Clinical trials: Sickle Cell Disorders
Sickle Cell Disease, Sickle Cell Anemia in Children
PINPOINT: Gaming Technology for SCD Pain
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Thalassemia, Sickle Cell SC Disease
- Trial name
- Pinpoint II (NCT04579926)
- Testing
- Pinpoint app
- Where
- 2 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Sickle Cell Anemia
Study of Ventilatory Mechanics in Patients With Sickle Cell Anemia
The Sickle Cell Anemia (SCA) is a recessive genetic condition, monogenic, resulting in defects in the red cell structure. In the investigators' country, this disease affects about 3,000 children each year and is considered one of…
- Trial name
- SCA (NCT02565849)
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Rio de Janeiro State University
Sickle Cell Disease
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
- Trial name
- NCT04055818
- Testing
- Nicotinamide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- EpiDestiny, Inc.
Sickle Cell Disease, Cerebral Ischemia
Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia
The purpose of the present observational study is to remotely reevaluate the cohort of 67 sickle cell patients with transcranial Doppler-detected cerebral vasculopathy included in the national "Sickle Cell Transplant" protocol…
- Conditions
- Sickle Cell Disease, Cerebral Ischemia, Stenosis
- Trial name
- DREPAGREFFE2 (NCT05053932)
- Testing
- blood collection
- Where
- 14 sites in France
- Volunteers
- 67 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Sickle Cell Disease
Sickle Cell Disease, Hemechip
Sickle cell disease is very common in Nigeria. Early diagnosis is important to prevent or reduce serious complications from the disease and to enable children stay healthy.
- Trial name
- NCT03948516
- Where
- 7 sites in United States, Nigeria
- Volunteers
- 738 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
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
Sickle Cell Disease
TMLI and Alemtuzumab for Treatment of Sickle Cell Disease
This phase I trial tests the safety and effectiveness of total marrow and lymphoid irradiation (TMLI) and alemtuzumab as a conditioning regimen in patients with sickle cell disease.
- Trial name
- NCT05384756
- Testing
- Alemtuzumab, Hematopoietic Cell Transplantation, Intensity-Modulated Radiation Therapy
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- City of Hope Medical Center
Sickle Cell Disease
A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects
The purpose of this study is to evaluate the relative bioavailability of a single 300 mg dose of GBT440 administered as a high strength (1 × 300 mg) capsule versus a low strength (3 × 100 mg) capsule formulation in healthy fasted…
- Trial name
- NCT02567695
- Testing
- GBT440
- Where
- United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
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
Vaso-occlusive Pain Episodes
Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits.
- Trial name
- NCT01796678
- Testing
- Arginine
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell…
- Trial name
- NCT03653247
- Testing
- Plerixafor, Busulfan, BIVV003
- Where
- 5 sites in United States
- Volunteers
- 7 people
- Sponsor
- Sangamo Therapeutics
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
Sickle Cell Clinical Research and Intervention Program
Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation.
- Trial name
- NCT02098863
- Where
- 6 sites in United States
- Volunteers
- 10,000 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
A Study of Oral L-citrulline in Sickle Cell Disease
Sickle cell disease is a genetic red blood cell disorder that can result in blocking of the small blood vessels from sickle shaped red blood cells. This causes pain, the main feature of sickle cell disease.
- Trial name
- NCT02659644
- Testing
- Oral L-citrulline
- Where
- United States
- Sponsor
- University of Mississippi Medical Center
Sickle Cell Anemia
Establishment of Functional MRI Imaging Parameters for Use in the Evaluation of Sickle Cell Disease
Patients with sickle cell anemia (SCA) are at an increased risk for damage to brain tissue due to their disease. The investigators are interested in how blood flow and cerebral inflammation are different in SCA patients and how…
- Trial name
- NCT02445144
- Where
- 2 sites in United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Sickle Cell Disease
Study of Vitamin D in Children With Sickle Cell Disease
This pilot study aims to answer the question whether monthly oral vitamin D3 supplementation, 100,000 IU, will be safe and effective in raising serum 25-hydroxyvitamin D (form of vitamin D measured in the blood) to levels…
- Trial name
- NCT01276587
- Testing
- Vitamin D3
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Gary M Brittenham, MD
Sickle Cell Disease, Acute Chest Syndrome
Tocilizumab for Acute Chest Syndrome
The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease.
- Trial name
- NCT05640271
- Testing
- Tocilizumab
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- University of Chicago
Sickle Cell Disease
Implications of a Paediatrician-psychologist Tandem for Sickle Cell Disease Care and Impact on Cognitive Functioning
Sickle cell disease (SCD) necessitates a paediatric treatment plan that considers the influence of psychological, family and intercultural factors.
- Trial name
- NCT03975699
- Testing
- Assessment of psychological repercussions of SCD
- Volunteers
- 155 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Sickle Cell Disease
Microvessels and Heart Problems in Sickle Cell Disease
Background: Small blood vessels (microvessels) in many different organs are affected by diseases such as diabetes and atherosclerosis. These microvessels may also be abnormal in people who have sickle cell disease.
- Trial name
- NCT01602809
- Where
- United States
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Pulmonary Hypertension, Sickle Cell Disease
Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease…
- Conditions
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Trial name
- NCT01048905
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
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