Clinical trials: Thalassemia in Children, open to volunteers
B Thalassemia Major
Oral Health Status in Children With β-Thalassemia Major
To evaluate the effectiveness of a structured oral health educational program on oral hygiene status and oral health-related quality of life (OHRQoL) among children diagnosed with β-thalassemia.
- Trial name
- NCT07614217
- Testing
- oral health educational program for Immediate Intervention Group, routine oral hygiene instructions
- Volunteers
- 46 people
- Sponsor
- Cairo University
Sickle Cell Disease
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical…
- Trial name
- NYMC-571 (NCT02675959)
- Testing
- Defibrotide
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
Thalassemia Majors (Beta-Thalassemia Major)
Risk Factors Affecting Growth in Thalassemic Children at AUCH
Thalassemia major is a hereditary hemoglobinopathy characterized by ineffective erythropoiesis and severe anemia, necessitating lifelong blood transfusions(1,2).
- Trial name
- NCT07509996
- Testing
- Non-interventional assessment of growth and risk factors in thalassemic children
- Volunteers
- 85 people
- Sponsor
- Assiut University
Incentive Spirometer, Pulmonary Function
Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major
The study was done to: 1. Investigate the effect of respiratory training on functional lung capacity and 2. To detect the effect of respiratory training on pulmonary functions in children with β-thalassemia major. 3.
- Conditions
- Incentive Spirometer, Pulmonary Function, Functional Capacity, Child, Only
- Trial name
- NCT07112703
- Testing
- Incentive spirometer exercises, Diaphragmatic breathing, Pursed lip breathing:
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Rawan Mohammed Khairy Mostafa Elsawy
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
Thalassemia Particularly Transfusion Dependent Thalassemia
Clinical, Economic,and Psychosocial Burden of Thalassemia in Sohag
This study aims to assess the clinical, economic, and psychosocial burden of thalassemia among affected children in Sohag. Clinically, the study will evaluate disease severity, transfusion requirements, iron overload…
- Trial name
- HBT-sohag (NCT07796022)
- Volunteers
- 100 people
- Sponsor
- Randa Elham Hamam
Thalassemia, Hematopoietic Stem Cell Transplantation
Low-Dose Liposomal Amphotericin B for Invasive Fungal Infection Prophylaxis in Neutropenic Children
This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (1 mg/kg/day, intravenous, once daily) for the prevention of invasive fungal infections in…
- Conditions
- Thalassemia, Hematopoietic Stem Cell Transplantation, Neutropenia, Invasive Fungal Infections
- Trial name
- NCT07725263
- Testing
- Liposomal Amphotericin B (LAmB)
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
Thalassaemia
Thalassaemia Severity
This study aims to develop a nuanced understanding of thalassemia severity in Upper Egypt by integrating various clinical parameters beyond transfusion frequency.
- Trial name
- NCT06720480
- Testing
- Homglobin Electrophoresis
- Volunteers
- 85 people
- Sponsor
- Assiut University
β-thalassemia
the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
- Trial name
- NCT05864170
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Shenzhen Hemogen
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who…
- Trial name
- NCT05991336
- Testing
- Gene therapy
- Where
- China
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S