Beta-Thalassemia Clinical Trial
Official title:
A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to <18 years with two dose escalation cohorts, followed by a dose expansion cohort. NTD Part A will be conducted in the same age group participants as TD Part A with dose confirmation and expansion phase. After Part A TD participants have completed at least one year of treatment, all available safety data from Part A adolescent participants will be evaluated before initiating TD and NTD Part B in the age group from 6 to <12 years old. Part B will consist of two dose escalation cohorts for TD and two dose escalation cohorts for NTD. Upon completion of the Treatment Period, participants of any cohort who are benefiting from the study treatment, will be offered the opportunity to continue luspatercept treatment in the Long-term Treatment Period for up to 5 years from their first dose. Participants who discontinue study treatment at any time will continue in the Posttreatment Follow-up Period for at least 5 years from their first dose of luspatercept, or 3 years from their last dose, whichever occurs later, or until they withdraw consent/assent, are lost to follow-up, or the End of Trial, whichever occurs first.
n/a
| 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| Withdrawn |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 | |
| Recruiting |
NCT05567458 -
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
|
Phase 2 | |
| 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 | |
| Terminated |
NCT03381833 -
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent 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 | |
| Completed |
NCT02268409 -
ACE-536 Extension Study - Beta Thalassemia
|
Phase 2 | |
| 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 |