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Protein Therapeutics

Luspatercept for Myelofibrosis (INDEPENDENCE Trial)

Phase 3

Waitlist Available

Research Sponsored by Celgene

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

RBC transfusions are scored in determining eligibility when given for treatment of symptomatic anemia with a pretransfusion Hgb ≤ 9.5 g/dL or asymptomatic anemia with a pretransfusion Hgb ≤ 7 g/dL.

a. Average RBC-transfusion frequency: 4 to 12 RBC units/12 weeks immediately up to randomization. There must be no interval > 6 weeks (42 days) without ≥ 1 RBC transfusion.

Must not have

Subject with a major bleeding event in the last 6 months prior to the date of randomization.

Subject with stroke, myocardial infarction, deep venous thrombosis, pulmonary or arterial embolism within 6 months immediately up to the date of randomization.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is studying how well luspatercept works in treating patients with myeloproliferative neoplasm-associated myelofibrosis and anemia who are on concomitant Janus kinase 2 inhibitor therapy and who require red blood cell count transfusions.

Who is the study for?

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.

What is being tested?

The 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.

What are the potential side effects?

While specific side effects for Luspatercept aren't listed here, similar medications can cause reactions at injection sites, tiredness, headache, muscle or joint pain. There's also a risk of more serious effects like high blood pressure or abnormal liver tests.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have received blood transfusions for low hemoglobin levels.

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I've had 4 to 12 blood transfusions in the last 3 months without going more than 6 weeks without one.

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I have been on a stable dose of JAK2 inhibitor for my condition for at least 16 weeks.

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I need regular blood transfusions, getting 4 to 12 units every 3 months without going more than 6 weeks between them.

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I am 18 years old or older.

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I can take care of myself but might not be able to do heavy physical work.

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My condition is officially diagnosed as primary or secondary myelofibrosis.

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My condition is officially diagnosed as primary or secondary myelofibrosis.

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I am a man who will not have sex or will use a condom with women who can get pregnant.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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

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My heart condition is not well-managed, and my heart's pumping ability is weak.

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I do not have any uncontrolled infections.

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My anemia is not caused by myeloproliferative neoplasm treatment.

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My blood pressure is not controlled by medication.

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I have HIV or active Hepatitis B/C.

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I have previously been treated with luspatercept or sotatercept.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Blood Transfusion

Secondary study objectives

Change in RBC transfusion burden

Change in serum ferritin from baseline

Cumulative duration of RBC-transfusion independence

+12 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

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)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ACE-536

2011

Completed Phase 1

~40

0 / 17Eligibility criteria met