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Compensation: Varies

Number of Visits: 8

Duration: 24 weeks

309 Participants Needed

Luspatercept for Myelofibrosis
(INDEPENDENCE Trial)

Recruiting at 363 trial locations

Overseen ByFirst line of the email MUST contain the NCT# and Site #.

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Celgene

Must be taking: JAK2 inhibitors

Must not be taking: Hydroxyurea, Immunomodulatory compounds

Disqualifiers: Uncontrolled hypertension, Prior malignancies, HIV, others

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

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

The purpose of this Phase 3 study is to evaluate the efficacy and safety of Luspatercept compared with placebo in subjects with myeloproliferative neoplasm (MPN)-associated Myelofibrosis (MF) and anemia on concomitant Janus kinase 2 (JAK2) inhibitor therapy and who require red blood cell count (RBC) transfusions. The study is divided into Screening Period, a Treatment Phase (consisting of a Blinded Core Treatment Period, a Day 169 Response Assessment, a Blinded Extension Treatment Period, and an Open-label Extension Treatment Period), and a Posttreatment Follow-up Period. Following the Day 169 Response Assessment, subjects who did not show clinical benefit will have the option to unblind. Subjects who were on placebo during the Blinded Core Treatment Period will have the opportunity to crossover into the Open-Label Extension Treatment Period and receive Luspatercept.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you must be on a stable dose of a JAK2 inhibitor for at least 16 weeks before and 24 weeks after joining the study. Some medications like hydroxyurea and certain immunomodulatory drugs must be stopped 8 weeks before starting the trial.

Research Team

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Eligibility Criteria

Adults diagnosed with Myelofibrosis associated with a myeloproliferative neoplasm, on stable JAK2 inhibitor therapy, needing regular blood transfusions due to anemia. Participants must have adequate organ function and not be pregnant or breastfeeding. They should agree to contraception if of childbearing potential and not have other significant health issues that could interfere with the study.

Inclusion Criteria

Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.

If you are sexually active with the opposite gender, you must agree to use effective contraception without interruption during the study and for 12 weeks after the study ends. If you choose not to use contraception, you must commit to not having any sexual contact and have it documented every month.

I am a male willing to use contraception or practice abstinence during and after the study for 12 weeks.

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Exclusion Criteria

I have not had a major bleeding event in the last 6 months.

Subject with any of the following laboratory abnormalities at screening: Neutrophils: < 1 x 109/L, WBC: > 100 x 109/L, Platelets: not < 25 x 109/L or > 1000 x 109/L, Peripheral blood myeloblasts:> 5%, Estimated glomerular filtration rate:< 40 mL/min/1.73 m2, AST or ALT:> 3.0 x ULN, Direct bilirubin: ≥ 2 x ULN.

I haven't had a stroke, heart attack, or blood clots in the past 6 months.

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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Luspatercept or placebo via subcutaneous injection every 3 weeks during the Blinded Core Treatment Period, followed by a Day 169 Response Assessment and potential crossover to Open-label Extension Treatment Period

24 weeks

8 visits (in-person)

Blinded Extension Treatment

Continuation of treatment for participants showing clinical benefit, with the option to unblind for those not benefiting

Up to 3 years

Open-label Extension

Participants may opt into continuation of treatment with Luspatercept long-term after unblinding

Up to 3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

42 days post last dose

Treatment Details

Interventions

Luspatercept
Placebo

Trial OverviewThe trial is testing Luspatercept (ACE-536) against a placebo in subjects who are anemic as part of their myelofibrosis condition while already taking JAK2 inhibitors. The study includes several phases: initial treatment, response assessment, possible unblinding for non-responders, and follow-up.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Experimental Arm: Luspatercept (ACE-536)Experimental Treatment1 Intervention

Luspatercept will be given to participants via subcutaneous injection (administered on Day 1 of each 21-day treatment cycle)

Group II: Control Arm: PlaceboPlacebo Group1 Intervention

Placebo starting dose with volume equivalent to experimental arm subcutaneous injection every 3 weeks (administered on Day 1 of each 21-day treatment cycle)

Luspatercept is already approved in United States, European Union for the following indications:

Approved in United States as Reblozyl for:

Anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions
Anemia in adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts

Approved in European Union as Reblozyl for:

Anemia in adults with transfusion-dependent beta thalassemia
Anemia in adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts

Find a Clinic Near You

Who Is Running the Clinical Trial?

Celgene

Lead Sponsor

Trials

649

Recruited

130,000+

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Luspatercept for Myelofibrosis (INDEPENDENCE Trial)