Pelabresib for Myelofibrosis
(MANIFEST-2 Trial)

Recruiting in Palo Alto (17 mi)

+205 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Constellation Pharmaceuticals

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial tests a new drug combination of pelabresib and ruxolitinib in patients with myelofibrosis who haven't tried certain treatments before. Pelabresib blocks harmful proteins, and ruxolitinib stops harmful signals to see if this combination works better.

Eligibility Criteria

This trial is for adults with myelofibrosis, including those who developed it from polycythemia vera or essential thrombocythemia. Participants must have certain symptoms and spleen enlargement, be in a specific risk category, and have good organ function. They can't join if they've had their spleen removed recently or used JAKi/BET inhibitors before.

Inclusion Criteria

I have been diagnosed with myelofibrosis.

My blood, kidney, and liver functions are all within normal ranges.

My condition is rated Intermediate-1 or higher on the DIPSS scale.

+4 more

Exclusion Criteria

I have had my spleen removed or treated with radiation in the last 6 months.

I don't have any health conditions or take medications that would stop me from receiving treatment.

I have been treated with JAKi or BET inhibitors for a blood disorder.

Participant Groups

The study tests Pelabresib (a BET protein inhibitor) combined with Ruxolitinib versus a placebo with Ruxolitinib in patients new to JAK inhibitor treatments. It's randomized and blinded, meaning participants are assigned by chance to either the test drug or placebo without knowing which one they receive.

2Treatment groups

Experimental Treatment

Active Control

Group I: Pelabresib + ruxolitinibExperimental Treatment2 Interventions

Pelabresib monohydrate tablets + ruxolitinib phosphate tablets

Group II: Placebo + ruxolitinibActive Control2 Interventions

Matching placebo tablets + ruxolitinib phosphate tablets