurs first.
Reasonable efforts should be made to have the patient return for the Safety Follow-up visit and review any AEs that may occur during this period. If the patient cannot return to the site for the Safety Follow-up visit, then the patient should be contacted by telephone for assessment of AEs.
The end of study will occur when all parent studies have been closed with no further patients expected to be enrolled into the extension study, and the last patient in the extension study has permanently discontinued study treatment (investigational pelabresib) and has completed the Safety Follow-up visit or is designated as lost to follow-up, died, or has withdrawn consent, whichever comes first. The sponsor reserves the right to close a study site or terminate the study at any time for any reason at the sole discretion of the sponsor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pelabresib | Experimental | Drug: PelabresibAll 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Eligibility for Ongoing Pelabresib Treatment
- Note: Women with amenorrhea due to chemo/radiotherapy are considered WOCBP and must use contraception.
- Eligibility for Survival Follow-up
Key exclusion criteria (14 listed)
- Eligibility for Ongoing Pelabresib Treatment
- Eligibility for Survival Follow-up • They are legally institutionalized or under judicial protection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PelabresibAlso: CPI-0610Small molecule inhibitor of bromodomain and extraterminal (BET) protein | Drug | Pelabresib |
Primary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2026.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
15 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pelabresib in people with Hematologic Malignancy and Solid Tumor, run by Novartis Pharmaceuticals at 15 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Italy, United Kingdom, Belgium, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-emergent adverse events (TEAEs) and serious TEAEs (time frame: 5 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2026.