Clinical trials: B Thalassemia
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Beta Thalassemia Major
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
the aim of this study is to evaluate the effect of Spirulina compared to Amlodipine on cardiac iron overload and cardiac functions in multi-transfused children with beta thalassemia major
- Trial name
- NCT02671695
- Testing
- Spirulina, Amlodipine
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Tanta University
Thalassemia Major
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT).
- Trial name
- NCT04009525
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- China
- Volunteers
- 823 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
This study aims to investigate the possible efficacy and safety of L_Carnitine and Sildenafil on patient with Beta thalassemia complicated with increased Tricuspid Regurgitant Jet Velocity
- Trial name
- NCT05584956
- Testing
- L_carnitine, Sildenafil
- Where
- Egypt
- Volunteers
- 44 people
- Sponsor
- Tanta University
Thalassemia, Pneumococcal Infection
Pneumococcal Vaccination for Splenectomised Thalassemia Major Patients in Indonesia
Splenectomized thalassemia major subjects were provided with PCV pneumococcal vaccine (Prevenar 13®) at the start of the trial, following which they were randomly assigned to 2 groups (zinc and placebo group).
- Trial name
- NCT03095326
- Testing
- Zinc, Sucrose, PCV Vaccine
- Where
- Indonesia
- Volunteers
- 56 people
- Sponsor
- Fakultas Kedokteran Universitas Indonesia
Beta-Thalassemia
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells.
- Trial name
- NCT06364774
- Testing
- ALS20
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Thalassemia Intermedia, Thalassemia Major
Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a…
- Conditions
- Thalassemia Intermedia, Thalassemia Major, Growth Delay, Puberty, Delayed
- Trial name
- NCT06931912
- Testing
- MENBS clinical interventions
- Where
- China
- Volunteers
- 385 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Beta-Thalassemia, Bone Marrow Transplantation
Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients
This is prospective randomized, double blind study designed to evaluate the use of zoledronic acid in the prevention prevention of bone loss post allogenic BMT done for beta-thalassemia major patients.
- Trial name
- NCT01016093
- Testing
- Zoledronic acid
- Where
- Iran
- Volunteers
- 50 people
- Sponsor
- Tehran University of Medical Sciences
Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.
- Conditions
- Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia
- Trial name
- NCT02986698
- Testing
- in utero hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, San Francisco
Beta-Thalassemia
Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
OBJECTIVES: I. Determine whether arginine butyrate with or without epoetin alfa can stimulate gamma-globin chain production to a degree that decreases anemia and results in hematologic improvement in patients with thalassemia…
- Trial name
- NCT00006136
- Testing
- arginine butyrate, epoetin alfa
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 15 people
- Sponsor
- Boston University
Iron Overload, Thalassemia Major
"Iron Overload and Endocrinological Diseases"
Patients with hemochromatosis or Thalassemia develop progressive tissue and organs damages secondary to iron overload. Iron overload can result both from transfusional hemosiderosis and excess gastrointestinal iron absorption.
- Conditions
- Iron Overload, Thalassemia Major, Thalassemia Intermedia, Hemochromatosis
- Trial name
- NCT06137079
- Where
- Italy
- Volunteers
- 62 people
- Sponsor
- Azienda Ospedaliero-Universitaria di Modena
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT02179359
- Testing
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Beta-thalassemia Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
The purpose of this research study is to study the safety of increasing doses of FBS0701, and to see how quickly the study medication is absorbed and how quickly it disappears from the bloodstream.
- Trial name
- NCT01039636
- Testing
- FBS0701
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 20 people
- Sponsor
- FerroKin BioSciences, Inc.
Beta-Thalassemia
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major
Thalassemia is considered the most common genetic disorder worldwide, occurring with high frequency in Mediterranean areas, the Middle East, Southeast Asia, and the Pacific Islands.
- Trial name
- NCT01206075
- Testing
- Mozobil
- Where
- Greece
- Volunteers
- 20 people
- Sponsor
- University of Washington
Transfusion-dependent Beta-Thalassemia
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT06219239
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Anemia
A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India
- Trial name
- NCT05891249
- Testing
- Luspatercept
- Where
- 9 sites in India
- Volunteers
- 85 people
- Sponsor
- Bristol-Myers Squibb
Iron Overload, Oxidative Stress
Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
The aim of the present study is evaluating the strength of combination therapy of hydroxy urea, omega 3, nigella sativa and honey on antioxidant-oxidant status (OXIDATIVE STRESS) in response to reactive oxygen species production…
- Conditions
- Iron Overload, Oxidative Stress, Thalassemia Major
- Trial name
- NCT04292314
- Testing
- Omega 3, Nigella Sativa Oil, Hydroxyurea
- Where
- 4 sites in Egypt, Saudi Arabia
- Volunteers
- 350 people
- Sponsor
- Beni-Suef University