Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Hydroxyurea and Transfusion
This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions.
- Trial name
- HAT (NCT03644953)
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Children's National Research Institute
Sickle Cell Disease, Acute Chest Syndrome
A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease
Acute chest syndrome (ACS), a lung complication in sickle cell disease (SCD), is the second most common cause of hospitalization and leading cause of death in SCD.
- Trial name
- NCT02630394
- Testing
- Azithromycin
- Where
- 2 sites in United States
- Sponsor
- University of Mississippi Medical Center
Sickle Cell Disease
Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab.
- Trial name
- NCT05833022
- Where
- 11 sites in United States
- Volunteers
- 297 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Sickle Cell Trait
Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
This study will determine the best ways to collect, process and store umbilical cord blood from babies with sickle cell disease, sickle cell trait and unaffected babies.
- Trial name
- NCT00012545
- Where
- United States
- Volunteers
- 307 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics.
- Trial name
- NCT05725902
- Testing
- Etavopivat
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Novo Nordisk A/S
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Study of Crizanlizumab for Prevention of Silent Cerebral Infarcts in SCA
In this prospective, single-arm, open-label, imaging and treatment study, the investigator will test the hypothesis that crizanlizumab will prevent the progression of silent cerebral infarcts in patients with sickle cell disease.
- Trial name
- CRIZ (NCT05334576)
- Testing
- Crizanlizumab
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Andria Ford
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 Disease, Sickle Cell Crisis
Lidocaine Intravenous in the Emergency Department For Sickle Cell Crisis
Sickle cell crisis continues to be a frequent presentation to emergency departments. Patients presenting will often require immediate treatment for their pain and often times this will include opioids.
- Conditions
- Sickle Cell Disease, Sickle Cell Crisis, Pain, Acute
- Trial name
- RELIEF-SCC (NCT04614610)
- Testing
- Lidocaine Iv
- Where
- 3 sites in United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Community Medical Center, Toms River, NJ
Sickle Cell Anemia in Children
Effectiveness of Innovative Educational Methods in Empowering Children With Sickle Cell Disease
Sickle-cell disease (SCD) is a genetic disorder that affects hemoglobin, the molecule in red blood cells responsible for carrying oxygen.
- Trial name
- NCT07802210
- Testing
- video intervention, game intervention
- Where
- Iraq
- Volunteers
- 120 people
- Sponsor
- Aula Ibrahim abd alrazak
Sickle Cell Disease
ElectroNic Hydroxyurea AdhereNCE: A Strategy to Improve Hydroxyurea Adherence in Patients With Sickle Cell Disease
HU is an FDA approved medication for the treatment of SCD. Many studies have shown that HU can reduce SCD related symptoms, but only 50% of patients take it as often as they should.
- Trial name
- ENHANCE (NCT02578017)
- Testing
- Mobile DOT
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- Nationwide Children's Hospital
Sickle Cell Disease
Quantitative MRI for Patients With Sickle Cell Disease Undergoing Hematopoietic Cell Transplant
The primary purpose of this research is to determine if it is feasible to perform serial magnetic resonance imaging (MRI) to evaluate the amount of bone marrow cells (also called cellularity) and iron stores before and after bone…
- Trial name
- NCT03458897
- Testing
- serial functional magnetic resonance imaging (MRI)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of Michigan
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
mAnaging siCkle CELl disEase Through incReased AdopTion of hydroxyurEa in Nigeria
Large knowledge gaps remain regarding strategies to promote the adoption of hydroxyurea (HU), particularly in sub-Saharan African countries including Nigeria, where more than 75% of annual sickle cell anemia births occur.
- Trial name
- ACCELERATE (NCT06318143)
- Testing
- TASSH, SIM
- Where
- Nigeria
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- New York University
Sickle Cell Disease
Acupuncture for Pain in Sickle Cell Disease
Sickle cell disease (SCD) is the most common genetic disorder in the United States affecting approximately 100,000 individuals primarily of African ancestry. Pain is the most common complication of SCD.
- Trial name
- NCT04122378
- Testing
- acupuncture
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Deepika Darbari
Sickle Cell Disease
Zinc for Infection Prevention in Sickle Cell Anemia-2 (ZIPS-2)
A randomized double-blinded placebo-controlled trial of zinc to reduce the incidence of severe or invasive infections in Ugandan children with sickle cell anemia (SCA)
- Trial name
- ZIPS-2 (NCT06561061)
- Testing
- zinc sulfate
- Where
- Uganda
- Volunteers
- 100 people
- Sponsor
- Indiana University
Sickle Cell Disease
Evaluation of Non-invasive Endothelial Function in Children Sickle by Vascular Ultrasound
Sickle cell disease (SCD) is an inherited disorder characterized by recurrent painful crises with ischemia resulting from vascular occlusion.
- Trial name
- NCT02829580
- Testing
- Echocardiography
- Where
- France
- Volunteers
- 31 people
- Sponsor
- University Hospital, Toulouse
Beta Thalassemia Intermedia, Sickle Cell Disease
The BENeFiTS Trial in Beta Thalassemia Intermedia
Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases…
- Trial name
- PB04-001 (NCT04432623)
- Testing
- Benserazide Only Product
- Where
- 5 sites in United States, Canada
- Volunteers
- 36 people
- Sponsor
- Phoenicia BioScience
Sickle Cell Disease
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell…
- Trial name
- SUN-RAY (NCT06358638)
- Testing
- Daratumumab, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's National Research Institute
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