Clinical trials: Sickle Cell Disorders
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease, Sickle Cell Anemia
Stroke Prevention in Young Adults With Sickle Cell Anemia
Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide. Approximately 150,000 Nigerian children are born each year with sickle cell disease (SCD), making it the country with the…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Stroke, Ischemic, Silent Stroke
- Trial name
- SPIYA (NCT04808778)
- Testing
- Hydroxyurea therapy per standard care
- Where
- 2 sites in United States, Nigeria
- Volunteers
- 250 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease.
- Trial name
- NCT01794000
- Testing
- Prasugrel
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 341 people
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle…
- Trial name
- Hibiscus 2 (NCT06612268)
- Testing
- Etavopivat
- Where
- 175 sites in United States, Australia and 19 more
- Volunteers
- 510 people
- Sponsor
- Novo Nordisk A/S
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
- 1 people
- Sponsor
- Fulcrum Therapeutics
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
Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without Hemolysis
People who have Sickle Cell Anemia (HbSS) produce red blood cells with shorter lifespans. These red blood cells breakdown faster, and this is called hemolysis.
- Trial name
- NCT01848691
- Testing
- Measurement of carbon monoxide level in exhaled breath
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Ashutosh Lal
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
- Trial name
- IMPACTS-2 (NCT01522196)
- Testing
- Varespladib
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Anthera Pharmaceuticals
Sickle Cell Anemia in Children, Malaria
Dihydroartemisinin-Piperaquine or Sulphadoxine-Pyrimethamine for the Chemoprevention of Malaria in Sickle Cell Anaemia
Sickle Cell Anaemia (SCA) is an inherited disease that makes the body produce red blood cells with abnormal sickle-shaped cells. The sickle-shaped cells are rigid, not flexible and break up easily resulting in anaemia.
- Trial name
- CHEMCHA (NCT04844099)
- Testing
- Dihydroartemisinin Piperaquine
- Where
- 3 sites in Malawi, Uganda
- Volunteers
- 723 people
- Sponsor
- Liverpool School of Tropical Medicine
Sickle Cell Anemia, Retinopathy
Ranibizumab for Neovascularization in Sickle Cell Retinopathy
The purpose of this study is to determine the ocular and non-ocular safety of a single dose of ranibizumab in treating neovascularization secondary to sickle cell retinopathy.
- Trial name
- NCT00618644
- Testing
- Ranibizumab
- Where
- United States
- Sponsor
- Kresge Eye Institute
Sickle Cell Disease
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of…
- Trial name
- LIBRA (NCT06975865)
- Testing
- Rilzabrutinib
- Where
- 63 sites in United States, Belgium and 12 more
- Volunteers
- 192 people
- Sponsor
- Sanofi
Sickle Cell Trait, Sickle Cell Disease
Blood Sampling for Research Related to Sickle Cell Disease
This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease.
- Conditions
- Sickle Cell Trait, Sickle Cell Disease, Sickle Cell Anemia
- Trial name
- NCT00542230
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Thalassemia, Sickle Cell Disease
Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations
The investigators' primary objective is to study prevalences of myocardial iron overload, defined as a cardiac T2*< 20 ms, in 3 populations of multiply transfused patients, affected with thalassemia, sickle cell disease, and…
- Conditions
- Thalassemia, Sickle Cell Disease, Myelodysplasia
- Trial name
- SUFEMYO (NCT01911871)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 110 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Stroke
Primary Prevention of Stroke in Children With Sickle Cell Anaemia in Nigeria in the Community
The overall goal of this feasibility study is to establish a standard of care stroke prevention program for children with sickle cell anemia in a community hospital by task shifting stroke detection and transcranial Doppler…
- Trial name
- SPRING-COM (NCT05434000)
- Testing
- Hydroxyurea
- Where
- Nigeria
- Volunteers
- 217 people
- Sponsor
- Barau Dikko Teaching Hospital
Sickle Cell Disease
A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
- Trial name
- NCT03401112
- Testing
- IMR-687
- Where
- 13 sites in United States, United Kingdom
- Volunteers
- 100 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Sickle Cell Anemia
Sickle Cell Disease and Endothelial Progenitor Cells (EPCs)
Sickle Cell Disease (SCD) is the most prevalent genetic disease of haemoglobin.The underlying abnormality in the red blood cell (RBC) of SCD is the presence of abnormal sickle cell hemoglobin (HbS), which, when deoxygenated…
- Trial name
- DREPANOPEC (NCT00783627)
- Testing
- Angiography
- Where
- France
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Anemia, Sickle Cell, Hematologic Diseases
Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG)
To determine whether hydroxyurea prevents the onset of chronic end organ damage in young children with sickle cell anemia.
- Conditions
- Anemia, Sickle Cell, Hematologic Diseases, Hemoglobinopathies
- Trial name
- NCT00000602
- Testing
- hydroxyurea
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
Sickle Cell Disease, Pregnancy, High Risk
TAPS2 Transfusion Antenatally in Pregnant Women With SCD
Sickle Cell Disease (SCD) is a serious inherited blood disorder affecting red blood cells. When oxygen levels drop the red cells become abnormally shaped and unable to move through the blood vessels easily.
- Conditions
- Sickle Cell Disease, Pregnancy, High Risk, Blood Transfusion Complication
- Trial name
- TAPS2 (NCT03975894)
- Testing
- Serial prophylactic exchange blood transfusion (SPEBT).
- Where
- 6 sites in United Kingdom
- Volunteers
- 50 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Sickle Cell Disease, Sickle Cell Anemia in Children
Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease
The goal of this interventional study is to learn about the impact of an intervention for health care providers that teaches individuation and perspective-taking (IPT) skills to enhance patient-centered communication in pediatric…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Disease (SCD), Sickle Cell
- Trial name
- NCT07540767
- Testing
- Individuation and Perspective-Taking (IPT), pediatric SCD pain management education session
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Connecticut Children's Medical Center