Clinical trials: Thalassemia Intermedia
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Beta Thalassemia Major
the Potential Immunomodulatory Effects of Spirulina on Thalassemic Children
The aim of this study is to evaluate the possible immunologic effects of spirulina in children with beta thalassemia.
- Trial name
- NCT02674607
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Tanta University
AL-Hijama in Thalassmia Major
Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major
1. Evaluate the therapeutic effects, health benefits, and immunological effect of Al-hijamah in children with beta thalassemia major. 2.
- Trial name
- Al-hijamah (NCT02761395)
- Testing
- Al-hijamah, Deferasirox
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Tanta University
Thalassemia, Sickle Cell Disease
Iron Overload and Growth Velocity in Thalassemia and Sickle Cell Anemia
Iron overload impaired growth in Thalassemia patients due to iron deposition in the endocrine glands, including the hypophysis and gonads.
- Trial name
- NCT00972231
- Testing
- Medical Chart Summary
- Where
- Israel
- Volunteers
- 93 people
- Sponsor
- HaEmek Medical Center, Israel
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
Transfusion Dependent Beta-Thalassemia
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced…
- Trial name
- NCT05762510
- Testing
- GMCN-508B (LentiRed)
- Where
- China
- Volunteers
- 5 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
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
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Beta Thalassemia Major Anemia
The Potential Hepatoprotective Effect of Metformin in Patients With Beta Thalassemia Major
Beta Thalassemia is a major public health problem in Mediterranean countries.In Egypt, it is considered as the most common chronic hemolytic anemia.one of the major complications in this inherited disorder is iron overload which…
- Trial name
- NCT02984475
- Testing
- Metformin
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Cairo University
Thalassemia, Beta
Clinical Experience of Thalidomide in Thalassemic Patients
Objectives Primary objective: To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
- Trial name
- NCT05132270
- Testing
- hydroxyurea and thalidomide
- Where
- Pakistan
- Volunteers
- 135 people
- Sponsor
- Children's Hospital Karachi
Thalassemia Major
Perceptions of Thalassemia Major in Singapore: An Exploratory Study of Stigma
Background: Thalassemia major (TM) is a chronic disorder that affects a person s ability to produce hemoglobin, resulting in anemia.
- Trial name
- NCT01129076
- Where
- 2 sites in Singapore
- Volunteers
- 30 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Beta-Thalassemia
The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients
To design an amplification-refractory mutation system (ARMS) for the DNA diagnosis of the IVS I-110 (G>A) [HBB:c.93-21G˃A] mutation. To detect the prevelence of the mutation among Assiut University Hospital patients.
- Trial name
- NCT05133388
- Testing
- ARMS PCR
- Where
- Egypt
- Volunteers
- 150 people
- Sponsor
- Assiut University
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Thalassemia Major, Acute Leukemia
Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors
Objective: To longitudinally track the dynamic changes in the survival quality of pediatric patients after hematopoietic stem cell transplantation at different time points within 1 year post-transplantation, analyze the…
- Trial name
- NCT06026839
- Testing
- Conventional nursing
- Where
- China
- Volunteers
- 250 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Beta-Thalassemia, Insulin Resistance
Resistance Exercise on Postprandial Hyperglycemia in Patients With B-thalassemia Exhibiting Resistance to Insulin
It is known that postprandial hyperglycemia increases the cardiometabolic risk in both diabetic and non-diabetic patients. Moreover, there is insufficient data on the effectiveness of exercise on preventing Type II diabetes…
- Conditions
- Beta-Thalassemia, Insulin Resistance, PreDiabetes, Diabetes Mellitus, Type 2
- Trial name
- NCT03889977
- Testing
- Resistance exercise
- Where
- Greece
- Volunteers
- 6 people
- Sponsor
- University of Thessaly
Sickle Cell Disease, Thalassemia, Beta
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow.
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Trial name
- NCT05736419
- Testing
- Fludarabine, Cyclophosphamide, Tacrolimus
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
β-thalassemia Major
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major.
- Trial name
- NCT01745120
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 6 sites in United States, Australia and 1 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Beta Thalassemia Transfusion Dependent
Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec
This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
- Trial name
- NCT00879242
- Testing
- Deferasirox
- Where
- 2 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Beta-Thalassemia, Non-transfusion-dependent Thalassemia
Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia
This is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent…
- Trial name
- VITHAL (NCT04364269)
- Testing
- VIT-2763 once a day (QD), VIT-2763 twice a day (BID)
- Where
- 16 sites in Greece, Israel and 3 more
- Volunteers
- 35 people
- Sponsor
- Vifor (International) Inc.
Beta Thalassemia Major
An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients
A prospective randomized study on Safety, Tolerability and Efficacy of oral Low dose DFP (50 mg/kg/day) in minimally transfused B-TM after 5 transfusions when SF reaches 500 ng/m and with either appearance of LPI > 0.2 or TSAT…
- Trial name
- NCT02173951
- Testing
- Deferiprone
- Where
- Egypt
- Volunteers
- 64 people
- Sponsor
- Ain Shams University