Clinical trials: Sickle Cell Disorders
Stroke Ischemic, Sickle Cell Disease
Evaluating Barriers to Stroke Screening and Prevention in Children With Sickle Cell Disease
DISPLACE is a three part, multi-center U.S. based study to evaluate the barriers to stroke screening and prevention in children with sickle cell anemia (SCA).
- Trial name
- DISPLACE (NCT03621826)
- Testing
- Questionnaire/Interview
- Where
- United States
- Volunteers
- 5,247 people
- Sponsor
- Medical University of South Carolina
Sickle Cell Disease, Priapism
Sildenafil for Treatment of Priapism in Men With Sickle Cell Anemia
This research is being done to evaluate if the phosphodiesterase type 5 (PDE5) inhibitor sildenafil has an effect on the frequency of recurrent priapism and the quality of life of males with sickle cell disease (SCD).
- Trial name
- NCT00940901
- Testing
- sildenafil
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Johns Hopkins University
Rheologic Disease, Sickle Cell Disease
Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of…
- Conditions
- Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT01294891
- Testing
- Imaging
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Sickle Cell Retinopathy, Sickle Cell Disease
The Role of Red Cell Characteristics, Angiogenesis, Viscosity and Oxygenation in the Pathophysiology of Sickle Cell Related Retinopathy
Objective: to gain insight in the pathogenesis, to identify biomarkers to recognize patients at risk for proliferative SCR and to investigate its associations with clinical and laboratory characteristics.
- Conditions
- Sickle Cell Retinopathy, Sickle Cell Disease, Sickle Cell SC Disease, Sickle Cell-SS Disease
- Trial name
- RAVOS (NCT06396403)
- Testing
- Collection of venous blood samples
- Where
- Netherlands
- Volunteers
- 68 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Sickle Cell Anemia
The Role of Endothelin-1 in Sickle Cell Disease
The primary goal of the study is to determine the safety and tolerability of ambrisentan. It is also expected that ambrisentan will improve blood flow in the lungs, decrease inflammation, and reduce pain in sickle cell patients.
- Trial name
- NCT02712346
- Testing
- Ambrisentan
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Augusta University
Sickle Cell Disease
Effect of Simvastatin Treatment on Vaso-occlusive Pain in Sickle Cell Disease
The purpose of this study is to determine whether simvastatin is effective in reducing the frequency and intensity of vaso-occlusive pain episodes in patients with sickle cell disease.
- Trial name
- NCT01702246
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell, PKLR Variants
Genotype -Phenotype Correlation of PKLR Variants With Pyruvate Kinase, 2,3-Diphosphglycerate and Adenosine Triphosphate Activities in Red Blood Cells of People With Sickle Cell Disease
Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms.
- Conditions
- Sickle Cell, PKLR Variants, Adenosine Triphosphate Activities
- Trial name
- NCT03685721
- Where
- United States
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Sickle Cell Abnormality
Steroid Injection for Sickle Cell Arthropathy Pain Therapy
Sickle cell disease (SCD) is associated with arthropathy. Arthropathy may require periarticular corticosteroid injection therapy. This observational study examines efficacy, and safety of steroid injections in SCD patients.
- Trial name
- NCT05983055
- Testing
- Dexamethasone 20mg
- Where
- Canada
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Salem Anaesthesia Pain Clinic
Sickle Cell Disease, Sleep Disturbance
Sleep and Pain in Sickle Cell Disease
This is a study testing the effects of behavioral sleep interventions on pain and brain function in sickle cell disease.
- Conditions
- Sickle Cell Disease, Sleep Disturbance, Pain
- Trial name
- NCT03150433
- Testing
- Behavioral symptom management, Sickle cell disease management
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Johns Hopkins University
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
Sickle Cell Disease, Central Venous Catheter Thrombosis
THromboprophylaxis In Sickle Cell Disease With Central Venous Catheters (THIS)
Research Question: In adult SCD patients with CVC, is it feasible and safe to conduct an adequately powered RCT to evaluate the use of rivaroxaban as thromboprophylaxis in this population? Study Design: The study is a vanguard…
- Conditions
- Sickle Cell Disease, Central Venous Catheter Thrombosis, Venous Thromboembolism
- Trial name
- THIS (NCT05033314)
- Testing
- Rivaroxaban 10 MG
- Where
- 3 sites in Canada
- Volunteers
- 4 people
- Sponsor
- University Health Network, Toronto
Sickle Cells Disease
Vitamin D and Bisphosphonates in the Treatment of Sickle Cell Disease
Sickle cell disease (SCD) is a worldwide distributed hereditary red cell disorder, which affects approximately 75,000 individuals in the United States and almost 20,000- 25,000 subjects in Europe, this latter mainly related to…
- Trial name
- NCT02972138
- Where
- 3 sites in Italy
- Volunteers
- 80 people
- Sponsor
- Società Italiana Talassemie ed Emoglobinopatie
Sickle Cell Disease
Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own…
- Trial name
- GRASP-EA (NCT07640815)
- Testing
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Where
- United States
- Sponsor
- David Williams
Sickle Cell Disease, Sickle Cell Anemia
A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy
This trial is a follow-up companion study to Protocol ICA-17043-10, a Phase III, multi-center, efficacy and safety study of ICA-17043.
- Trial name
- NCT00294541
- Testing
- ICA-17043
- Where
- 31 sites in United States
- Volunteers
- 51 people
- Sponsor
- Icagen
Sickle Cell Disease
Trial of Zileuton CR in Children and Adults With Sickle Cell Disease
The purpose of this research study is to test the safety of Zileuton and see what effects (good and bad) it has on you, other children and adults with Sickle Cell Disease (SCD).
- Trial name
- Zileuton (NCT01136941)
- Testing
- Zileuton
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Priapism
A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism
The goal of the study was to evaluate the efficacy and safety of crizanlizumab in sickle cell disease (SCD) patients with priapism.
- Trial name
- SPARTAN (NCT03938454)
- Testing
- Crizanlizumab
- Where
- 13 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Transfusion-dependent Hemachromatosis, Thalassemia Major
Pilot Study for Patients With Poor Response to Deferasirox
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron…
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Trial name
- NCT00749515
- Testing
- Deferoxamine, Deferasirox, HIDA
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease, Measles Vaccination
Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease
The goal of this study is to learn if infants with sickle cell disease (SCD) develop adequate protection after measles vaccines. (not looking at any prolonged duration)
- Conditions
- Sickle Cell Disease, Measles Vaccination, Sickle Cell Anemia
- Trial name
- VALID (NCT07356050)
- Where
- 3 sites in United States, Ghana and 1 more
- Volunteers
- 130 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Genotype, Pain
Genetics and Pain Severity in Sickle Cell Disease
Background: Pain is the most common symptom of sickle cell disease. Episodes of severe sickle cell pain are known as "crises." High rates of pain crises are associated with a higher risk of early death.
- Conditions
- Genotype, Pain, Genetic Variation, Quantitative Sensory Testing (QST)
- Trial name
- NCT01441141
- Where
- United States
- Volunteers
- 67 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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