What is luspatercept studying for anemia due to myelodysplastic syndromes with del5q?

Luspatercept is being studied to see if it can reduce the need for red blood cell transfusions in adults with certain types of myelodysplastic syndromes who do not respond well to previous treatments like lenalidomide. This research aims to find new options for managing anemia in this specific patient group. The study is sponsored by Associazione Qol-one and focuses on improving patient outcomes.

Who may be eligible for this myelodysplastic syndromes clinical trial?

Adults aged 18 or older with a diagnosis of myelodysplastic syndromes featuring chromosome 5q deletion, who are dependent on red blood cell transfusions and have not responded or are intolerant to prior treatments like lenalidomide, may be eligible. Both men and women can participate. Please review eligibility criteria for more details.

Is there any cost to participate in this myelodysplastic syndromes clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses to attend visits may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel costs.

Actively Recruiting

Phase 2

Age: 18Years +

All Genders

ID05924100

Study of Luspatercept to Treat Anemia in Adults with Myelodysplastic Syndromes with del5q Who Are Resistant or Intolerant to Prior Treatments and Need Red Blood Cell Transfusions

Led by Associazione Qol-one · Updated on 2024-11-18

Participants Needed

Research Sites

260 weeks

Total Duration

AI-Summary

What this Trial Is About

Myelodysplastic syndromes MDS primarily affect older adults and involve problems with blood cell production, leading to anemia, low white blood cells, and low platelets. These conditions often cause patients to depend on red blood cell transfusions and carry risks like infection and bleeding. This research evaluates luspatercept, a protein designed to help mature red blood cells, in adults with MDS who have a specific chromosome deletion del5q and who are resistant or intolerant to prior treatments like lenalidomide. The study focuses on patients with low to intermediate risk MDS and aims to assess whether luspatercept can reduce the need for transfusions. Participants will receive luspatercept injections under the skin every three weeks, starting at a dose of 1.0 mgkg, with possible increases up to 1.75 mgkg. The study has a screening period, followed by a two-year treatment phase where patients receive the drug and are closely monitored. After treatment, there is a three-year follow-up to observe longer-term effects. Researchers will track responses such as transfusion independence, hemoglobin levels, quality of life, and safety during and after treatment. Throughout the study, participants will have regular visits for assessments including blood tests, quality of life questionnaires, and monitoring for side effects. The main outcome is whether patients achieve at least eight weeks without needing red blood cell transfusions within the first 24 weeks. Additional measures include long-term transfusion independence, duration of response, reduction in transfusions, changes in iron levels, and overall safety over five years. The total participation lasts up to five years, ensuring comprehensive evaluation of luspatercepts effects.

CONDITIONS

Brief Title

Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD

Who Can Participate

Age: 18Years +

All Genders

Eligibility Criteria

You may qualify if you...

Understand and voluntarily sign informed consent before any study procedures
Documented diagnosis of myelodysplastic syndromes with del5q per 2018 WHO classification
Very low, low, or intermediate risk disease by IPSS-R with less than 5% bone marrow blasts and peripheral blood WBC count under 13,000/µL
Refractory or intolerant to, or ineligible for, prior erythropoiesis-stimulating agents (ESAs)
Discontinuation of ESAs or G-CSF at least 4 weeks before screening
Refractory/resistant/intolerant to prior lenalidomide or ineligible with specific blood count criteria, with lenalidomide stopped at least 4 weeks before screening
Requires red blood cell transfusions averaging at least 2 units per 8 weeks for a minimum of 16 weeks before enrollment
Hemoglobin level of 10.0 g/dL or less at transfusion times
No continuous 56-day period free of transfusions during the 16 weeks before screening
ECOG performance status of 0, 1, or 2
Females of childbearing potential must have negative pregnancy tests and agree to effective contraception during and after study
Male subjects must agree to use condoms during and after the study
Willing and able to follow study visit schedule and protocol requirements

You will not qualify if you...

Presence of P53 mutation at screening
Prior therapy with disease-modifying agents for MDS (hypomethylating agents), except one dose allowed with 5 weeks washout
Previous treatment with luspatercept or sotatercept
Secondary MDS caused by chemical injury or prior chemotherapy/radiation
Clinically significant anemia due to iron, vitamin B12, folate deficiency, autoimmune/hereditary hemolytic anemia, or gastrointestinal bleeding
Prior stem cell transplant
Known diagnosis of acute myeloid leukemia
Use of anticancer chemotherapy, corticosteroids (unless stable dose), iron-chelating agents (unless stable dose), other hematopoietic growth factors, or investigational drugs within 5 weeks before study entry
Uncontrolled hypertension with repeated diastolic pressure ≥100 mmHg
Severe kidney impairment (eGFR or creatinine clearance <40 mL/min)
Liver enzymes ≥3 times the upper limit of normal or total bilirubin ≥2 times upper limit unless due to ineffective erythropoiesis or Gilbert Syndrome
History of other malignancies unless disease-free for 5 years, except certain skin and cervical/breast/prostate carcinomas

Research Team

Esther N Oliva, MD

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Study of Luspatercept to Treat Anemia in Adults with Myelodysplastic Syndromes with del5q Who Are Resistant or Intolerant to Prior Treatments and Need Red Blood Cell Transfusions

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria