Clinical trials: Thalassemia
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Transfusion Dependent Beta Thalassemia, Hemoglobinopathies
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
- Conditions
- Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
- Trial name
- NCT05444894
- Testing
- EDIT-301
- Where
- 8 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Editas Medicine, Inc.
Anemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the…
- Trial name
- NCT05664737
- Testing
- Luspatercept
- Where
- 31 sites in China, Greece and 8 more
- Volunteers
- 189 people
- Sponsor
- Bristol-Myers Squibb
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
Sickle Cell Disease, Sickle-Beta Zero Thalassemia
Dose-Escalation Study of SCD-101 in Sickle Cell Disease
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
- Trial name
- NCT02380079
- Testing
- SCD-101
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Invenux, LLC
β -Thalassemia
A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia
This is a phase II, randomized, double-blinded, placebo-controlled study to treat patients with transfusion-dependent and non-transfusion dependent β -thalassemia with AND017 and optimal supportive care, including blood…
- Trial name
- NCT06302491
- Testing
- AND017 capsules
- Where
- 5 sites in China
- Volunteers
- 64 people
- Sponsor
- Kind Pharmaceuticals LLC
Beta-Thalassemia
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.
- Trial name
- NCT06065189
- Testing
- CS-101 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Children's Hospital of Fudan University
β-thalassemia
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector…
- Trial name
- NCT06465550
- Testing
- BD211
- Where
- 3 sites in China
- Volunteers
- 9 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Transfusion Dependent Beta-Thalassaemia
Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Trial name
- NCT04390971
- Testing
- ET-01
- Where
- 3 sites in China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Iron Overload, Oxidative Stress
Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
The aim of the present study is evaluating the strength of combination therapy of hydroxy urea, omega 3, nigella sativa and honey on antioxidant-oxidant status (OXIDATIVE STRESS) in response to reactive oxygen species production…
- Conditions
- Iron Overload, Oxidative Stress, Thalassemia Major
- Trial name
- NCT04292314
- Testing
- Omega 3, Nigella Sativa Oil, Hydroxyurea
- Where
- 4 sites in Egypt, Saudi Arabia
- Volunteers
- 350 people
- Sponsor
- Beni-Suef University
Sickle Cell Disease, Beta-thalassemia
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions.
- Trial name
- NCT07599176
- Testing
- Total Body Irradiation, Alemtuzumab, Abatacept
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
SCD With Severe Phenotype (HbSS, HbSβ0 Thalassemia, HbSOARab)
QST-Pupillometry in Sickle Cell Disease Patients
There has been little progress for effective treatment of pain in sickle cell disease (SCD) patients. Many organizations have recognized that understanding the causes and reducing the burden of pain in SCD is critical in order to…
- Trial name
- QST (NCT02242058)
- Testing
- Quantitative sensory testing
- Where
- 2 sites in United States
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Julia Finkel
Thalassemia
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels.
- Trial name
- NCT00661726
- Testing
- Decitabine (USAN, INN)
- Where
- 3 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- Carelon Research
Iron Overload, Transfusion-dependent Thalassemia
Endocrine Function During Deferasirox Therapy
A national survey on the prevalence and natural history of endocrine complications in thalassemia transfusion--dependent patients treated with deferasirox was designed, in order to assess a larger population during a longer…
- Conditions
- Iron Overload, Transfusion-dependent Thalassemia, Endocrine; Complications
- Trial name
- NCT04515680
- Where
- Italy
- Volunteers
- 426 people
- Sponsor
- University of Campania Luigi Vanvitelli
Osteoporosis, Thalassemia Major
Diagnostic Pilot Study of Dual Energy Absorptiometry in the Detection of Osteopenia or Osteoporosis in Patients With Thalassemia Major
OBJECTIVES: I. Determine the frequency and severity of osteopenia and osteoporosis in patients with thalassemia major who undergo dual energy x-ray absorptiometry, and correlate these findings with other relevant endocrinologic…
- Conditions
- Osteoporosis, Thalassemia Major, Osteopenia
- Trial name
- NCT00006138
- Volunteers
- 35 people
- Sponsor
- National Center for Research Resources (NCRR)
Beta Thalassemia
Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram
Heart failure from myocardial iron deposition is a severe complication for patients with transfusion-dependent beta thalassemia .
- Trial name
- NCT04929574
- Testing
- Echocardiogram and Hematological Analysis
- Where
- China
- Volunteers
- 1,200 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Alpha and Beta Thalassemia, Sickle Cell Disease
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Background: Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders.
- Conditions
- Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
- Trial name
- NCT03937817
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
β-thalassemia Major
β-globin Restored Autologous HSC in β-thalassemia Major Patients
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
- Trial name
- NCT04205435
- Testing
- β-globin restored autologous HSC
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Bioray Laboratories
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
Thalassemia Major, Osteoporosis
Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis
This is a single-site, randomized, placebo-controlled, double blind phase 2b clinical trial. Patients with Thalassemia will participate in this study and will be treated with Denosumab or placebo.
- Trial name
- NCT02559648
- Testing
- Denosumab
- Where
- Greece
- Volunteers
- 63 people
- Sponsor
- Ersi Voskaridou