Clinical trials: Hemoglobinopathy
Sickle Cell Disease
Arterial Function Parameters and Transcranial Doppler Velocity in Paediatric Patients With Sickle Cell Disease
Structural and functional changes in arteries are increasingly being recognized as significant features of sickle cell disease. This study aims to determine whether there are differences in arterial function parameters between…
- Trial name
- AFAT (NCT05748717)
- Where
- Jamaica
- Volunteers
- 34 people
- Sponsor
- The University of The West Indies
Thalassemia Major
NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major
The effect of N_acetylcystein as an antioxidant on iron overload and frequency of blood transfusion in β-thalassemia major patients at Assiut Childern Hospital University And its cosubmitted for partial fulfillment of master…
- Trial name
- NCT05777733
- Testing
- n-acetylcystine
- Volunteers
- 100 people
- Sponsor
- Assiut University
Sickle Cell Disease
Sickle Cell Anemia WE CARE
This mixed-methods study aims to understand the implementation of a previously tested, efficacious social determinants of health (SDoH) screening and referral intervention in the outpatient pediatric hematology setting…
- Trial name
- SCAWECARE (NCT03716726)
- Testing
- WE CARE SDoH Screening Survey, Family Resource Book, Standard of care
- Where
- United States
- Volunteers
- 112 people, healthy volunteers welcome
- Sponsor
- Boston Medical Center
Sickle Cell Disease
Support Strategies for Parents During the First Year Following Their Child's Diagnosis of Sickle Cell Disorder
Background: Sickle cell disorder (SCD), the commonest genetic (faulty gene inherited from both parents) condition in the UK, affects mainly underserved groups.
- Trial name
- NCT06251843
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- King's College London
Genetic Disorders, Sickle Cell Anemia
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
The purpose of this study is to determine and confirm the role of bone marrow transplantation in the treatment of disorders of the red cell and hemoglobin including sickle cell anemia, thalassemia and diamond blackfan anemia.
- Trial name
- NCT00578435
- Testing
- Busulfan, Cyclophosphamide, BMD
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Anemia, Sickle Cell Thalassemia
Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With Fever
This study will summarized the clinical and laboratory data and the outcome of all the patients suffering from Sickle Cell Anemia (Including Sickle cell thalassemia) admitted to the pediatric ward.
- Trial name
- NCT00736060
- Testing
- Observational
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease
Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients
The constitution of blood relies upon hematopoietic stem cells (HSCs), which stay in the bone marrow and differentiate to all lineages of peripheral blood cells.
- Trial name
- NCT03226691
- Testing
- Plerixafor
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Sickle Cell Crisis
A Comprehensive Care Plan for Pediatric Patients With Vaso-Occlusive Crises
Sickle cell disease (SCD) is the most common inherited blood disorder affecting 80,000 to 90,000 individuals in the United States.[10] There are 13,000 hospital admissions for a sickle cell crises, costing $448 million dollars…
- Conditions
- Sickle Cell Disease, Sickle Cell Crisis, Vaso-Occlusive Crises
- Trial name
- NCT02698761
- Testing
- Comprehensive Care Plan
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Valley Anesthesiology Consultants
Sickle Cell Disease
HRV-B for Symptom Management in Sickle Cell Patients
This study will test the hypothesis that Heart Rate Variability Biofeedback (HRV-B) restores autonomic balance and reduces pain and other symptoms among patients with sickle cell disease (SCD).The specific aims of this study are…
- Trial name
- NCT03383913
- Testing
- Heart Rate Variability Biofeedback
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Prisma Health-Upstate
Thalassemia, Folic Acid Deficiency Anemia
Effect of Folic Acid Supplementation in Pregnant Women Having Thalassaemia Trait
Folic acid supplementation has been recommended for prevention of neural tube defects in pregnancy when taken periconceptionally up to 12 weeks of gestation.
- Trial name
- NCT04310059
- Testing
- Folic acid, Materna
- Volunteers
- 270 people, healthy volunteers welcome
- Sponsor
- The University of Hong Kong
Thalassemia, Sickle Cell Anemia
Blood Transfusions in Thalassemia Patients, Complications and Adverse Effects
Patients suffering from Thalassemia or another hemoglobinopathies required regular blood transfusions. The complications and adverse effects of blood transfusions can be classified as immediate and late.
- Trial name
- NCT00971880
- Testing
- Clinical medical record summary
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- HaEmek Medical Center, Israel
Anemia, Sickle Cell, Sickle Cell Anemia
Safety Study of MP4CO in Adult Sickle Cell Patients
Sickle Cell Anemia is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body.
- Conditions
- Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders
- Trial name
- NCT01356485
- Testing
- MP4CO, Sodium chloride solution
- Where
- 5 sites in France, Jamaica and 2 more
- Volunteers
- 32 people
- Sponsor
- Sangart
Sickle Cell Disease
A Phase Ib Study of NVX-508 in Sickle Cell Disease
This Phase 1b study in adults with sickle cell disease (SCD) in steady-state (non-acutely ill) aims to evaluate safety and toxicity of NVX-508 in a multi-dosing paradigm as well as to determine the maximum tolerated dose (MTD) in…
- Trial name
- NCT03013426
- Testing
- NVX-508
- Sponsor
- Amma Owusu-Ansah, MD
Sickle Cell Disease, Stroke
Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease
This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease.
- Trial name
- HUPrevent (NCT01389024)
- Testing
- Hydroxyurea
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Johns Hopkins University
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
Sickle Cell Disease
Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes.
- Trial name
- SCURT (NCT00745420)
- Testing
- Hematopoietic Stem Cell Transplantation
- Where
- 19 sites in United States
- Volunteers
- 30 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease
The Brigance Assessment Of Individual Neurodevelopment In Young Children With Sickle Cell Disease- 2
A preliminary study was conducted involving 88 three-year-old children with sickle cell disease (SCD) who were followed at the St. Jude Children's Research Hospital Sickle Cell Center.
- Trial name
- NCT01123863
- Testing
- Brigance Preschool Screen -II
- Where
- United States
- Volunteers
- 248 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Anemia, Sickle Cell, Sickling Disorder Due to Hemoglobin S
Comparison of Two Methods of Transfusion for Stroke Prevention in Sickle Cell
Chronic blood transfusions are essential supportive care for sickle cell patients at high risk for morbidity and mortality due to stroke. These patients, however, are at risk for iron overload.
- Trial name
- NCT02561312
- Testing
- Rapid manual partial exchange transfusion, Simple Transfusion
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Chattanooga-Hamilton County Hospital Authority
Sickle Cell Disease
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
- Trial name
- PRECISE (NCT06923111)
- Testing
- Xromi
- Where
- 12 sites in Germany, United Kingdom
- Volunteers
- 180 people
- Sponsor
- Nova Laboratories Limited
Transfusion-dependent Thalassemia
Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia
Transfusion Dependent Thalassemia (TDT) is emerging as a global public health concern. Hemopoietic stem cell transplantation (HSCT) is the only curative treatment.
- Trial name
- NCT06098014
- Testing
- Thalidomide Capsules
- Where
- Bangladesh
- Volunteers
- 60 people
- Sponsor
- Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh