A Phase II Study of Luspatercept Plus Darbepoetin Alfa in Non-mutated SF3B1 Lower-risk Myelodysplastic Syndromes
Yale University
Summary
This is a single arm open-label Phase II trial of luspatercept and darbepoetin alfa in non-mutated SF3B1 , lower-risk, RBC transfusion dependent MDS participants with an endogenous erythropoietin (EPO) level \< 500 IU/L.
Description
This is a single arm open-label Phase II trial of luspatercept and darbepoetin alfa in non-mutated SF3B1 , lower-risk, RBC transfusion dependent MDS participants with an endogenous erythropoietin (EPO) level \< 500 IU/L. Each participant will be treated with luspatercept in combination with darbepoetin alfa every 21 days (three weeks). Both agents will be administered on day 1 of each 21-day (three weeks) cycle. Based on the individual participant's response to combination therapy of luspatercept and darbepoetin alfa, the doses of luspatercept and/or darbepoetin alfa can be either decreased o…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: 1. Ability to understand and the willingness to sign a written informed consent document. 2. Participant is 18 years or older at the time of signing informed consent. 3. Participant has lower-risk myelodysplastic syndrome (MDS) defined as very low, low and intermediate risk by International Prognostic Scoring System-Revised (IPSS-R) criteria (3). 4. Bone marrow biopsy within 90 days of screening demonstrated less than 5% blasts in the aspirate and/or core biopsy. If no bone marrow biopsy was done within 90 days of screening it is mandatory to repeat it at screening. Otherw…
Interventions
- DrugLuspatercept
Luspatercept will be administered every 3 weeks (21 days) at a starting dose of 1.0 mg/kg (Dose Level 0). Luspatercept will be administered to participants by the study staff at the clinical site and administration will be documented in the participant's source record. Participants must have Hgb, blood pressure and weight assessed (changes of body weight of ≤ ± 5% do not require a dose adjustment) prior to each luspatercept dose administration. Subcutaneous injections will be given in the upper arm, thigh, and/or abdomen. Volume for subcutaneous injection will be per institutional standard/guidelines.
- DrugDarbeopoetin
Darbepoetin alfa will be administered every 3 weeks (21 days), at a starting dose of 300 ug (Dose Level 0). Darbepoetin alfa will be administered to participants by the study staff at the clinical site and administration will be documented in the participant's source record. Darbepoetin alfa will be administered as a subcutaneous injection by the site staff in the upper arm, thigh, and/or abdomen per institutional standard/guidelines.
Locations (3)
- Yale UniversityNew Haven, Connecticut
- Robert H. Lurie Comprehensive Cancer Center of Northwestern UniversityChicago, Illinois
- Harold C. Simmons Comprehensive Cancer Center of UT SouthwesternDallas, Texas