Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06287099
- Testing
- BRL-101
- Volunteers
- 5 people
- Sponsor
- Bioray Laboratories
Sickle Cell Disease
Near Infrared Spectroscopy in Sickle Cell Pediatric Patients
Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease. Near infrared spectroscopy (NIRS) technology can measure tissue oxygenation and endothelial function.
- Trial name
- NCT04031521
- Testing
- Near infrared spectroscopy
- Where
- United States
- Sponsor
- Children's National Research Institute
Sickle Cell Disease
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.
The purpose of this study is to evaluate the Safety and Efficacy of DREAM01, a gene therapy for Sickle Cell Disease (SCD). The therapy consists of transplanting autologous CD34+ cells transduced ex vivo with a bifunctional…
- Trial name
- DREPAMIR (NCT07432867)
- Testing
- DREAM01 drug product, anti-inflammatory therapy
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Pulmonary Hypertension Associated With Sickle Cell Disease
Study to Evaluate Efficacy and Safety of Oral Treprostinil in Subjects With Pulmonary Hypertension (PH) Associated With Sickle Cell Disease (SCD)
This is a multicenter, randomized (2:1; oral treprostinil:placebo), double-blind, placebo-controlled event-driven (time to pulmonary hypertension [PH] clinical worsening) study in subjects with PH associated with sickle cell…
- Trial name
- NCT03055234
- Testing
- Oral Treprostinil
- Sponsor
- United Therapeutics
Sickle Cell Disease
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of…
- Trial name
- LIBRA (NCT06975865)
- Testing
- Rilzabrutinib
- Where
- 63 sites in United States, Belgium and 12 more
- Volunteers
- 192 people
- Sponsor
- Sanofi
Sickle Cell Disease
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice…
- Trial name
- KID-BID (NCT06578507)
- Testing
- Hydroxycarbamid
- Where
- 7 sites in France, French Guiana
- Volunteers
- 50 people
- Sponsor
- Theravia
Asthma, Atrial Fibrillation
Improving Quality by Maintaining Accurate Problems in the EHR
The overall goal of the IQ-MAPLE project is to improve the quality of care provided to patients with several heart, lung and blood conditions by facilitating more accurate and complete problem list documentation.
- Conditions
- Asthma, Atrial Fibrillation, Chronic Obstructive Pulmonary Disease, Coronary Artery Disease
- Trial name
- IQ-MAPLE (NCT02596087)
- Testing
- Problem List Suggestion
- Where
- 4 sites in United States
- Volunteers
- 2,386 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
Sickle Cell Disease
Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text Messaging
The investigators will explore barriers to improving self-efficacy, or the ability to feel in control of their disease, and medication adherence with text messaging through surveys and interviews with adolescents and adults with…
- Trial name
- NCT02785848
- Testing
- Interviews and Surveys
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Vanderbilt University
Sickle Cell Anemia
Bone Marrow Transplantation in Treating Children With Sickle Cell Disease
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ…
- Trial name
- NCT00004485
- Testing
- cyclosporine, fludarabine, mycophenolate mofetil
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease
Efficacy of a Decision Aid for Hydroxyurea in Sickle Cell Disease
The trial is a prospective, randomized study to determine the efficacy of training for use of a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD).
- Trial name
- NCT03577691
- Testing
- Training for use of web based Decision Aid, No training for use of web based Decision Aid, Pretest surveys and posttest surveys
- Where
- 3 sites in United States
- Volunteers
- 2 people
- Sponsor
- Emory University
Diabetic Retinopathy, Sickle Cell Trait
Diabetic Retinopathy and Sickle Trait
To more clearly ascertain the relationship between ocular manifestations of sickle cell disease and diabetes, specifically; whether the presence of sickle cell trait exacerbates the disease progression of diabetic retinopathy.
- Trial name
- NCT00720317
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical University of South Carolina
Hematologic Malignancies, Nonmalignant Diseases
Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies
- Trial name
- NCT03609840
- Testing
- Thiotepa
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease
A Sickle CEll Disease ComplicatioN Trial
The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
- Trial name
- ASCENT (NCT02604368)
- Testing
- SC411
- Volunteers
- 210 people
- Sponsor
- Micelle BioPharma Inc
Acute Chest Syndrome, Sickle Cell Disease
Combined Use of a Respiratory Broad Panel Multiplex PCR and Procalcitonin to Reduce Antibiotics Exposure in Hospitalized Sickle-cell Adults With Acute Chest Syndrome.
Many patients with Sickle Cell Disease (SCD) may develop Acute Chest Syndrome (ACS). ACS is usually caused by a Lower respiratory tract infection (LRTI) which may be caused by either a bacterium or a virus.
- Trial name
- Antibio_STA (NCT03919266)
- Testing
- Intervention: Combined use of a respiratory broad panel multiplex PCR and procalcitonin, Control: usual antibiotic treatment
- Where
- France
- Volunteers
- 72 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Study on Use of Omega-3 Fatty Acids to Improve Outcomes in Individuals With Sickle Cell Disease
Sickle cell disease (SCD) is associated with significant morbidity and mortality. Pain and many adverse outcomes occurring in sickle cell disease are inflammatory driven.
- Trial name
- NCT05758766
- Testing
- Plant-based omega-3-FA
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease
Evaluation of Therapeutic Adherence to Inciting Spirometry in Sickle Cell Patients
Sickle cell disease is one of the most common genetic diseases in the world. (1) It is characterized by the production of abnormal hemoglobin mainly responsible for vaso-occlusive clinical manifestations and chronic hemolysis…
- Trial name
- NCT04667325
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Sickle Cell Disease
Pilates Physical Activity in Sickle Cell Disease
Sickle cell anemia (SCA) is a life-threatening hereditary hemoglobinopathy characterized by hemoglobin (Hb) polymerization that affects many people worldwide.Reduced physical capacity is common in people with SCA.
- Trial name
- NCT05519852
- Testing
- Pilates training
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Cairo University
Sickle Cell Disease, Sickle Cell Anemia
Sickle Cell Disease (SCD) Bone Pain Study
A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Low Bone Density, Osteoporosis
- Trial name
- NCT05283148
- Testing
- Dual-energy X-ray absorptiometry, Vertebral fracture analysis, Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- University of California, Davis
Sickle Cell Disease
Support Strategies for Parents During the First Year Following Their Child's Diagnosis of Sickle Cell Disorder
Background: Sickle cell disorder (SCD), the commonest genetic (faulty gene inherited from both parents) condition in the UK, affects mainly underserved groups.
- Trial name
- NCT06251843
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- King's College London
Sickle Cell Disease, Anemia
SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain
The overall goal of the project is to reduce pain-related, 30-day readmission rates for sickle cell disease (SCD) patients. The investigators want to see if a mobile phone application (app) can help decrease the need for repeat…
- Conditions
- Sickle Cell Disease, Anemia, Anemia, Hemolytic, Anemia, Sickle Cell
- Trial name
- NCT03006718
- Testing
- PROMIS for Pain Management App
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Julia Finkel
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.