RecruitingPhase 2

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

United States40 participantsStarted 2024-03-01

Plain-language summary

To learn if luspatercept is more effective in helping to reduce the number of blood transfusions needed by patients with LR-MDS.

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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).

✕. 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).

Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

Timeframe: through study completion; an average 1 year.

NCT IDNCT06113302

SponsorM.D. Anderson Cancer Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-06-02

Guillermo Garcia-Manero, M D

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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).

✕. 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).