Clinical trials: Thalassaemia
Transfusion-dependent β-thalassemia Patients, Cardiac Iron Overload
Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload
The primary efficacy endpoint of this interventional study was to evaluate the number of patients achieving a complete response (CR), defined as patients switching from intensive deferasirox -DFO treatment, at any time point…
- Trial name
- NCT01459718
- Testing
- Deferasirox, Deferoxamine (DFO)
- Where
- 4 sites in Greece
- Volunteers
- 32 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia, Iron Overload
Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients
The safety and efficacy of CN128 is studied in thalassaemia with sever liver iron overloaded patients.
- Trial name
- NCT05355766
- Testing
- CN128 Tablets
- Where
- China
- Volunteers
- 16 people
- Sponsor
- Hangzhou Zede Pharma-Tech Co., Ltd.
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
Thalassemia, Iron Deficiency
Detection of β Thalassemia Carriers by Red Cell Parameters Obtained From the H2 Automatic Counter
β thalassemia is an autosomal recessive hemoglobinopathy and considered as the most widespread genetic mutation. According to the World Health Organization (WHO) between 1.5-7% of the world population are carriers for this…
- Trial name
- NCT00481221
- Testing
- Observation of results from laboratory tests
- Where
- Israel
- Volunteers
- 30,000 people
- Sponsor
- HaEmek Medical Center, Israel
Thalassemia in Children
Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia
The purpose of this study is to evaluate a novel transplant strategy for the long-term benefit of patients with transfusion dependent high-risk thalassemia.
- Trial name
- NCT05426252
- Testing
- Abatacept, Sirolimus
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- The Hospital for Sick Children
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
Iron Overload, Thalassemia
Combination Iron Chelation Therapy
Patients with beta-thalassemia (Cooley's Anemia) continue to suffer from the transfusion-induced iron overload due to the inadequacies of current iron-chelation therapy.
- Trial name
- NCT00004982
- Testing
- Combination Iron Chelation Therapy
- Where
- United States
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Beta-thalassemia Major
Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients
Background: Three iron chelators now available on the market differ in toxicity and organ specificity; evidence on standardized chelation protocol remains inconclusive, but patients with transfusion-dependent beta-thalassemia…
- Trial name
- NCT02198508
- Testing
- DFX(Deferasirox), DFP(Deferiprone)
- Volunteers
- 13 people
- Sponsor
- China Medical University Hospital
Beta-Thalassemia Trait, Coronary Artery Disease
B-Thalasaemia Trait and Its Possible Cardioprotective Role
Our purpose is to investigate whether thallasaemia trait offers protection against cardiovascular events and whether this can be applied to specific characteristics of these people ( better lipid profile , better blood pressure…
- Trial name
- NCT02428023
- Where
- Greece
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- 404 General Military Hospital, Larissa
Thalassemia
Assessment of Nutritional Status and Role of Insulin-like Growth Factor-1 in Children
Many studies done on children with thalassemia have shown a variable prevalence of defective IGF-I concentrations have been shown to be low in the majority of children, with or without GH deficiency.
- Trial name
- NCT05529550
- Where
- Egypt
- Volunteers
- 102 people
- Sponsor
- Menoufia University