Pelabresib for Cancer
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.12345Why 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.12367
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Extension Treatment
Continued pelabresib treatment for patients who previously received pelabresib in a parent study
Survival Follow-up
Collection of survival data, including overall survival and leukemia-free survival, for patients who continued or discontinued treatment
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?
1
Treatment groups
Experimental Treatment
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.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Constellation Pharmaceuticals
Lead Sponsor
Novartis Pharmaceuticals
Lead Sponsor
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
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 ...
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, ...
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 ( ...
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.
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 ...
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_28rfHQIQnZ87BnDK19-5K1aPelabresib (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.
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