Ignite Creation Date:
2025-12-24 @ 8:03 PM
Ignite Modification Date:
2026-01-04 @ 1:57 PM
Study NCT ID:
NCT03342404
Status:
COMPLETED
Last Update Posted:
2023-12-20
First Post:
2017-10-30
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
Sponsor:
Celgene
Organization:
Study Overview
Official Title:
A Phase 2, Double-Blind, Randomized, Placebo-Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Adults With Non-Transfusion Dependent Beta (β)-Thalassemia (The BEYOND™ Study)
Status:
COMPLETED
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
BEYOND
Brief Summary:
This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia. The study is divided into the Screening Period, Double-blind Treatment Period (DBTP), Open-label Phase (OLP), and Post-Treatment Follow-up Period (PTFP).
It is planned to randomize approximately 150 subjects at a 2:1 ratio of luspatercept versus placebo.
It is planned to randomize approximately 150 subjects at a 2:1 ratio of luspatercept versus placebo.
Detailed Description:
The primary objective is:
\- To evaluate the effect of luspatercept (BMS-986346) versus placebo on anemia, as measured by mean hemoglobin concentration in the absence of transfusions over a continuous 12-week interval, from Week 13 to Week 24, compared to baseline.
The secondary objectives are:
* To evaluate the effect of luspatercept versus placebo in anemia-related symptoms in participants with β-thalassemia, as measured by non-transfusion dependent β-thalassemia-patient reported outcome (NTDT-PRO) over continuous 12-week intervals (from Weeks 13 to 24 and from Weeks 37 to 48) compared to baseline.
* To evaluate the effect of luspatercept versus placebo on functional and health-related quality of life (QoL) as measured by Medical Outcomes Study 36-Item Short Form (SF-36) and Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) questionnaires
* To evaluate the long-term effect of luspatercept versus placebo on anemia, as measured by mean hemoglobin concentration in the absence of transfusions over a continuous 12-week interval from Week 37 to Week 48, compared to baseline
* To evaluate the effect of luspatercept versus placebo on iron overload, as measured by liver iron concentration (LIC) and iron chelation therapy (ICT) daily dose
* To evaluate the effect of luspatercept versus placebo on iron overload, as measured by serum ferritin
* To evaluate the duration of erythroid response
* To evaluate the effect of luspatercept versus placebo on physical activity measured by 6-minute walk test (6MWT)
Safety and Pharmacokinetics (PK) Objectives
* To evaluate safety and tolerability of luspatercept, including immunogenicity
* To evaluate population pharmacokinetics (PK) of luspatercept in subjects with β-thalassemia
The exploratory objectives are:
* To evaluate the effect of luspatercept versus placebo on measures of extra-medullary hematopoietic (EMH) masses, bone mineral density, splenomegaly, pulmonary hypertension, and leg ulcers, when present
* To evaluate the effect of luspatercept versus placebo on the β-thalassemia severity as measured by the NTDT severity score system
* To evaluate the effect of luspatercept versus placebo on the β-thalassemia severity as measured by the Morbidity-free Survival parameters
* To examine the relationship of baseline and change in serum Growth Differentiation Factor 11 (GDF11) and other related biomarkers with response to treatment with luspatercept
* To examine the effect of luspatercept on fetal hemoglobin (HbF)
* To examine the effect of luspatercept on Health Resource Utilization (HRU)
\- To evaluate the effect of luspatercept (BMS-986346) versus placebo on anemia, as measured by mean hemoglobin concentration in the absence of transfusions over a continuous 12-week interval, from Week 13 to Week 24, compared to baseline.
The secondary objectives are:
* To evaluate the effect of luspatercept versus placebo in anemia-related symptoms in participants with β-thalassemia, as measured by non-transfusion dependent β-thalassemia-patient reported outcome (NTDT-PRO) over continuous 12-week intervals (from Weeks 13 to 24 and from Weeks 37 to 48) compared to baseline.
* To evaluate the effect of luspatercept versus placebo on functional and health-related quality of life (QoL) as measured by Medical Outcomes Study 36-Item Short Form (SF-36) and Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) questionnaires
* To evaluate the long-term effect of luspatercept versus placebo on anemia, as measured by mean hemoglobin concentration in the absence of transfusions over a continuous 12-week interval from Week 37 to Week 48, compared to baseline
* To evaluate the effect of luspatercept versus placebo on iron overload, as measured by liver iron concentration (LIC) and iron chelation therapy (ICT) daily dose
* To evaluate the effect of luspatercept versus placebo on iron overload, as measured by serum ferritin
* To evaluate the duration of erythroid response
* To evaluate the effect of luspatercept versus placebo on physical activity measured by 6-minute walk test (6MWT)
Safety and Pharmacokinetics (PK) Objectives
* To evaluate safety and tolerability of luspatercept, including immunogenicity
* To evaluate population pharmacokinetics (PK) of luspatercept in subjects with β-thalassemia
The exploratory objectives are:
* To evaluate the effect of luspatercept versus placebo on measures of extra-medullary hematopoietic (EMH) masses, bone mineral density, splenomegaly, pulmonary hypertension, and leg ulcers, when present
* To evaluate the effect of luspatercept versus placebo on the β-thalassemia severity as measured by the NTDT severity score system
* To evaluate the effect of luspatercept versus placebo on the β-thalassemia severity as measured by the Morbidity-free Survival parameters
* To examine the relationship of baseline and change in serum Growth Differentiation Factor 11 (GDF11) and other related biomarkers with response to treatment with luspatercept
* To examine the effect of luspatercept on fetal hemoglobin (HbF)
* To examine the effect of luspatercept on Health Resource Utilization (HRU)
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: