Beta-thalassemia Clinical Trial
Official title:
A Phase 2, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Luspatercept (ACE-536) in Chinese Adult Subjects Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of luspatercept plus best supportive care (BSC) versus placebo plus BSC in participants who require regular red blood cell transfusions due to β-thalassemia.
Status | Recruiting |
Enrollment | 90 |
Est. completion date | July 2, 2025 |
Est. primary completion date | February 3, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Participant is willing and able to adhere to the study visit schedule (for example, not scheduled to receive hematopoietic stem cell transplantation [HSCT]) and other protocol requirements. - Participant has documented diagnosis of ß-thalassemia or Hemoglobin E/ß-thalassemia (ß-thalassemia with mutation and/or multiplication of alpha (a) globin is allowed). - Participant is regularly transfused, defined as: 6-25 RBC units in the 24 weeks prior to randomization and no transfusion-free period for >42 days during that period. - Participant has Eastern Cooperative Oncology Group (ECOG) score of 0 or 1. Exclusion Criteria: - Participant has a diagnosis of Hemoglobin S/ß-thalassemia or a-thalassemia (for example, Hemoglobin H). - Participant has active hepatitis C virus (HCV) infection as demonstrated by a positive HCVribonucleic acid (RNA) test of sufficient sensitivity, or active infectious hepatitis B virus (HBV) as demonstrated by the presence of hepatitis B surface antigen (HBsAg) and/or HBVdeoxyribonucleic acid (DNA) positive, or known positive human immunodeficiency virus (HIV). - Participant has a history of deep venous thrombosis or stroke or thromboembolic events (venous or arterial) requiring medical intervention =24 weeks prior to randomization. - Participant uses chronic anticoagulant therapy, unless the treatment stopped at least 28 days prior to randomization. Anticoagulant therapies used for prophylaxis for surgery or high-risk procedures as well as low-molecular-weight heparin for superficial venous thrombosis and chronic aspirin are allowed. |
Country | Name | City | State |
---|---|---|---|
China | Nanfang Hospital of Southern Medical University | Guangzhou | |
China | Sun Yat-sen Memorial Hospital, Sun Yat-Sen University | Guangzhou | |
China | Hainan General Hospital | Haikou | |
China | Hainan Medical College - First Affiliated Hospital | Haikou | |
China | First People's Hospital of Yunnan Province | Kunming | Yunnan |
China | Liuzhou General Hospital | Liuzhou | |
China | Maoming People's Hospital | Maoming | |
China | People's Liberation Army The 923rd Hospital | Nanning | Guangxi |
China | The First Affiliated Hospital of Guangxi Medical University | Nanning | |
China | Shenzhen Second People's Hospital | Shenzhen | Guangdong |
Lead Sponsor | Collaborator |
---|---|
Bristol-Myers Squibb |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Proportion of participants with = 33% reduction from baseline in red blood cell (RBC) transfusion burden over any consecutive 24 weeks | Up to 48 weeks | ||
Secondary | Proportion of participants with = 33% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks | Up to 48 weeks | ||
Secondary | Proportion of participants with = 50% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks | Up to 48 weeks | ||
Secondary | Proportion of participants with = 50% reduction from baseline in RBC transfusion burden over any consecutive 24 weeks | Up to 48 weeks | ||
Secondary | Proportion of participants with = 33% reduction from baseline in RBC transfusion burden over Weeks 13-24 | Weeks 13 to 24 | ||
Secondary | Proportion of participants with = 33% reduction from baseline in RBC transfusion burden over Weeks 37-48 | Weeks 37 to 48 | ||
Secondary | Proportion of participants with = 33% reduction from baseline in RBC transfusion burden over Weeks 1-24 | Weeks 1 to 24 | ||
Secondary | Proportion of participants with = 33% reduction from baseline in RBC transfusion burden over Weeks 25-48 | Weeks 25 to 48 | ||
Secondary | Proportion of participants with = 50% reduction from baseline in RBC transfusion burden over Weeks 13-24 | Weeks 13 to 24 | ||
Secondary | Proportion of participants with = 50% reduction from baseline in RBC transfusion burden over Weeks 37-48 | Weeks 37 to 48 | ||
Secondary | Proportion of participants with = 50% reduction from baseline in RBC transfusion burden over Weeks 1-24 | Weeks 1 to 24 | ||
Secondary | Proportion of participants with = 50% reduction from baseline in RBC transfusion burden over Weeks 25-48 | Weeks 25 to 48 | ||
Secondary | Mean change from baseline in total RBC units transfused in 24 weeks within the first 48-week treatment period | Baseline up to Week 48 | ||
Secondary | Change from baseline in total RBC units transfused over Weeks 1-24 | Baseline up to Week 24 | ||
Secondary | Change from baseline in total RBC units transfused over Weeks 25-48 | Weeks 25 to 48 | ||
Secondary | Mean change from baseline in serum ferritin | Baseline, Weeks 37 to 48 | ||
Secondary | Change from baseline in Liver Iron Concentration (LIC) (mg/g dw) by magnetic resonance imaging (MRI) | Up to 96 weeks | ||
Secondary | Change from baseline in myocardial iron by T2-star (T2*) MRI | Up to 96 weeks | ||
Secondary | Change from baseline in mean daily dose of iron chelation therapy (ICT) | Baseline, Weeks 37 to 48 | ||
Secondary | Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL | Up to 48 weeks | ||
Secondary | Change from baseline in self-reported HRQoL assessed by SF-36 | Up to 48 weeks | ||
Secondary | Proportion of participants who are transfusion independent for any consecutive =8 weeks during treatment | Up to 48 weeks | ||
Secondary | Proportion of participants who are transfusion independent for any consecutive =12 weeks during treatment | Up to 48 weeks | ||
Secondary | Duration of reduction in transfusion burden | Up to 48 weeks | ||
Secondary | Duration of RBC transfusion independence (TI) | Up to 48 weeks | ||
Secondary | Time to response | Up to 48 weeks | ||
Secondary | Mean number of transfusion events in 24 weeks within the first 48-week treatment period | Baseline up to Week 48 | ||
Secondary | Number of participants with Adverse Events (AEs) | Up to 48 weeks | ||
Secondary | Frequency of Antidrug antibodies (ADA) | Up to 2 years | ||
Secondary | Maximum plasma concentration (Cmax) | Up to 2 years | ||
Secondary | Area under the curve (AUC) | Up to 2 years | ||
Secondary | Change in spleen volume | Up to 96 weeks |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00069862 -
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
|
Phase 1/Phase 2 | |
Completed |
NCT00733811 -
Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
|
Phase 4 | |
Completed |
NCT05506358 -
Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada
|
N/A | |
Withdrawn |
NCT04938635 -
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
|
Phase 2 | |
Active, not recruiting |
NCT03655678 -
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia
|
Phase 2/Phase 3 | |
Completed |
NCT06239389 -
Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
|
Phase 2 | |
Recruiting |
NCT05635266 -
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
|
||
Completed |
NCT03271541 -
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia
|
Phase 2 | |
Terminated |
NCT02274233 -
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
|
Phase 1 | |
Completed |
NCT01206075 -
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major
|
N/A | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Completed |
NCT03961828 -
Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus
|
Phase 4 | |
Recruiting |
NCT06065189 -
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
|
Early Phase 1 | |
Recruiting |
NCT04143724 -
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
|
Phase 2 | |
Terminated |
NCT03381833 -
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
|
Phase 2 | |
Completed |
NCT02268409 -
ACE-536 Extension Study - Beta Thalassemia
|
Phase 2 | |
Not yet recruiting |
NCT01996683 -
Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
|
N/A | |
Active, not recruiting |
NCT01016093 -
Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients
|
Phase 2/Phase 3 | |
Completed |
NCT01039636 -
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
|
Phase 1 | |
Withdrawn |
NCT01927913 -
Treatment of Iron Overload Requiring Chelation Therapy
|
Phase 2 |