Clinical trials: Sickle Cell Disorders
Sickle Cell Disease
A Study of SHP655 (rADAMTS13) in Sickle Cell Disease
TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants.
- Trial name
- RAISE (NCT03997760)
- Testing
- TAK-755
- Where
- 14 sites in United States
- Volunteers
- 19 people
- Sponsor
- Shire
Heart Failure, Coronary Artery Disease
COVID-19 Testing in Underserved and Vulnerable Populations
As part of National Institutes of Health Rapid Acceleration of Diagnostics-Underserved Populations (RADx-UP) program, the goal of the RADxUP study is to develop, test, and evaluate a rapid, scalable capacity building project to…
- Conditions
- Heart Failure, Coronary Artery Disease, Cancer, Chronic Kidney Diseases
- Trial name
- NCT05315908
- Testing
- Community outreach method
- Where
- United States
- Volunteers
- 9,120 people
- Sponsor
- Jesse Nodora
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
Anemia, Sickle Cell
Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the…
- Trial name
- NCT01054768
- Testing
- alpha-lipoic acid and acetyl-L-carnitine, Control
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Pulmonary Hypertension, Sickle Cell Anemia
Secondary Pulmonary Hypertension in Adults With Sickle Cell Anemia
The purpose of this study is to determine how often people with sickle cell anemia develop pulmonary hypertension a serious disease in which blood pressure in the artery to the lungs is elevated.
- Conditions
- Pulmonary Hypertension, Sickle Cell Anemia, Sickle Cell Disease
- Trial name
- NCT00011648
- Where
- 2 sites in United States
- Volunteers
- 986 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Anemia, Sickle Cell
Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis
- Trial name
- NCT02411708
- Testing
- SANGUINATE
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Prolong Pharmaceuticals
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Anemia, Sickle Cell, Hematologic Diseases
Penicillin Prophylaxis in Sickle Cell Disease (PROPS)
To determine whether the regular daily administration of oral penicillin would reduce the incidence of documented infection due to Streptococcus pneumoniae in children with sickle cell anemia.
- Conditions
- Anemia, Sickle Cell, Hematologic Diseases, Hemoglobinopathies, Infection (S. Pneumoniae)
- Trial name
- NCT00000585
- Testing
- penicillin
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease (SCD)
Early Diagnosis of Sickle Acute Chest Syndrome Using a Combination of Plasma Bimarkers and Chest Imaging
Background: Painful vasoocclusive crisis (VOC) occurs in people with sickle cell disease (SCD). People with VOC have many visits to the hospital.
- Trial name
- NCT03478917
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Monocytic Expression of Heme Oxidase-1 (HO-1) in Sickle Cell Patients and Correlation With the Humoral Immune Response to Vaccine and With Allo-immunization.
Sickle cell disease (SCD) is an autosomal recessive disorder resulting from a substitution in the β chain of hemoglobin (Hb) which causes hemoglobin S to polymerize when deoxygenated.
- Trial name
- NCT03111589
- Testing
- Inactivated influenza A (H1N1) virus vaccine, Blood sampling
- Where
- 2 sites in Belgium
- Volunteers
- 102 people
- Sponsor
- Francis Corazza
Sickle Cell Disease
Assessing Physical Function in Sickle Cell Patients Taking Voxelotor
Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels.
- Trial name
- NCT06023199
- Testing
- Voxelotor
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Inova Health Care Services
Sickle Cell Disease
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
Sickle Cell Disease
Age of Blood in Sickle Cell Transfusion
The Investigators hypothesize that older red cell units trigger phagocytosis and activation of circulating macrophages with a downstream immunomodulatory cascade and release of excess Non Transferrin Bound Iron(NTBI) that leads…
- Trial name
- NCT03704922
- Testing
- Transfusion
- Where
- 3 sites in United States
- Volunteers
- 26 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
Realizing Effectiveness Across Continents With Hydroxyurea (REACH)
REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for for pediatric patients with sickle cell anemia (SCA).
- Trial name
- REACH (NCT01966731)
- Testing
- Hydroxyurea
- Where
- 4 sites in Angola, Democratic Republic of the Congo and 2 more
- Volunteers
- 635 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Kidney Injury
Acute Kidney Injury in Patients With Sickle Cell Disease
Patients with sickle cell disease may be at risk for acute kidney injury (AKI)during sickle cell crisis (pain or acute chest syndrome).
- Conditions
- Sickle Cell Disease, Kidney Injury, Kidney Diseases, Kidney Disease, Chronic
- Trial name
- NCT03105271
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease and Pregnancy
HEMAGO Monitoring and Impact on the Occurrence of Complications During Pregnancy in Sickle Cell Patients
Sickle cell disease is the most common monosemic genetic disease in the world and affects approximately 32,000 people in France.
- Trial name
- HEMAGO (NCT06620887)
- Testing
- Evaluate the impact of HEMAGO program on maternal morbidity and mortality in sickle cell disease patients during their pregnancies.
- Where
- 3 sites in France
- Volunteers
- 100 people
- Sponsor
- Hospices Civils de Lyon
Sickle Cell Disease
A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease
- Trial name
- Hestia2 (NCT02482298)
- Testing
- Ticagrelor
- Where
- 20 sites in United States, Egypt and 6 more
- Volunteers
- 87 people
- Sponsor
- AstraZeneca
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
Anemia, Sickle Cell, Sickle Cell Disease
Epidemiology of Silent and Overt Strokes in Sickle Cell Disease
Sickle Cell Disease (SCD) is a rare disease occurring in an estimated 100,000 individuals, often poor and underserved, in the US.
- Conditions
- Anemia, Sickle Cell, Sickle Cell Disease, Stroke, Sickle Cell Thalassemia
- Trial name
- ESCD (NCT03376893)
- Where
- 4 sites in United States
- Volunteers
- 102 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in participants with SCD.
- Trial name
- NCT06924970
- Testing
- Tebapivat
- Where
- 20 sites in United States, Belgium and 5 more
- Volunteers
- 59 people
- Sponsor
- Agios Pharmaceuticals, Inc.
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