Clinical trials: Thalassemia Intermedia
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Thalassemia Major (TM), Thalassemia Intermedia (TI)
Beta-thalassemia and Microparticles
The results will allow us to evaluate the role of MP in the thrombo-embolic risk observed in thalassemic patients and to underline a possible difference between TM and TI.
- Conditions
- Thalassemia Major (TM), Thalassemia Intermedia (TI), Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes
- Trial name
- NCT01284738
- Testing
- Physiopathology
- Where
- France
- Volunteers
- 33 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Beta-Thalassemia
Iron Status in BTM With Blood Transfusion
To assess the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.
- Trial name
- NCT04947540
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assiut University
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
Beta Thalassemia Major
Effect of Spirulina on Serum Hyaluronic Acid in Beta Thalassemic Children With Hepatitis C
Thalassemics can develop liver fibrosis because of iron overload and hepatitis C infection. The latter is the main risk factor for liver fibrosis in transfusion dependent thalassemics.
- Trial name
- NCT02744547
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Tanta University
Thalassemia Major
Observing the Changes of Endocrine and Metabolism in Patients With Thalassemia Major
Patient with thalassemia major have many endocrine diseases, such as hypopituitarism, hypothyroidism, hypoparathyroidis, osteoporosis, and etc.. These problems may be due to anemia itself or related to iron deposition.
- Trial name
- NCT03951818
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- National Taiwan University Hospital
Beta-Thalassemia
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in…
- Trial name
- NCT06308159
- Testing
- Vebeglogene autotemcel
- Where
- 2 sites in China
- Volunteers
- 6 people
- Sponsor
- Lantu Biopharma
Beta-Thalassemia, Iron Overload
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients
This is a randomized, open label, two arms superiority trial of a representative population of patients with a primary diagnosis of transfusion dependent thalassemia with evidence of moderate cardiac iron overload, defined as an…
- Trial name
- CANALI (NCT02474420)
- Testing
- Amlodipine, Deferasirox
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- Kevin H.M. Kuo, MD, MSc, FRCPC
Myelodysplastic Syndromes, Beta-thalassemia
A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia
The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic…
- Trial name
- NCT07681440
- Testing
- Luspatercept
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Bristol-Myers Squibb
Beta Thalassemia Major
Effect of Spirulina on Liver Iron Concentration in Beta Thalassemic Children With Hepatitis C
Thalassemics can develop liver fibrosis because of iron overload and hepatitis C infection. The latter is the main risk factor for liver fibrosis in transfusion dependent thalassemics.
- Trial name
- NCT02744560
- Testing
- spirulina
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Tanta University
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
β-thalassemia, Ineffective Erythropoiesis
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment…
- Conditions
- β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
- Trial name
- TRANSCEND (NCT03802201)
- Testing
- PTG-300
- Where
- 33 sites in United States, Greece and 7 more
- Volunteers
- 63 people
- Sponsor
- Protagonist Therapeutics, Inc.
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Thalassemia Major
The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study
The purpose of this pilot study is to determine the effect of various doses of vitamin D supplementation on vitamin D stores and calcium excretion in the urine in subjects with Thalassemia Major (TM).
- Trial name
- NCT01323608
- Testing
- Vitamin D3
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Weill Medical College of Cornell University
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Diabetes, Prediabetes
The Use of CGMS to Detect Alterations of Blood Glucose in Thalassemic Patients
The aim of the study is to: Assess the pattern of glucose homeostasis in patients with B thalassemia . To detect early impairment in glucose metabolism and prediabetic state in B thalassemia patients using continuous glucose…
- Conditions
- Diabetes, Prediabetes, B-Thalassemia
- Trial name
- NCT03591900
- Testing
- insulin pump/CGMS
- Volunteers
- 200 people
- Sponsor
- Ain Shams 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
Beta Thalassemia Major Anemia
Thrombin Generation in Beta-thalassemia Major
This study aims to evaluate the persistence of a hypercoagulable state in chronically-transfused patients with beta thalassemia major, by using the thrombin generation test (performed in whole blood and plasma).
- Trial name
- TG-THAL (NCT04962984)
- Testing
- blood sampling
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Central Hospital, Nancy, France
Transfusion Dependent Beta-thalassemia
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism…
- Trial name
- NCT06606886
- Testing
- Tablet Metformin (Group A), Tablet Hydroxyurea (Group B)
- Where
- Pakistan
- Volunteers
- 96 people
- Sponsor
- Bahria University
Beta-Thalassemia
Dermatological Abnormalities in Beta-thalassemia Major
to study the frequency and pattern of dermatological abnormalities in egyptian childern with beta thalassemia.relationof abnormalities to duration of disease and frequency of transfusion
- Trial name
- NCT03894605
- Testing
- thalassemia
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Assiut University