Clinical trials: Thalassemia Intermedia
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 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
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 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
Beta-Thalassemia
CS-101 in Patients With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06328764
- Testing
- CS-101
- Where
- China
- Volunteers
- 10 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Beta Thalassemia Intermedia
Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon
Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body.
- Trial name
- LB-04-THAL (NCT01642758)
- Testing
- Sodium 2,2 dimethylbutyrate
- Where
- Lebanon
- Volunteers
- 10 people
- Sponsor
- Boston University
Thalassemia Major, Transfusion-dependent Anemia
The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron…
- Trial name
- NCT05462548
- Testing
- Luspatercept Injectable Product
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Sun Yat-sen University
Sickle Cell Disease, Thalassemia
A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell…
- Trial name
- GLADIOLUS (NCT04987489)
- Testing
- Etavopivat tablets
- Where
- 32 sites in United States, Canada and 4 more
- Volunteers
- 53 people
- Sponsor
- Forma Therapeutics, Inc.
Thalassemia Major, Chronic Periodontitis
Periodontal Therapy in Thalassemia Major Patients
The study aims to evaluate the effect of local non surgical periodontal therapy on the systemic pro-inflammatory markers in the β-thalassemia (TM-β) patients with chronic periodontitis and systemically healthy demographically…
- Trial name
- NCT03311243
- Testing
- Non surgical periodontal therapy, Chemical plaque control
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- King Saud University
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
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-06 (NCT06024876),No investigational drug product will be administered in the study.
- Trial name
- NCT06685536
- Testing
- Safety and efficacy assessments
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Thalassemia Major
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.
- Trial name
- TICATS (NCT04291352)
- Testing
- Taurine
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- University Health Network, Toronto
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
Microcytic Hypochromic Anemia
Red Cell Distribution Width Index Versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .
Red Cell Distribution Width Index versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .
- Trial name
- NCT03868306
- Testing
- CBC , Iron study and Haemoglobin electrophoresis .
- Volunteers
- 100 people
- Sponsor
- Assiut University
Thalassemia Major, Thalassemia Intermedia
Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia
This is a multicenter observational case-control analysis lasting 12 months aimed at determining the prevalence of pulmonary hypertension (PAH) in patients with Thalassemia Major and Intermedia.
- Conditions
- Thalassemia Major, Thalassemia Intermedia, Pulmonary Arterial Hypertension
- Trial name
- PAH2010 (NCT01496963)
- Testing
- Physician standard-of-care according to ESC/ERS Guidelines
- Where
- 8 sites in Italy
- Volunteers
- 1,500 people
- Sponsor
- Ente Ospedaliero Ospedali Galliera