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thalassemia Clinical Trials

A listing of thalassemia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 71 clinical trials

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia.

7 - 35 years of age Both Phase 1
h hagar El-Hadidy, lecturer

Inspiratory Muscle Training in Children With Beta Thalasemia

respiratory complications are very prevalent in children with beta thalasemia major (BTM), so choosing inspiratory muscle trianing to decrease of prevent it and to improve pulmoanry funntions is important

8 - 12 years of age Both Phase N/A

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia

The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia. The main questions it aims to answer are How efficient is SP-420 in cleaning iron from the liver? How is the safety and tolerability of ascending doses of …

18 - 100 years of age Both Phase 2

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the …

18 - 100 years of age Both Phase 3

Study of Safety & PK 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 …

6 - 17 years of age Both Phase 2

A Study to Determine the Efficacy and Safety of Luspatercept in Adult and Adolescent Participants With Alpha (α)-Thalassemia

The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.

12 - 100 years of age Both Phase 2

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

18 - 35 years of age Both Phase 1/2

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

2 - 11 years of age Both Phase 3
R RBCD Trial Info Cellphone

Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation

Hypothesis: Taurine, in combination with standard iron chelation therapy, is more effective than chelation therapy alone in reducing cardiac iron overload, oxidative stress and cardiac damage in β-Thalassemia. Protocol: Sixty subjects with transfusion dependent β-Thalassemia receiving deferasirox iron chelation therapy will be recruited and randomized in a 1:1 ratio to …

18 - 100 years of age Both Phase N/A
J Jingyu Zhao, MPH

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.

3 - 35 years of age Both Phase N/A

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