Navitoclax + Ruxolitinib for Myelofibrosis
(TRANSFORM-2 Trial)

Recruiting in Palo Alto (17 mi)

+329 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: AbbVie

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?Myelofibrosis (MF) is a rare blood cancer, notable for scarring of the bone marrow (the spongy tissue inside bones) and the spleen becoming larger. The purpose of this study is to assess safety and change in spleen volume when navitoclax is given in combination with ruxolitinib, compared to best available therapy, for adult participants with MF. Navitoclax is an investigational drug (not yet approved) being developed for the treatment of MF. Participants in this study will be randomly selected (like picking numbers out of a hat) to be in 1 of 2 treatment arms. Neither participants nor the study doctor will be able to pick which treatment arm a participants enters. In Arm A, participants will receive navitoclax in combination with ruxolitinib. In Arm B, participants will receive the best available therapy (BAT) for MF. Adult participants with a diagnosis of MF that came back or did not get better after earlier treatment will be enrolled. Approximately 330 participants will be enrolled in approximately 210 sites across the world. In Arm A, participants will receive navitoclax tablet by mouth once daily with by mouth ruxolitinib tablet twice daily. In Arm B, participants will receive the BAT available to the investigator. Participants will receive the study drug until they experience no benefit (determined by the investigator), participants cannot tolerate the study drugs, or participants withdraw consent. The approximate treatment duration is about 3 years. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of treatment will be checked by medical assessments, blood and bone marrow tests, checking for side effects, and completing questionnaires.

Eligibility Criteria

Adults with relapsed or refractory Myelofibrosis, a type of blood cancer that causes bone marrow scarring and spleen enlargement. Participants must have specific symptoms and spleen size criteria, be classified as intermediate-2 or high-risk by DIPSS+, and have been treated with ruxolitinib previously but not responded well.

Inclusion Criteria

My spleen has grown and can be felt at least 5 cm below my left rib cage since starting ruxolitinib.

My myelofibrosis is classified as intermediate-2 or high-risk.

I can take care of myself and am up and about more than half of my waking hours.

+13 more

Exclusion Criteria

I have previously been treated with specific cancer drugs or had a stem cell transplant.

I am not on blood thinners, except for low dose aspirin or LMWH.

I've completed my current cancer treatment at least a week ago, or it's been 30 days, whichever is shorter.

+1 more

Participant Groups

The trial is testing the safety and effectiveness of an oral Navitoclax tablet in combination with Ruxolitinib versus Best Available Therapy (BAT) for reducing spleen volume in Myelofibrosis patients. It's a randomized study where neither participants nor doctors choose the treatment group.

2Treatment groups

Experimental Treatment

Active Control

Group I: Arm A: Navitoclax + RuxolitinibExperimental Treatment2 Interventions

Participants will receive navitoclax tablets once daily and ruxolitinib tablets twice daily.

Group II: Arm B: Best Available Therapy (BAT)Active Control1 Intervention

Participants will receive one of the BAT options, per the investigator's discretion.