Clinical trials: Sickle Cell Disorders
Vaso-occlusive Crisis
Music Therapy in Sickle Cell Disease Vaso-occlusive Crisis
Treatment of painful vaso-occlusive crises, the most common manifestation of sickle cell disease, is notoriously limited. vaso-occlusive crises pain is multifactorial with a psychological component.
- Trial name
- MUSICODREP (NCT03639805)
- Testing
- Music therapy program MUSIC CARE® + standard care, standard care of vaso-occlusive crises
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Thalassemia
Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere
Primary Objective 1. Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years) 2. Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18…
- Trial name
- NCT03825341
- Testing
- Hydroxyurea, Hydroxyurea Oral Capsule
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Jude Children's Research Hospital
Hemoglobinopathies
A Clinical Exploratory Study of YOLT-204 in the Treatment of Hemoglobinopathies
This is a single-arm, open-label, dose-escalation study designed to enroll approximately 5-30 patients with TDT or SCD. The objectives are to evaluate the safety and tolerability in patients following intravenous administration…
- Trial name
- NCT07774351
- Testing
- YOLT-204
- Volunteers
- 30 people
- Sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Sickle Cell Disease
Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
The use of analgesics can lead to cases of drug abuse and dependence. It can also cause pseudo-addiction in patients suffering from pain.
- Trial name
- PHEDRE (NCT02580565)
- Testing
- telephone interview
- Where
- France
- Volunteers
- 1,004 people
- Sponsor
- Nantes University Hospital
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
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
- Trial name
- NCT01090323
- Testing
- ICL670
- Where
- 39 sites in United States, Canada and 3 more
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Chronic Pain
Predictors of Pain in Sickle Cell Disease
Sickle cell disease is a painful inherited disorder that affects approximately 100,000 people in the United States, and more than half of these individuals develop chronic or persistent pain that is often severe and disabling.
- Trial name
- NCT06139510
- Testing
- Quantitative sensory testing
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Duke University
Chronic Cutaneous Ulcer, Sickle Cell Disease
Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment
Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).
- Trial name
- D-SCOUT (NCT04058197)
- Testing
- Deferoxamine Product
- Where
- 4 sites in United States
- Sponsor
- TauTona Group
Sickle Cell Disease, Vaso-occlusive Crisis
A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease
This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT05348915
- Testing
- Inclacumab
- Where
- 50 sites in United States, Brazil and 8 more
- Volunteers
- 242 people
- Sponsor
- Pfizer
Sickle Cell Anaemia
Prevention Of Morbidity In Sickle Cell Disease Pilot Phase
The hypothesis is that in sickle cell anaemia, nocturnal oxyhaemoglobin desaturation, is associated with low processing speed index, and this morbidity can be reduced with overnight auto Continuous Positive Airways Pressure…
- Trial name
- NCT00415727
- Testing
- auto Continuous Positive Airways Pressure (CPAP) with oxygen supplementation
- Where
- 2 sites in United Kingdom
- Volunteers
- 22 people
- Sponsor
- Institute of Child Health
Sickle Cell Disease
Survey in a Population of Sickle Cell Disease Patients to Evaluate the Transition Between the Queen Fabiola Children Hospital and the CHU Brugmann Hospital, and the Quality of the Hospital Care Within the CHU Brugmann Hospital.
Sickle cell disease is a genetic disease responsible for an abnormal hemoglobin.The anomaly has several consequences: a low hemoglobin rate (chronic anemia), plugs formed by red blood cells in blood vessels (extremely painful…
- Trial name
- NCT02608580
- Testing
- Survey
- Where
- Belgium
- Volunteers
- 30 people
- Sponsor
- Brugmann University Hospital
Hemoglobin SC Disease, Anemia, Sickle Cell
Parent Educational Program for Children With Sickle Cell Disease
Children with sickle cell disease (SCD) are at risk for central nervous system (CNS) complications, which may affect academic achievement.
- Trial name
- NCT00860782
- Testing
- Parent Educational Support
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- University of Miami
Pulmonary Hypertension
Anticoagulation For Pulmonary Hypertension in Sickle Cell Disease
Sickle cell disease (SCD) is often referred to as a hypercoagulable state. However, the contribution of coagulation activation to the pathogenesis of SCD remains uncertain.
- Trial name
- NCT01036802
- Testing
- Warfarin
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of North Carolina, Chapel Hill
Genetic Disease, Genomics
Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study)
Background: People with sickle cell disease and other blood disorders sometimes get chronic leg ulcers. These are wounds that develop on the skin and don t go away.
- Trial name
- NCT02156102
- Where
- 3 sites in United States, Sierra Leone
- Volunteers
- 405 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Other Sickle-cell Disorders With Crisis, Unspecified
Quality of Life Study for Sickle Cell Patients Treated With Jobelyn (Sorghum Bicolor Extract)
The purpose of this study is to determine the antioxidant effect of prolonged use of sorghum bicolor (jobelyn) to increase the level of plasma superoxide dismutase and glutathione reductase in patients with sickle cell disease…
- Trial name
- NCT01704794
- Testing
- Folic Acid + Paludrine + Jobelyn (500mg), Folic Acid + Paludrine + Jobelyn (250mg), Folic Acid + Paludrine + Jobelyn (2mg)
- Where
- Nigeria
- Volunteers
- 96 people
- Sponsor
- Lagos State University
Sickle Cell Disease
Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
Children with sickle cell disease (SCD) are at risk for neurobehavioral problems because of the impact the disease can have on the central nervous system.
- Trial name
- NCT02227472
- Testing
- Evaluation of cognitive and pre-academic skills, Parent questionnaires
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Nano-rheological Biomarkers for Patients With Sickle Cell Disease (SCD) Versus Control Subjects (Other Constitutional Red Blood Cell Diseases and Healthy Subjects)
Numerous pathologies (sickle cell disease, thalassemia, spherocytosis, etc.) lead to changes in the rheological properties of the blood, in particular via alterations in the deformability of red blood cells.
- Trial name
- DREPNANO (NCT05530239)
- Testing
- Blood sample collection
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
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
Assessing Function in Pediatric Patients With Sickle Cell Disease
The purpose of this study is to evaluate the FIM™ as a measure of daily function in children with sickle cell disease hospitalized with vasoocclusive pain.
- Trial name
- NCT00590148
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- Connecticut Children's Medical Center
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