Study to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia
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|ClinicalTrials.gov Identifier: NCT01749540|
Recruitment Status : Completed
First Posted : December 13, 2012
Last Update Posted : December 14, 2016
|Condition or disease||Intervention/treatment||Phase|
|B-Thalassemia||Drug: ACE-536||Phase 2|
To evaluate the proportion of β-thalassemia patients who have an erythroid response, defined as:
- a hemoglobin increase of ≥ 1.5 g/dL from baseline for ≥ 14 days (in the absence of red blood cell [RBC] transfusions) in non-transfusion dependent patients, or
- ≥ 20% reduction in RBC transfusion burden compared to pretreatment in transfusion dependent patients.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||64 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients With Beta-Thalassemia Intermedia|
|Study Start Date :||February 2013|
|Actual Primary Completion Date :||November 2015|
|Actual Study Completion Date :||November 2015|
Experimental: ACE 536
ACE-536 - 1 of 7 possible dose levels.
Subjects receive ACE-536 administered subcutaneously (SC) every 3 weeks for up to 5 cycles.
Other Name: luspatercept
- Proportion of patients who have an erythroid response. [ Time Frame: Assessed at approximately 24 weeks from patient screening. ]Proportion of patients who have an erythroid response, defined as a 1) a hemoglobin increase of ≥ 1.5 g/dL from baseline for ≥ 14 days (in the absence of red blood cell [RBC] transfusions) in non-transfusion dependent patients, or 2) ≥ 20% reduction in RBC transfusion burden compared to pretreatment in transfusion dependent patients.
- Number of patients with adverse events. [ Time Frame: From treatment initiation to End-of-Study visit (approximately 24 weeks later). ]
- Change in hemoglobin level in non-transfusion dependent patients. [ Time Frame: Baseline to approximately 24 weeks. ]
- Changes in biomarkers of erythropoiesis, hemolysis, iron metabolism and bone metabolism. [ Time Frame: Baseline to approximately 24 weeks. ]
- ACE-536 pharmacokinetics. [ Time Frame: Measured at multiple time points over the course of treatment, from study day 1 to approximately 24 weeks. ]
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): NCT01749540
|Laiko General Hospital, Ampelokipi|
|Ospedale "A. Perriino" U.O Ematologia|
|ARNAS Garibaldi - P.O. Garibaldi Centro|
|A.O.U. Arcispedale S. Anna|
|CEMEF Medicina 2|
|A.O.U. Seconda Università degli Studi di Napoli|
|AORN A. Cardarelli|
|A.O.U. San Luigi Gonzaga|