A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)

Launched by M.D. ANDERSON CANCER CENTER · Oct 27, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called luspatercept to see if it can help patients with lower risk Myelodysplastic Syndromes (MDS) need fewer blood transfusions. MDS is a condition where the bone marrow doesn't produce enough healthy blood cells, and some patients may rely on regular blood transfusions to stay healthy. The researchers want to find out if luspatercept can improve patients' blood counts and reduce their need for these transfusions.

To participate in this study, patients need to be at least 18 years old and diagnosed with MDS. They can either be dependent on transfusions, meaning they need regular blood to maintain a healthy level, or be transfusion-independent but still experiencing symptoms of anemia (low red blood cells). Participants will receive the treatment and will be monitored for its effects. It’s important to note that the study is open to both men and women, but there are specific health conditions that may exclude someone from joining, such as active infections or recent cancer treatments. If you or someone you know is considering joining the trial, they will need to discuss their medical history with the study team to see if they qualify.

Gender

ALL

Eligibility 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).