Clinical trials: Sickle Cell Anaemia
Sickle Cell Disease
Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease
The purpose of this study is to use cardiac magnetic resonance imaging (CMR) and echocardiographic tissue Doppler imaging to demonstrate a unique restrictive cardiomyopathy of sickle cell disease.
- Trial name
- NCT02410811
- Testing
- Cardiac magnetic resonance imaging (CMR)
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Graft Versus Host Disease
Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease
To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving…
- Trial name
- NCT02867800
- Testing
- Diphenhydramine, Acetaminophen, Methylprednisolone
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Monica Bhatia
Beta-Thalassemia, Thalassemia
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD).
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn
- Trial name
- NCT05477563
- Testing
- CTX001
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Anemia in Children
Low Systemic/High Local Exercise Load in Peds SCD
This research study wants to learn about what kind of exercise is best for kids with sickle cell disease. Participating children will have a small amount of blood drawn one time at the beginning of the study.
- Trial name
- NCT06387758
- Testing
- Low systemic strength training, Moderate systemic exercise
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Mississippi Medical Center
Blood Disease, Anemia, Sickle Cell
Transcranial Doppler (TCD) Ultrasound of Subjects Enrolled in BABY HUG - Ancillary to BABY HUG
To use transcranial Doppler (TCD) ultrasound to detect stroke risk in children with sickle cell disease.
- Conditions
- Blood Disease, Anemia, Sickle Cell, Cerebrovascular Disorders, Cerebral Embolism and Thrombosis
- Trial name
- NCT00059293
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Simvastatin (Zocor) Therapy in Sickle Cell Disease
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium.
- Trial name
- NCT00508027
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
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
Sickle Cell Disease
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD).
- Trial name
- NCT04293185
- Testing
- bb1111
- Where
- 9 sites in United States
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
Hemoglobinopathies
Screening for Hemoglobinopathies in Pregnant Women
This prospective monocentric study project is to identify hemoglobinopathies in pregnant women in order to optimize antenatal care and to investigate the prevalence of hemoglobinopathies in pregnant women in Switzerland.
- Trial name
- NCT04029142
- Testing
- Review of routine lab data
- Where
- Switzerland
- Volunteers
- 1,785 people, healthy volunteers welcome
- Sponsor
- University Hospital, Basel, Switzerland
Anemia, Sickle Cell
Dipyridamole/Magnesium To Improve Sickle Cell Hydration
The purpose of this study is to determine the benefits as well as side effects of giving drugs called dipyridamole and magnesium to patients with sickle cell anemia (SCA).
- Trial name
- NCT00276146
- Testing
- oral dipyridamole, oral magnesium, or a combination of both
- Where
- 3 sites in United States
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hemoglobin SC Disease, Sickle Cell Anemia
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders
Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation…
- Trial name
- NCT00005783
- Testing
- Recombinant-methionyl human stem cell factor
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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, Hemolytic Anemia
Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.
- Trial name
- NCT07566494
- Testing
- VAS-101
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes.
- Trial name
- SCURT (NCT00745420)
- Testing
- Hematopoietic Stem Cell Transplantation
- Where
- 19 sites in United States
- Volunteers
- 30 people
- Sponsor
- Medical College of Wisconsin
Anemia, Sickle Cell, Blood Disease
Investigation of Selected Patient Groups From The Cooperative Study of Sickle Cell Disease
To continue to follow the newborn cohort and the over-35 years of age cohort from the Cooperative Study of Sickle Cell Disease (CSSCD), a study of the natural history of sickle cell disease.
- Trial name
- NCT00005300
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor bone marrow transplant helps stop the growth of abnormal cells.
- Trial name
- NCT00489281
- Testing
- Cyclophosphamide, Fludarabine, Mycophenolate mofetil
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Healthy, Sickle Cell Disease
Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease
The purpose of the study is to determine whether imaging techniques, such as magnetic resonance imaging (MRI), near infrared spectroscopy (NIRS), laser speckle contrast imaging (LSCI), and optical imaging (OI), can detect…
- Trial name
- NCT02447627
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Bioverativ Therapeutics Inc.
Sickle Cell Disease
Pilot Evaluation of a Motivational Interviewing Intervention Targeting Adherence Behaviors in Youth With Sickle Cell Disease
Sickle cell disease (SCD) is a group of inherited blood disorders affecting 100,000 individuals in the United States. SCD often leads to complications, including pain crises and organ damage.
- Trial name
- NCT04877054
- Testing
- Adherence Treatment Program, Education only
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins All Children's Hospital
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