Status:
RECRUITING
A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)
Lead Sponsor:
M.D. Anderson Cancer Center
Conditions:
Myelodysplastic Syndromes
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
To learn if luspatercept is more effective in helping to reduce the number of blood transfusions needed by patients with LR-MDS.
Detailed Description
Objectives: Primary Objectives: * To assess the clinical activity and safety of luspatercept in patients with transfusion independent lower risk MDS * To assess the clinical activity and safety of l...
Eligibility Criteria
Inclusion
- Age ≥ 18 years; as MDS there is no significant experience with luspatercept in pediatric patients
- 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)
- 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.
- 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.
- MDS patients with either a platelet count of ≤100 K/uL and/or ANC of ≤1.8 K/uL
- Patient must have signed an informed consent and is willing to participate in the study.
- Adequate hepatic function with total bilirubin ≤3 x ULN, AST or ALT ≤3xULN.
- Serum creatinine clearance ≥40mL/min and no end/stage renal disease (using Cockcroft-Gault).
- ECOG performance status \</=2.
Exclusion
- Active infection not adequately responding to appropriate antibiotics.
- Prior treatment with luspatercept or sotarcetept
- Female patients who are pregnant or lactating.
- 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.
- Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening.
- 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.
- Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy (within 14 days of initiating study treatment).
Key Trial Info
Start Date :
March 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 2 2026
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT06113302
Start Date
March 1 2024
End Date
June 2 2026
Last Update
October 24 2025
Active Locations (1)
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1
M D Anderson Cancer Center
Houston, Texas, United States, 77030