Navitoclax + Ruxolitinib for Myelofibrosis (TRANSFORM-1 Trial)

Palo Alto (17 mi)

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 is a type of bone marrow cancer that usually develops slowly and disrupts body's normal production of blood cells. It causes bone marrow scarring, leading to severe anemia that can cause weakness and fatigue. It can also cause a low number of blood-clotting cells called platelets, which increases risk of bleeding. Myelofibrosis often causes an enlarged spleen. The purpose of this study is to see if a combination of navitoclax and ruxolitinib is more effective and safe in assessment of change in spleen volume when compared to ruxolitinib in participants with myelofibrosis. Navitoclax is an investigational drug for the treatment of myelofibrosis. Participants in this study are divided into two groups, called treatment arms. Each group receives a different treatment. Adult participants with a diagnosis of myelofibrosis will be enrolled. Around 230 participants will be enrolled in approximately 190 sites worldwide. Participants will receive oral navitoclax tablet with oral ruxolitinib tablet or oral ruxolitinib tablet with oral placebo (no active drug) tablet and treatment may continue untill the participant cannot tolerate the study drug, or benefit is not achieved, or other reasons which qualify for discontinuation of the study drug. There may be a higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, magnetic resonance imaging (MRI) or computed tomography (CT) scan, bone marrow tests, checking for side effects, and completing questionnaires.

Eligibility Criteria

Adults with myelofibrosis, a bone marrow cancer causing anemia and enlarged spleen. Participants must have certain levels of disease severity and symptoms, be able to complete symptom assessments, and not be eligible for stem cell transplantation due to various reasons.

Inclusion Criteria

My spleen is enlarged, confirmed by a scan.

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

I have severe symptoms of my condition, scoring 3 or more on at least two.

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

I have been diagnosed with Primary MyeloFibrosis or Secondary MyeloFibrosis.

Exclusion Criteria

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

I have been treated with a JAK-2 inhibitor before.

Treatment Details

The trial is testing if taking Navitoclax tablets along with Ruxolitinib is better than just Ruxolitinib for reducing spleen size in myelofibrosis patients. It's a global study involving around 230 participants who will receive treatments orally.

2Treatment groups

Experimental Treatment

Active Control

Group I: Navitoclax + RuxolitinibExperimental Treatment2 Interventions

Participants will receive Navitoclax in combination with Ruxolitinib

Group II: Placebo for Navitoclax + RuxolitinibActive Control2 Interventions

Participants will receive placebo for Navitoclax and Ruxolitinib