Pelabresib for Cancer

Recruiting at 18 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Constellation Pharmaceuticals

Must be taking: Pelabresib

Disqualifiers: Pregnancy, Breastfeeding, Uncontrolled illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the effectiveness and safety of a treatment called pelabresib for individuals with certain types of cancer. It includes those who participated in previous pelabresib studies and experienced benefits, as well as those who received a placebo and wish to try the actual treatment. Participants who previously found improvement with pelabresib might be suitable candidates. The trial aims to determine if pelabresib can extend life or help individuals remain free from leukemia, a type of blood cancer. As a Phase 3 trial, it represents the final step before FDA approval, providing an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that pelabresib is likely to be safe for humans?

Research has shown that pelabresib has been tested for safety in people with various types of cancer. In one study, researchers monitored 426 patients taking pelabresib for side effects. Anemia (a low red blood cell count) and tiredness were the most common side effects. About 44% of patients experienced anemia, while tiredness was common but usually not severe.

In another study with 64 patients, 58% completed at least one round of treatment. This indicates that more than half of the patients could continue the treatment for some time, suggesting it is generally well-tolerated.

These studies indicate that while pelabresib has some side effects, many patients can continue taking it. For those considering joining a trial, these findings suggest the treatment is manageable for many people. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for cancer?

Pelabresib is unique because it targets a specific protein involved in cancer cell growth, offering a novel approach compared to the standard chemotherapy or targeted therapies like tyrosine kinase inhibitors. This treatment stands out due to its mechanism of inhibiting bromodomain and extra-terminal (BET) proteins, which play a role in regulating cancer-related genes. Researchers are excited about Pelabresib because it has the potential to stop cancer progression more effectively and with fewer side effects than traditional treatments.

What evidence suggests that pelabresib might be an effective treatment for cancer?

Studies have shown that pelabresib, particularly when combined with ruxolitinib, effectively treats myelofibrosis, a type of bone marrow cancer. Patients experienced significant symptom improvement and reduced spleen size, both important treatment goals. In some cases, patients achieved complete remission, with no signs of cancer detected. These results suggest that pelabresib holds promise for treating blood-related cancers. In this trial, all eligible participants will continue receiving pelabresib, underscoring its strong potential as a treatment option for these conditions. ^{1 2 3 6 7}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for adult patients who have solid or blood cancers and were previously treated with Pelabresib in an earlier study. They must be benefiting from the treatment, able to follow the study plan, and give informed consent. Only those still receiving benefits from Pelabresib will continue treatment; others can participate in survival data collection.

Inclusion Criteria

Eligibility for Ongoing Pelabresib Treatment: Receiving clinical benefit from treatment with pelabresib in the parent study

Eligibility for Survival Follow-up (Overall Survival and Leukemia-Free Survival): Patient has been enrolled in a clinical study with pelabresib

I am willing and able to follow the study's treatment and visit schedule.

Exclusion Criteria

Eligibility for Ongoing Pelabresib Treatment: 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

Eligibility for Survival Follow-up (Overall Survival and Leukemia-Free Survival): Patients who are legally institutionalized or under judicial protection

I am not pregnant or breastfeeding.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Extension Treatment

Continued pelabresib treatment for patients who previously received pelabresib in a parent study

5 years

Regular visits as per study protocol

Survival Follow-up

Collection of survival data, including overall survival and leukemia-free survival, for patients who continued or discontinued treatment

5 years

Data collection visits

What Are the Treatments Tested in This Trial?

Interventions

• Pelabresib

Trial Overview The trial provides ongoing access to Pelabresib for eligible patients from a previous study. It aims to gather more information on safety, effectiveness, leukemia-free survival, and overall lifespan after treatment ends.

How Is the Trial Designed?

Group I: Pelabresib Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Constellation Pharmaceuticals

Lead Sponsor

Trials

10

Recruited

1,500+

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10010313/

A Phase I Study of Pelabresib (CPI-0610), a Small- ... Patients treated with as low as 48 mg QD pelabresib achieved complete remission during the study. This preliminary efficacy data suggests modest clinical ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10431317/

P1018: UPDATED RESULTS FROM MANIFEST ARM 2 ... Treatment with PELA as add-on to RUX in pts with a suboptimal/lost response to RUX monotherapy resulted in durable and deepening splenic and symptom responses, ...

3. en.wikipedia.org en.wikipedia.org/wiki/Pelabresib

Pelabresib As of May 2025, pelabresib is in Phase III clinical trials for MF and has shown promising results in combination with ruxolitinib, a Janus kinase inhibitor ( ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04603495

NCT04603495 | Phase 3 Study of Pelabresib (CPI-0610) in ... A Phase 3, randomized, blinded study comparing pelabresib (CPI-0610) and ruxolitinib with placebo and ruxolitinib in myelofibrosis (MF) patients.

5. ash.confex.com ash.confex.com/ash/2024/webprogram/Paper193193.html

Paper: Updated Results from the Phase 3 Manifest-2 Study ... As of March 29, 2024, all pts assessed had been followed for ≥48 wks; 58.9% (126/214) and 62.0% (134/216) of pts continued on double-blind treatment in the PELA ...

6. asco.org asco.org/abstracts-presentations/ABSTRACT448880

Updated safety and efficacy data from the phase 3 ... Of 426 pts evaluated for safety, the most common treatment-emergent AEs (≥20%) in the PELA+RUX vs PBO+RUX arms were anemia (43.9% vs 55.6% [Grade ≥3, 23.1% vs ...

7. medchemexpress.com medchemexpress.com/CPI-0610.html?srsltid=AfmBOoqXZ1EhdKWvjeavskAWlj16rtbf_28rfHQIQnZ87BnDK19-5K1a

Pelabresib (CPI-0610) | BET Inhibitor Pelabresib (CPI-0610) is a potent, selective, orally active and cell-active BET inhibitor. Pelabresib inhibits BRD4-BD1 with an IC 50 of 39 nM.