Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require Transfusions
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity of Leipzig
About this trial
Anemia in patients with very low, low or intermediate risk myelodysplastic syndromes (MDS), that are non-transfusion dependent
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Diagnosis of myelodysplastic syndrome (MDS) according to WHO classification
Very low-, low-, or intermediate-risk disease MDS with up to 3.5 according to revised International Prognostic Scoring System (IPSS-R)
Less than 5% blasts in bone marrow
Peripheral blood white blood cell (WBC) count < 13,000/μL
Disqualifiers
Patient does not accept bone marrow sampling during screening and during treatment
Patient does not accept regular peripheral blood sampling for screening and during treatment.
Patient does not accept subcutaneous application of LUS every three weeks
Prior treatment for anemia associated with MDS (i.e. ESA, luspatercept), except previously treated with G-CSF/granulocyte-macrophage colony-stimulating factor (GM-CSF), both agents must be discontinued at least 4 weeks before registration
Trial design
Design model
Single group
Treatments tested in this trial
Luspatercept Injection
DrugAll formally included patients will receive 1.75 mg/kg luspatercept administered subcutaneously every three weeks (on day 1 of each 21-day cycle) for a duration of 24 weeks. Responders at the response assessment (according to HI-E) in week 25 will be further treated with 1.75 mg/kg luspatercept until loss of response for an expected maximum of 18 months.
Treatment groups
213 Participants
are divided into 1 treatment groupGroup A: LuspaterceptOther 1 intervention
Trial outcomes
Primary outcomes
1
Erythroid response (HI-E)
To evaluate the proportion of patients who have an erythroid response (HI-E) according to the modified IWG 2018 criteria separately for four different clinical situations (cohorts) distinguished by two factors: Serum erythropoietin (sEPO) level AND Ring sideroblast (RS) status
Time frame
At the end of cycle 8 (each cycle is 21 days)Secondary outcomes
1
HI-E response (erythroid response) duration
To evaluate HI-E response from the first day of response until loss of response.
Time frame
From the date of treatment start until date of documented loss of response, assessed up to 18 months.]2
Time to HI-E (erythroid response)
To evaluate the time between start of treatment and first day of response.
Time frame
From the date of treatment start until first day of response, assessed up to end of cycle 8 (each cycle is 21 days)3
Neutrophil (HI-N) responses
Neutrophil (HI-N) responses according to IWG 2018 criteria
Time frame
At the end of cycle 8 (each cycle is 21 days)4
Platelet (HI-P) responses
Platelet (HI-P) responses according to IWG 2018 criteria
Time frame
At the end of cycle 8 (each cycle is 21 days)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
University of Leipzig
Lead sponsor
Celgene Corporation
Collaborator
Source ClinicalTrials.gov