Clinical trials: Hereditary Anemias
Sickle Cell Disease, Cerebral Stroke
Cerebrovascular Reactivity Measurements With High-Density Diffuse Optical Tomography
The purpose of the study protocol is to identify imaging biomarkers for brain tissue under high metabolic stress at risk for permanent injury.
- Trial name
- HDDOT CVR (NCT07776444)
- Testing
- Carbon Dioxide
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Crohn Disease
Study to Evaluate the Efficacy and Safety of PF-06480605 in Adult Participants With Moderate to Severe Crohn's Disease
This Phase 2a, multicenter, randomized, double-blind, placebo-controlled study examines subcutaneous dose of PF-06480605 150 mg administered every 4 weeks in participants with moderate to severe active Crohn's Disease to…
- Trial name
- NCT05471492
- Testing
- Investigational Product
- Sponsor
- Telavant, Inc.
Sickle Cell Disease
School Readiness Intervention for Preschool Children With Sickle Cell Disease
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease.
- Trial name
- NCT06367192
- Testing
- The adapted Kids in Transition to School Intervention, Standard school resources
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)
'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949.
- Trial name
- NCT02824471
- Testing
- SCD Group
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Hematologic Diseases, Anaemia, Sickle Cell
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years
This will be a first time in human (FTIH) study in sickle cell diseases (SCD) participants. The FTIH study is planned to evaluate the safety, tolerability, and pharmacokinetics of GSK4172239D.
- Trial name
- NCT05660265
- Testing
- GSK4172239D
- Where
- 6 sites in United States
- Volunteers
- 11 people
- Sponsor
- GlaxoSmithKline
Sickle Cell Disease, Venous Thromboembolism
Comparison of Thrombin Generation, Platelet Activation and Aggregation Profiles in Patients With Sickle Cell Disease Depending on Their SS or SC Genotype (DrepAgreg)
Sickle cell disease (SCD) is characterized by chronic hemolytic anemia, painful crises called vaso-occlusive crises (VOC) and chronic inflammation.
- Trial name
- DrepAgreg (NCT07852442)
- Where
- 2 sites in France, Guadeloupe
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
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
Sickle Cell Disease
Pathophysiology of Acute Pain in Patients With Sickle Cell Disease
Background: Sickle Cell Disease (SCD) is a blood disorder that occurs mainly in people of African descent. Researchers want to learn more about the painful attacks and complications associated with SCD.
- Trial name
- NCT03049475
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Vaso-Occlusive Crises, Sickle Cell Disease
Evaluation of the Efficacy of Iloprost in the Management of Vaso-occlusive Crises in Adult Patients With Sickle Cell Disease
Sickle cell disease is a severe monogenic genetic disorder caused by an autosomal recessive mutation of the β-globin gene, leading to production of abnormal hemoglobin (HbS).
- Trial name
- PROSTASICKLE (NCT07498309)
- Testing
- Iloprost
- Where
- France
- Volunteers
- 144 people
- Sponsor
- University Hospital, Rouen
Sickle Cell Anemia
The Efficacy of Jobelyn (Sorghum Bicolor Extract)in the Treatment of Sickle Cell Anemia
The primary objective is to determine if there is a significant increase in the haematocrit value of patients on Jobelyn and standard therapy compared to those on standard therapy alone.
- Trial name
- NCT01703104
- Testing
- Paludrine + Folic Acid, Paludrine + Folic Acid + Jobelyn
- Where
- Nigeria
- Volunteers
- 150 people
- Sponsor
- Lagos State University
Sickle Cell Disease
Near Infrared Spectroscopy in Sickle Cell Pediatric Patients
Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease. Near infrared spectroscopy (NIRS) technology can measure tissue oxygenation and endothelial function.
- Trial name
- NCT04031521
- Testing
- Near infrared spectroscopy
- Where
- United States
- Sponsor
- Children's National Research Institute
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
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
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
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
Study of a Red Blood Cell Deformability Parameter in Patients With Sickle Cell Disease
Sickle cell disease is the most common genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS) which, in its deoxygenated form, polymerizes and causes structural changes in red blood cells…
- Trial name
- Drepaforme (NCT03977532)
- Testing
- Micro-blood sampling
- Where
- France
- Volunteers
- 21 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Sickle-Cell Disease
Sevuparin Infusion for the Management of Acute VOC in Subjects With SCD
A Multi-Centre, Phase II, Randomized, Double-Blind, Placebo-Controlled Study to investigate Efficacy and Safety of Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell Disease…
- Trial name
- NCT02515838
- Testing
- Sevuparin
- Where
- 20 sites in Bahrain, Jamaica and 5 more
- Volunteers
- 147 people
- Sponsor
- Modus Therapeutics AB
Sickle Cell Disease
A Safety Study of Eptifibatide in Patients With Sickle Cell Disease
This study will evaluate the safety of eptifibatide in sickle cell patients and how well it works during the course of painful crises.
- Trial name
- NCT00834899
- Testing
- Eptifibatide
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke.
- Trial name
- HIBISCUS 3 (NCT05953584)
- Testing
- Etavopivat, Hydroxyurea
- Where
- 9 sites in India, Nigeria and 1 more
- Volunteers
- 27 people
- Sponsor
- Forma Therapeutics, Inc.
Cystic Fibrosis, Anemia, Iron-Deficiency
Does a Daily Iron Tablet Improve Anemia in Cystic Fibrosis
This study questions whether low-dose ferrous sulfate taken by mouth daily for 6 weeks increases hemoglobin concentration in adult subjects with cystic fibrosis and hypoferremic anemia.
- Trial name
- NCT01755455
- Testing
- Ferrous sulfate 325mg
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Dartmouth-Hitchcock Medical Center
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