Clinical trials: Thalassaemia
Beta Thalassemia Major
Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism
Hematopoietic stem cell transplantation is currently the only way to cure thalassemia, one of its main obstacles is the rejection after transplantation, chimerism continued to decline, which eventually lead to transplant failure.
- Trial name
- NCT03101423
- Testing
- Interleukin-2, Donor Regulatory T-Lymphocytes
- Where
- China
- Volunteers
- 30 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Thalassemia, Iron Overload
Renal Function Among Thalassemia Patients Treated by a Oral Chelator Deferasirox
Thalassemia Major patients developed Iron Overload due to blood transfusions and intestinal iron absorption. Renal function caused by Iron overload was studied in a previous study and shows principally tubular disfunction.
- Trial name
- NCT01905774
- Testing
- Deferasirox
- Where
- Israel
- Volunteers
- 36 people
- Sponsor
- HaEmek Medical Center, Israel
Hematologic Malignancies, Nonmalignant Diseases
Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of melphalan in their bodies and if drug…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609827
- Testing
- Melphalan
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Thalassemia Major With Severe Transfusional Iron Overload
Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.
- Trial name
- NCT01709032
- Testing
- Deferasirox and deferiprone
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital of Philadelphia
Beta-thalassemia
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants 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…
- Trial name
- NCT05567458
- Testing
- Luspatercept
- Where
- 10 sites in China
- Volunteers
- 94 people
- Sponsor
- Bristol-Myers Squibb
Thalassemia, Iron Overload
Oral Nifedipine to Treat Iron Overload
This study will determine if nifedipine, a medication used to treat high blood pressure, can help treat iron overload, a condition in which the body contains too much iron.
- Conditions
- Thalassemia, Iron Overload, Hemochromatosis
- Trial name
- NCT00712738
- Testing
- Nifedipine, Phlebotomy, Urinalysis
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
Open-Label Extension Study to Pioneer Study 6058-SCD-101
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder…
- Trial name
- NCT07401823
- Testing
- Pociredir
- Where
- 8 sites in United States
- Volunteers
- 1 people
- Sponsor
- Fulcrum Therapeutics
Thalassemia Major
Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase
Luspatercept represents the first and only erythroid maturation agent (EMA) approved by the European Commission (EC) and the Food and Drug Administration (FDA) capable of enhancing advanced erythrocyte maturation.
- Trial name
- LUSPA001 (NCT06913634)
- Testing
- Luspatercept (ACE-536)
- Where
- Italy
- Volunteers
- 140 people
- Sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
Thalassemia, Sickle Cell Disease
Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations
The investigators' primary objective is to study prevalences of myocardial iron overload, defined as a cardiac T2*< 20 ms, in 3 populations of multiply transfused patients, affected with thalassemia, sickle cell disease, and…
- Conditions
- Thalassemia, Sickle Cell Disease, Myelodysplasia
- Trial name
- SUFEMYO (NCT01911871)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 110 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Thalassemia Major
Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients.
- Trial name
- NCT03992001
- Testing
- Blood component A, Blood component B
- Where
- Italy
- Volunteers
- 55 people
- Sponsor
- Università degli Studi di Ferrara
Sickle Cell Disease
A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
- Trial name
- NCT03401112
- Testing
- IMR-687
- Where
- 13 sites in United States, United Kingdom
- Volunteers
- 100 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
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
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
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
Sickle Cell Disease, Sickle-Beta Zero Thalassemia
Dose-Escalation Study of SCD-101 in Sickle Cell Disease
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
- Trial name
- NCT02380079
- Testing
- SCD-101
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Invenux, LLC
Beta Thalassemia Intermedia
Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand
Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells which carries oxygen throughout the body.
- Trial name
- ST20-P2T (NCT01609595)
- Testing
- sodium 2,2 dimethylbutyrate
- Where
- Thailand
- Volunteers
- 10 people
- Sponsor
- Boston University
Thalassemia
Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia Intermedia
The definition of Thalassemia Intermedia is not generally accepted and the specific clinical and laboratory characteristics varies between patients.
- Trial name
- NCT01443312
- Testing
- Laboratory analysis.
- Where
- Israel
- Volunteers
- 28 people
- Sponsor
- HaEmek Medical Center, Israel
Beta Thalassemia Major, Congenital Anemias
Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
Betathalassemia major is a disease of the blood and bone marrow. You were born with it and it has made you unable to make normal hemoglobin and red cells. You have been receiving red blood cell transfusions all your life.
- Trial name
- NCT00658385
- Testing
- GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Thalassemia
Thalassemia Knowledge in Young People of Indonesia
There is still an ongoing lack of awareness about thalassemia among Indonesians despite high disease prevalence, and this is associated with poor implementation of preventive and promotive measures.
- Trial name
- NCT04706585
- Where
- Indonesia
- Volunteers
- 1,010 people, healthy volunteers welcome
- Sponsor
- Fakultas Kedokteran Universitas Indonesia