Clinical trials: Transfusion-dependent Thalassemia
Erythrocyte Transfusion, Beta-Thalassemia
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require…
- Trial name
- BELIEVE (NCT02604433)
- Testing
- Luspatercept
- Where
- 76 sites in United States, Australia and 13 more
- Volunteers
- 336 people
- Sponsor
- Celgene
Transfusion-dependent β-thalassemia Patients
Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
β-thalassemia is one of the most common inherited hemoglobinopathies worldwide and a major public health issue that severely impacts birth quality, human health, and social progress.
- Trial name
- NCT07338344
- Testing
- Luspatercept combined with low-dose thalidomide
- Where
- 8 sites in China
- Volunteers
- 78 people
- Sponsor
- Rongrong Liu
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
Thalassemia
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT).
- Trial name
- NCT03692052
- Testing
- AG-348
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 20 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pulmonary Artery Pressure, Thalassemia
Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia
The goal of this clinical trial is to learn whether L-glutamine can help lower pulmonary artery pressure in adults with non-transfusion-dependent thalassemia (NTDT).
- Conditions
- Pulmonary Artery Pressure, Thalassemia, Non-transfusion Dependent Thalassemia
- Trial name
- NCT07210450
- Testing
- L-glutamine
- Where
- Iran
- Volunteers
- 8 people
- Sponsor
- Mazandaran University of Medical Sciences
Transfusion Dependent Beta-Thalassaemia
A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.
This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Trial name
- NCT05752123
- Testing
- ET-01
- Where
- China
- Volunteers
- 2 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Thalassemia, Hepatitis C
Sofosbuvir/Ledipasvir for Hepatitis C Genotype 1-6 in Patients With Transfusion-Dependent Thalassemia: An Open Label Trial
Sustained Viral Response following 12-week therapy (SVR 12) with sofosbuvir/ledipasvir in transfusion-dependent patients with HCV genotype 1-6 Secondary Objective(s): Assessment of transfusion requirements Adverse events Efficacy…
- Trial name
- NCT03032666
- Testing
- sofosbuvir/velpatasvir
- Where
- Lebanon
- Volunteers
- 7 people
- Sponsor
- Ala'a Sharara
Anemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in 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…
- Trial name
- NCT05664737
- Testing
- Luspatercept
- Where
- 31 sites in China, Greece and 8 more
- Volunteers
- 189 people
- Sponsor
- Bristol-Myers Squibb
Transfusion Dependent Beta-Thalassaemia
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia.
- Trial name
- ET-01 (NCT04925206)
- Testing
- ET-01
- Where
- 4 sites in China
- Volunteers
- 8 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Β-thalassemia
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia.
- Trial name
- NCT06655662
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- 3 sites in China
- Volunteers
- 8 people
- Sponsor
- Shenzhen Hemogen
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Non-transfusion Dependent Thalassemia
Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia
Assessed the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks of treatment.
- Trial name
- THETIS (NCT01709838)
- Testing
- deferasirox
- Where
- 11 sites in China, Greece and 6 more
- Volunteers
- 134 people
- Sponsor
- Novartis Pharmaceuticals
β-thalassemia, Ineffective Erythropoiesis
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment…
- Conditions
- β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
- Trial name
- TRANSCEND (NCT03802201)
- Testing
- PTG-300
- Where
- 33 sites in United States, Greece and 7 more
- Volunteers
- 63 people
- Sponsor
- Protagonist Therapeutics, Inc.