Clinical trials: Sickle Cell Disorder
Sickle Cell Disease, Hyperbaric Oxygen Therapy
Hyperbaric-oxygen Therapy (HBOT) Versus Placebo for Treating Vaso-Occlusive Crisis (VOC) in Sickle Cell Disease (SCD)
This is a randomised, controlled, double-blind, placebo trial of HBOT (intervention) superiority in the treatment of VOC in SCD, to demonstrate the effectiveness of HBOT for the decrease in pain level in the treatment of SCD-VOC.
- Conditions
- Sickle Cell Disease, Hyperbaric Oxygen Therapy, Vaso-occlusive Crisis
- Trial name
- HBOT-SCD (NCT05289700)
- Testing
- HBOT in Hyperbaric chamber
- Where
- 3 sites in France, Switzerland
- Volunteers
- 100 people
- Sponsor
- University Hospital, Geneva
Sickle Cell Disease, Pregnancy Related
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of…
- Trial name
- ProTIP (NCT06979492)
- Testing
- Prophylactic Transfusion Intervention group: Transfusion, Control group
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Emory University
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Sickle Cell Disease, Thalassaemia
Integrative Diagnosis for SCD and Other RADs
INTEGRA aims at enabling personalized medicine for RHADs patients by the establishment of an integrative diagnostic approach based on deep phenotypic and genetic characterization through combining new generation methodologies.
- Conditions
- Sickle Cell Disease, Thalassaemia, Congenital Dyserythropoietic Anemia (CDA), Enzyme Disorder; Anemia
- Trial name
- INTEGRA (NCT07206095)
- Testing
- Analysis of genetic modifiers, Disease phenotyping
- Where
- 9 sites in Spain
- Volunteers
- 200 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Sickle Cell Disease, Venous Thromboembolism
Comparison of Thrombin Generation, Platelet Activation and Aggregation Profiles in Patients With Sickle Cell Disease Depending on Their SS or SC Genotype (DrepAgreg)
Sickle cell disease (SCD) is characterized by chronic hemolytic anemia, painful crises called vaso-occlusive crises (VOC) and chronic inflammation.
- Trial name
- DrepAgreg (NCT07852442)
- Where
- 2 sites in France, Guadeloupe
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Sickle Cell Disease, COVID-19 Vaccine
COVID-19 Vaccination Hesitancy in Adults With Sickle Cell Disease
The goal of this clinical trial is to test an COVID-19 vaccination information video in adults with sickle cell disease. The main questions it aims to answer are why are some adults with sickle cell disease hesitant to receive…
- Conditions
- Sickle Cell Disease, COVID-19 Vaccine, Vaccine Hesitancy
- Trial name
- NCT05998824
- Testing
- SCD-specific COVID-19 vaccination information (SCVI) video
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Duke University
Sickle Cell Disease
Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening
Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-long debilitation, evidence-based strategies to control SCD are not…
- Trial name
- SIMCS SCD (NCT06563440)
- Testing
- Digital app and information system
- Where
- 2 sites in United States, Uganda
- Volunteers
- 24,000 people, healthy volunteers welcome
- Sponsor
- Makerere University
Sickle Cell Disease
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to…
- Trial name
- NCT05031780
- Testing
- Mitapivat
- Where
- 91 sites in United States, Belgium and 14 more
- Volunteers
- 286 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pneumococcal Conjugate Vaccine
Study Evaluating 13-valent Pneumococcal Conjugate Vaccine (13vPnC) in Children With Sickle Cell Disease
This study is to evaluate the safety, tolerability and immunogenicity of 13-valent pneumococcal Conjugate Vaccine in children with Sickle Cell Disease who have already been vaccinated with 23-valent polysaccharide vaccine.
- Trial name
- NCT00918580
- Testing
- 13-valent Pneumoccocal Conjugate Vaccine
- Where
- 19 sites in United States, Egypt and 5 more
- Volunteers
- 158 people
- Sponsor
- Pfizer
Sickle Cell Disease
Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease
The purpose of this research study is to learn about ways to help children and adults with sickle cell disease who are taking the medication, hydroxyurea.
- Trial name
- SCD Mobile Dot (NCT02371720)
- Testing
- Mobile DOT
- Where
- 4 sites in United States
- Volunteers
- 164 people
- Sponsor
- Emory University
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
A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease
This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD.
- Trial name
- NCT02140554
- Testing
- lovo-cel
- Where
- 11 sites in United States
- Volunteers
- 54 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease, Pain
Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD
Pain is the primary complication of sickle cell disease (SCD), including vaso-occlusive crises and more persistent, chronic pain.
- Trial name
- NCT04388241
- Testing
- The Balance Program
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Children's National Research Institute
Sickle Cell Disease
Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease
Sickle cell anemia (SCA) is a life-threatening, monogenic disorder associated with early death when compared to individuals without SCA.
- Trial name
- NCT02960503
- Testing
- Azithromycin
- Where
- United States
- Sponsor
- Vanderbilt University
Sickle Cell Disease
Prevalence of Osteoporosis in Sickle Cell Disease
Sickle cell disease is the most common single-gene disease in the world. Its prevalence is increasing in France, with patients' life expectancy increasing into developed countries.
- Trial name
- DREPAN'OS (NCT04299594)
- Testing
- Questionnaire
- Where
- France
- Volunteers
- 142 people
- Sponsor
- Hospices Civils de Lyon
Sickle Cell Disease
Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial
The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.
- Trial name
- STArT (NCT04839354)
- Testing
- L-Arginine Hydrochloride, Normal saline
- Where
- 10 sites in United States
- Volunteers
- 271 people
- Sponsor
- Claudia R. Morris
Sickle Cell Disease, Kidney Diseases, Chronic
The Predictive Capacity of Machine Learning Models for Progressive Kidney Disease in Individuals With Sickle Cell Anemia
This is a multicenter prospective, longitudinal cohort study which will evaluate the predictive capacity of machine learning (ML) models for progression of CKD in eligible patients for a minimum of 12 months and potentially for…
- Trial name
- PREMIER (NCT05214105)
- Testing
- Biospecimen/DNA collection and analysis
- Where
- 3 sites in United States
- Volunteers
- 400 people
- Sponsor
- University of Tennessee
Anemia, Sickle Cell
Establishing a Database of People With Sickle Cell Disease (Comprehensive Sickle Cell Centers Collaborative Data Project [C-Data])
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes.
- Trial name
- C-Data (NCT00529061)
- Where
- 19 sites in United States
- Volunteers
- 3,640 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
BEATS 2: Music Therapy in Sickle Cell
The purpose of this study is to investigate the effects of the BEATS music therapy program on the self-efficacy, trust, knowledge, and adherence of young adult patients with SCD.
- Trial name
- NCT03233269
- Testing
- Music Therapy
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Disease
A Study to Learn About Sickle Cell Disease In Adult Patients
The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment.
- Trial name
- NCT05407805
- Testing
- Electronic Diary
- Where
- 4 sites in United States
- Volunteers
- 98 people
- Sponsor
- Pfizer
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