Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require Transfusions (LENNON)
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|ClinicalTrials.gov Identifier: NCT05384691|
Recruitment Status : Recruiting
First Posted : May 20, 2022
Last Update Posted : January 27, 2023
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndromes Anemia||Drug: Luspatercept Injection||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||213 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II, Open-label, Single Arm Study to Evaluate the Efficacy of Luspatercept in Erythropoiesis-stimulating Agent Naive Lower-risk MDS Patients With or Without Ring Sideroblasts Who do Not Require RBC Transfusions|
|Actual Study Start Date :||September 27, 2022|
|Estimated Primary Completion Date :||July 31, 2026|
|Estimated Study Completion Date :||July 31, 2027|
Single-arm design: All patients are treated with 1.75 mg Luspatercept per kg body weight subcutaneously on day 1 of each 21 day cycle for up to 24 weeks and in case of response for up to 1.5 years.
Drug: Luspatercept Injection
All 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.
Other Name: LUS
- Erythroid response (HI-E) [ Time Frame: At the end of cycle 8 (each cycle is 21 days) ]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
- HI-E response (erythroid response) duration [ Time Frame: From the date of treatment start until date of documented loss of response, assessed up to 18 months.] ]To evaluate HI-E response from the first day of response until loss of response.
- Time to HI-E (erythroid 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) ]To evaluate the time between start of treatment and first day of response.
- Neutrophil (HI-N) responses [ Time Frame: At the end of cycle 8 (each cycle is 21 days) ]Neutrophil (HI-N) responses according to IWG 2018 criteria
- Platelet (HI-P) responses [ Time Frame: At the end of cycle 8 (each cycle is 21 days) ]Platelet (HI-P) responses according to IWG 2018 criteria
- Safety of luspatercept (toxicities and adverse events) [ Time Frame: From the date of treatment start until the end of study, assessed up to 48 months ]Assessments will include characterization of toxicities; characterization of AEs including type, incidence, severity, seriousness, and relationship to treatment
- Impact of treatment assessed by using the validated European Organisation for Research and Treatment of Cancer Core Quality of Life questionnaire (EORTC QLQ-C30) [ Time Frame: From the date of treatment start until the end of study, assessed up to 48 months. ]
To assess patient-reported quality of life during luspatercept treatment: 30 questions assessing the quality of life of oncology patients across 10 subscales will be analyzed. All subscales have a score range from 0 to 100 points.
Function subscales: a higher score represents a higher quality of life. Symptoms subscales: higher score represents higher level of symptoms/problems, i.e., represents lower quality of life.
- Impact of luspatercept on quality of life by using the validated Quality of Life in Myelodysplasia Scale (QUALMS) [ Time Frame: From the date of treatment start until the end of study, assessed up to 48 months ]
QoL assessment using the QUALMS questionnaire up to end of treatment:
38-item assessment tool for patients with Myelodysplastic Syndromes (MDS) QUALMS scores ranged from 24 to 99, with higher scores for better outcome
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05384691
|Contact: Anne Sophie Kubasch, Dr.||+49 341 email@example.com|
|Contact: Uwe Platzbecker, Prof. Dr.||firstname.lastname@example.org|
|Principal Investigator:||Anne Sophie Kubasch, Dr.||University Leipzig|