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Luspatercept for Myelodysplastic Syndrome

Phase 2

Recruiting

Led By Guillermo Garcia-Manero, M D

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

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

Must have

Cohorts #1 and #2: Diagnosis of MDS according to WHO 2023 criteria and with low or int-1 risk by IPSS or a score of ≤ 3.5 by IPSS-R

Cohort #2: Patient defined as transfusion dependent by documentation of receiving at least 2 units of packed red blood cells (PRBCs) for a hemoglobin of less than 8.0 g/dL during an 8-week period prior to study enrollment

Must not have

Active infection not adequately responding to appropriate antibiotics

Prior treatment with luspatercept or sotarcetept

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial will test if luspatercept can reduce the need for blood transfusions in people with LR-MDS.

Who is the study for?

Adults diagnosed with low-risk Myelodysplastic Syndromes (MDS) who need fewer blood transfusions. Cohort #1 includes those not needing transfusions for a hemoglobin level under 8.0 g/dL in the past 8 weeks, while Cohort #2 includes those dependent on at least two units of red blood cells within the same period. Participants must have adequate liver and kidney function, an ECOG performance status of ≤2, and agree to use contraception if they can have children.

What is being tested?

The study is testing Luspatercept's effectiveness in reducing the need for blood transfusions in patients with lower risk MDS. It's an open-label pilot trial where all participants receive Luspatercept; there are no comparison groups or placebos involved.

What are the potential side effects?

While specific side effects for Luspatercept aren't listed here, similar medications often cause fatigue, muscle pain, dizziness, high blood pressure, and potential allergic reactions. The exact side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with MDS and my risk level is low to intermediate-1.

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I needed at least 2 blood transfusions due to low hemoglobin in the last 8 weeks.

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My liver functions are within the required range.

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My kidneys work well enough (creatinine clearance ≥40mL/min), and I don't have end-stage kidney disease.

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I can take care of myself and perform daily activities.

Exclusion Criteria

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

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I have an infection that isn't getting better with antibiotics.

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I have been treated with luspatercept or sotatercept before.

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I am willing to use effective birth control during the study.

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I am currently pregnant or breastfeeding.

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I am a woman who can have children and have not had a negative pregnancy test.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

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

Treatment Details

Side effects data

From 2021 Phase 3 trial • 336 Patients • NCT02604433

42%

Upper respiratory tract infection

35%

Headache

32%

Back pain

23%

Arthralgia

22%

Bone pain

22%

Cough

21%

Pyrexia

18%

Oropharyngeal pain

18%

Diarrhoea

17%

Fatigue

16%

Pharyngitis

15%

Pain in extremity

13%

Abdominal pain

13%

Nausea

13%

Vomiting

13%

Myalgia

13%

Dizziness

12%

Asthenia

11%

Abdominal pain upper

11%

Influenza

10%

Hypertension

Dyspepsia

Influenza like illness

Musculoskeletal pain

Nasal congestion

Nasopharyngitis

Gastroenteritis

Urticaria

Toothache

Tonsillitis

Hyperuricaemia

Pain

Urinary tract infection

Viral upper respiratory tract infection

Neck pain

Osteoporosis

Constipation

Injection site pain

Transfusion reaction

Alanine aminotransferase increased

Spinal pain

Lethargy

Menstruation irregular

Liver iron concentration increased

Fall

Musculoskeletal chest pain

Anaemia

Septic shock

Extramedullary haemopoiesis

Deep vein thrombosis

Transient ischaemic attack

Cerebrovascular accident

Cholangitis

Cholecystitis acute

Cellulitis

Pneumonia

100%

80%

60%

40%

20%

Study treatment Arm

Luspatercept + BSC

Placebo + BSC

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment1 Intervention

Participants with lower risk MDS that are transfusion dependent (TD). Participants will be asked to come to the study clinic weekly for the first 3 weeks and then 1 time every 3 weeks after that to have tests and procedures (such as physical exams and blood draws). Luspatercept will be administered once every 3 weeks.

Group II: Cohort 1Experimental Treatment1 Intervention

Participants with lower risk MDS patients who have symptomatic anemia that are transfusion independent (TI). Participants will be asked to come to the study clinic weekly for the first 3 weeks and then 1 time every 3 weeks after that to have tests and procedures (such as physical exams and blood draws). Luspatercept will be administered once every 3 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Luspatercept

2018

Completed Phase 3

~1240

0 / 10Eligibility criteria met