Using luspatercept to reduce blood transfusions in patients with lower risk myelodysplastic syndromes
A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)
This study is testing if the drug luspatercept can help people with lower risk myelodysplastic syndromes need fewer blood transfusions and improve their blood counts.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | M.D. Anderson Cancer Center Academic / other |
| Drugs / interventions | chemotherapy, immunotherapy |
| Locations | 1 site (Houston, Texas) |
| Trial ID | NCT06113302 on ClinicalTrials.gov |
What this trial studies
This pilot study evaluates the effectiveness and safety of luspatercept in patients with lower risk myelodysplastic syndromes (MDS), focusing on both transfusion-dependent and transfusion-independent patients. The study aims to assess the drug's impact on reducing the need for blood transfusions, improving hematopoiesis, and monitoring overall survival and response duration. Participants will be closely monitored for changes in blood counts and transfusion-free survival periods. The study is designed to gather valuable data on the clinical activity of luspatercept in this patient population.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older diagnosed with lower risk MDS who are either transfusion dependent or transfusion independent.
Not a fit: Patients with higher risk MDS or those who do not meet the specific eligibility criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the need for blood transfusions in patients with lower risk MDS, improving their quality of life.
How similar studies have performed: While this approach is being explored in this specific context, similar studies have shown promise in using luspatercept for related conditions, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Age ≥ 18 years; as MDS there is no significant experience with luspatercept in pediatric patients 2. Cohorts #1 and #2: Diagnosis of MDS according to WHO 2023 criteria (5) and with low or int-1 risk by IPSS or a score of ≤ 3.5 by IPSS-R.(3, 22) 3. 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. 4. Cohort #1: Patients with symptomatic anemia that are transfusion independent defined as not requiring a transfusion for a hemoglobin of less than 8.0 g/dL during an 8-week period prior to study enrollment. 5. MDS patients with either a platelet count of ≤100 K/uL and/or ANC of ≤1.8 K/uL 6. Patient must have signed an informed consent and is willing to participate in the study. 7. Adequate hepatic function with total bilirubin ≤3 x ULN, AST or ALT ≤3xULN. 8. Serum creatinine clearance ≥40mL/min and no end/stage renal disease (using Cockcroft-Gault). 9. ECOG performance status \</=2. Exclusion Criteria: 1. Active infection not adequately responding to appropriate antibiotics. 2. Prior treatment with luspatercept or sotarcetept 3. Female patients who are pregnant or lactating. 4. Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives \[birth control pills\], contraceptive injections, intrauterine devices \[IUD\], double-barrier method \[spermicidal jelly or foam with condoms or diaphragm\], contraceptive patch, or surgical sterilization) throughout the study. Reproductive potential is defined as no previous surgical sterilization or females that are not post-menopausal for 12 months. 5. Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening. 6. History of an active malignancy within the past 2 years prior to study entry, with the exception of: a. Adequately treated in situ carcinoma of the cervix uteri b. Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin or any other malignancy with a life expectancy of more than 2 years. 7. Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy (within 14 days of initiating study treatment).
Where this trial is running
Houston, Texas
- M D Anderson Cancer Center — Houston, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Guillermo Garcia-Manero, M D — M.D. Anderson Cancer Center
- Study coordinator: Guillermo Garcia-Manero, M D
- Email: ggarciam@mdanderson.org
- Phone: (713) 745-3428
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.