Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Pilot Study on the Value of Bedside Pleuropulmonary Ultrasound in Patients With Sickle Cell Disease Presenting With Vaso-occlusive Crisis
Describe the proportion of patients with major sickle cell syndrome in vaso-occlusive crisis presenting at least one pleuropulmonary ultrasound abnormality during one of the ultrasounds performed at D0, between D2 and D5 during…
- Trial name
- DREPP (NCT06755385)
- Testing
- Pleuropulmonary ultrasound
- Volunteers
- 25 people
- Sponsor
- University Hospital, Grenoble
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, Chronic Pain
Use of a Mobile-based App for SCD Patients
The study will seek to enroll 100 sickle cell or thalassemia patients who are age 12 or older who have access to a smartphone or tablet with Internet access daily.
- Trial name
- SMART (NCT01833702)
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Duke University
Sickle Cell Disease
Study of Blood Platelets in Sickle Cell Disease
We are studying if sickle cell disease blood platelets are larger than normal and how they may cause obstruction of blood vessels.
- Trial name
- NCT00226083
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Rockefeller University
Sickle Cell Disease
Sleep Study in Adult Patients With Major Sickle Cell Disease With Paroxysmal Nocturnal Events
Hypothesis is that the occurrence of nocturnal Vaso-Occlusive Crisis (VOC) and priapism in adults might be related to episodes of nocturnal desaturation secondary to a sleep apnea syndrome.
- Trial name
- DREPASOM (NCT02539771)
- Testing
- Polysomnography and blood sample
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Sickle Cell Anemia
Carbon Monoxide Levels and Sickle Cell Disease Severity
Background: Some people with sickle cell disease have different health problems than others. This may be related to how easily and frequently the red blood cells break apart in the blood.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Anemia, Sickle Cell
- Trial name
- NCT01547793
- Where
- United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
A Safety Study of Eptifibatide in Patients With Sickle Cell Disease
This study will evaluate the safety of eptifibatide in sickle cell patients and how well it works during the course of painful crises.
- Trial name
- NCT00834899
- Testing
- Eptifibatide
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
Risk Factors for Allo-immunization in Sickle Cell Disease
Sickle cell patients have a high prevalence of alloimmunization. This high rate of alloimmunization can be partially explained by the existence of an antigenic difference between the predominantly Caucasian donor population and…
- Trial name
- NCT03401125
- Testing
- Medical file data collection
- Where
- 2 sites in Belgium
- Sponsor
- Hanane EL KENZ
Sickle Cell Trait, Environmental Exposure
Sickle Cell Trait and Exercise, Effect of Hot Environment
The heterozygous form of sickle cell disease is clinically asymptomatic. Nevertheless, it was observed that, the sickle cell trait is associated with serious medical complications especially during intense physical efforts.
- Conditions
- Sickle Cell Trait, Environmental Exposure, Adverse Effect
- Trial name
- TDex (NCT04028791)
- Testing
- Exercise on ergocycle
- Where
- Guadeloupe
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- University of the French West Indies and French Guiana
Anemia, Sickle Cell
Gene Transfer for Patients With Sickle Cell Disease
The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease
- Trial name
- NCT02186418
- Testing
- ARU-1801
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care
Background: Sickle cell disease (SCD) is caused by a genetic defect that affects how hemoglobin is made. Due to this, people with SCD have abnormally-shaped red blood cells, which can result in poor oxygen transport in the body…
- Trial name
- NCT03167450
- Where
- United States
- Volunteers
- 109 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Sickle Cell Disease
Hydroxyurea Optimisation in Sickle Cell Disease
The wide interindividual variability in clinical response to hydroxyurea therapy in the management of sickle cell disease has limited its use.
- Trial name
- PHOENIX-NG (NCT07543289)
- Testing
- Hydroxyurea (HU)
- Where
- Nigeria
- Volunteers
- 100 people
- Sponsor
- Ochuko Orherhe
Sickle Cell Disease (SCD)
Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire
The goal of this clinical trial is to learn if a multifaceted intervention composed of Gazelle-Multispectral sickle cell disease (SCD) point of care testing (POCT) and early initiation of comprehensive SCD care in children with…
- Trial name
- STEP (NCT07488520)
- Testing
- Gazelle-Multispectral point of care testing for newborn sickle cell disease screening; and initiation of early comprehensive clinical interventions
- Where
- 2 sites in Côte d’Ivoire
- Volunteers
- 120 people
- Sponsor
- Swiss Tropical & Public Health Institute
Vaso-occlusive Crisis, Sickle Cell Anemia Crisis
Hyperbaric Oxygen Therapy in Sickle Cell Pain
Sickle cell anemia is a genetic blood disorder where red blood cells are abnormally shaped, crescent- or sickle-shaped, instead of the normal, round shape.
- Trial name
- HAVOC (NCT03412045)
- Testing
- hyperbaric oxygen therapy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Nebraska
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 Disease, Hemoglobinopathies
Non-Myeloablative Conditioning and Bone Marrow Transplantation
Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia.
- Trial name
- NCT01850108
- Testing
- Thymoglobulin, Fludarabine, Cyclophosphamide (CTX)
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Vanderbilt-Ingram Cancer Center
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
Sickle Cell Disease, Sickle Cell Anemia
Actigraphy Improvement With Voxelotor (ActIVe) Study
This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia.
- Trial name
- ActIVe (NCT04400487)
- Testing
- Voxelotor
- Where
- 10 sites in United States
- Volunteers
- 25 people
- Sponsor
- Pfizer
Sickle Cell Disease
Gerofit Exercise Intervention for Older Adults With Sickle Cell Disease (SICKLE-FIT Study)
The purpose of this study to assess the feasibility, acceptability, and safety of a personalized exercise training program adapted from Gerofit to improve physical health and quality of life for adults with SCD
- Trial name
- SICKLE-FIT (NCT05714098)
- Testing
- Gerofit Exercise Program
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Duke University
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