Clinical trials: Thalassemia
Hematologic Malignancies, Nonmalignant Diseases
Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609814
- Testing
- Clofarabine, Fludarabine Injection
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Beta Thalassemia Major
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
- Trial name
- NCT03947632
- Testing
- serum ferritin and hemoglobin test
- Volunteers
- 367 people
- Sponsor
- Aga Khan University
Atrial Fibrillation, Thalassemia
Atrial Fibrillation in Beta-Thalassemia
The study aims to evaluate the clinical, laboratory and instrumental differences that exist between beta-thalassemia patients with atrial fibrillation and those not affected by arrhythmia.
- Trial name
- NCT05508932
- Where
- Italy
- Volunteers
- 350 people
- Sponsor
- University Hospital of Ferrara
Sickle Cell Disease
Screening Patients With Sickle Cell Disease for Kidney Damage
This study aims to study the temporal course of sickle nephropathy and assess novel biomarkers that can predict patients prone to nephropathy.
- Trial name
- NCT02239016
- Where
- 4 sites in United States
- Volunteers
- 320 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Open-Label Extension Study to Pioneer Study 6058-SCD-101
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder…
- Trial name
- NCT07401823
- Testing
- Pociredir
- Where
- 8 sites in United States
- Volunteers
- 50 people
- Sponsor
- Fulcrum Therapeutics
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Beta-Thalassemia
Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen
repeated transfusions are the mainstay of disease management in most patients with transfusion dependent beta thalassemia.iron overload predispose to oxidative stress and tissue injury.
- Trial name
- NCT06509581
- Testing
- vitamin E
- Where
- Egypt
- Volunteers
- 180 people
- Sponsor
- Ain Shams 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
Thalassemia
Development of THALEA Kit and Its Impact on Knowledge, Attitude, and Intention for Premarital Thalassemia Screening
Thalassemia, a common genetic disease, is often overlooked compared to infectious diseases like HIV, despite its significant effects for families planning to have children.
- Trial name
- THALEA (NCT06800495)
- Testing
- THALEA (Thalassemia Education and Awareness) Kit, Basic thalassemia and family health education
- Where
- Malaysia
- Volunteers
- 148 people, healthy volunteers welcome
- Sponsor
- Universiti Teknologi Mara
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
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
Pain
The Effect of Acupressure on Pain in Children With Thalassemia
This randomized controlled trial evaluates the effect of acupressure on reducing the pain of blood collection in children with Thalassemia. The hypothesis of this study is that acupressure application reduces acute pain.
- Trial name
- acupressure (NCT06163196)
- Testing
- acupressure
- Where
- Turkey (Türkiye)
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Mersin University
Iron Overload, Cardiomyopathy
Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major
Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces myocardial iron and improves cardiac function.
- Trial name
- DFODFPTM (NCT00800761)
- Testing
- Deferoxamine and Deferiprone, Deferoxamine
- Where
- Italy
- Sponsor
- Ospedale Microcitemico
Hemolytic Anemia, Thalassemia
Native T1 Mapping and ECV in Chronic Hemolytic Anemia
This cross-sectional study aims to evaluate the diagnostic performance of native T1 mapping and extracellular volume fraction for the detection of myocardial iron overload and fibrosis in patients with chronic hemolytic anemia…
- Conditions
- Hemolytic Anemia, Thalassemia, Iron Overload
- Trial name
- NCT07882303
- Volunteers
- 54 people
- Sponsor
- Assiut University
Thalassemia Majors (Beta-Thalassemia Major), Haplo-identical Donors
Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
- Trial name
- NCT06734520
- Testing
- super-transplantation in 3 severe β -thalassemia pediatric patients.
- Where
- 2 sites in China
- Volunteers
- 3 people
- Sponsor
- Guangzhou Women and Children's Medical Center
β-thalassemia Major
A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-Thalassemia Major
- Trial name
- NCT07489196
- Testing
- CS-101 injection
- Where
- 3 sites in China
- Volunteers
- 20 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
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
Transfusion Dependent Beta Thalassemia
Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation.
- Trial name
- Thal-H (NCT07292259)
- Testing
- ADDITION OF THALIDOMIDE AND HYDROXYUREA
- Where
- 9 sites in Pakistan
- Volunteers
- 100 people
- Sponsor
- Pakistan Blood and Marrow Transplant (PBMT) Group
Β-thalassemia
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia.
- Trial name
- NCT06655662
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- 3 sites in China
- Volunteers
- 8 people
- Sponsor
- Shenzhen Hemogen
Thalassemia, Sickle Cell Anemia
Sickle Cell Anemia Screening and Prevention in Northern Israel
Since 1987, a screening for β Thalassemia in pregnant women is carried on in northern Israel, and from 1999 all the samples were tested also for Hgb S, Hgb C, Hgb D, Hgb O Arab and others.
- Trial name
- NCT00735488
- Testing
- Observational
- Where
- Israel
- Volunteers
- 69 people
- Sponsor
- HaEmek Medical Center, Israel