A Phase 2 Study for the Treatment of Anemia with Alpha (α)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents

Conditions

Alpha (α)-thalassemia

Brief summary

Adult cohorts, Primary endpoints: - TD: Achievement of ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Weeks 13-48 compared to the 12-week interval immediately prior to the date of first dose., Adult cohorts, Primary endpoints: - NTD: Achievement of an increase from baseline of ≥ 1.0 g/dL in mean hemoglobin values over the continuous 12-week interval from Week 13 to Week 24 in the absence of RBC transfusion., Adolescent cohorts, Primary endpoints: - Dose-limiting toxicities (DLTs) defined as observance of ≥ Grade 3-related hemolytic crises or ≥ Grade 3-related event outside of the known safety profile occurring within the 21 days from their first dose of study therapy. - PK parameters - Frequency, severity, and seriousness of AEs

Related papers

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Interventions

DRUG0.9% sodium chloride injection

DRUGsolution for injection

DRUGReblozyl 75 mg powder for solution for injection

DRUGReblozyl 25 mg powder for solution for injection

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Adult cohorts, Primary endpoints: - TD: Achievement of ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Weeks 13-48 compared to the 12-week interval immediately prior to the date of first dose., Adult cohorts, Primary endpoints: - NTD: Achievement of an increase from baseline of ≥ 1.0 g/dL in mean hemoglobin values over the continuous 12-week interval from Week 13 to Week 24 in the absence of RBC transfusion., Adolescent cohorts, Primary endpoints: - Dose-limiting toxicities (DLTs) defined as observance of ≥ Grade 3-related hemolytic crises or ≥ Grade 3-related event outside of the known safety profile occurring within the 21 days from their first dose of study therapy. - PK parameters - Frequency, severity, and seriousness of AEs	—

Measure

Time frame

Adult cohorts, Primary endpoints: - TD: Achievement of ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Weeks 13-48 compared to the 12-week interval immediately prior to the date of first dose., Adult cohorts, Primary endpoints: - NTD: Achievement of an increase from baseline of ≥ 1.0 g/dL in mean hemoglobin values over the continuous 12-week interval from Week 13 to Week 24 in the absence of RBC transfusion., Adolescent cohorts, Primary endpoints: - Dose-limiting toxicities (DLTs) defined as observance of ≥ Grade 3-related hemolytic crises or ≥ Grade 3-related event outside of the known safety profile occurring within the 21 days from their first dose of study therapy. - PK parameters - Frequency, severity, and seriousness of AEs

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Countries

Greece, Italy

Outcome results

None listed