Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease
In a hybrid type I effectiveness-implementation trial, our three-center research teams aim to examine whether empowering adults with sickle cell disease (SCD) with patient-facing SCD-specific guidelines through an mHealth…
- Trial name
- PF-Guide (NCT06919224)
- Testing
- mHealth app + booklets
- Where
- 4 sites in United States
- Volunteers
- 287 people
- Sponsor
- Ohio State University
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
Sickle Cell Disease, Vaso-occlusive Crisis
Steroid Treatment for Sickle Cell Pain Crisis
The painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication…
- Trial name
- NCT00263562
- Testing
- Steroid arm
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Disease
Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell…
- Trial name
- TRANSPLANTORN2 (NCT06351462)
- Testing
- Spermogram, Hospital Anxiety and Depression Scale (HADS), SF36 Quality of life questionnaire
- Volunteers
- 220 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet assessments to identify type, intensity, frequency, and phase of pain among SCD adolescents is lacking.
- Trial name
- NCT03291613
- Testing
- Pinpoint App
- Where
- 2 sites in United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Sickle Cell Crisis, Functional Abdominal Pain
Music Therapy: Sickle Cell and Pain Crisis
This study will assess whether participants who receive music therapy during inpatient care report a decrease in pain and whether music therapy influences factors such as family satisfaction.
- Conditions
- Sickle Cell Crisis, Functional Abdominal Pain, Patients Followed by the Pain Team
- Trial name
- NCT03901560
- Testing
- Music Therapy
- Volunteers
- 150 people
- Sponsor
- University of Oklahoma
Sickle Cell Disease, HEMOGLOBIN SS
A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)
The purpose of the study is to determine the effectiveness of LOVAZA (fish oil capsules) to decrease inflammation in children and adolescents with Sickle Cell Disease (SCD).
- Conditions
- Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation
- Trial name
- NCT01202812
- Testing
- Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA)
- Where
- 2 sites in United States
- Volunteers
- 48 people
- Sponsor
- Thomas Jefferson University
Sickle Cell Disease
A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered
To evaluate the relative bioavailability of ticagrelor for the different formulations. A randomized cross-over design has been chosen to minimize the effects of between-subject variability and any period effects on the overall…
- Trial name
- NCT03126695
- Testing
- Ticagrelor granule, Ticagrelor pediatric tablets, Ticagrelor pediatric tablets suspended in water
- Where
- Germany
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Sickle Cell Disease, Thalassemia
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and…
- Conditions
- Sickle Cell Disease, Thalassemia, Pediatric Cancer
- Trial name
- RBC-IMPACT (NCT05255445)
- Testing
- Red Blood Cell (RBC) Transfusion
- Where
- 14 sites in United States, Brazil
- Volunteers
- 157 people
- Sponsor
- Westat
Sickle Cell Disease
Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Many youth with chronic disease have difficulty taking medication every day and therefore do not receive full benefit from treatment.
- Trial name
- NCT03462511
- Testing
- HABIT Intervention, Standard of Care, Education materials
- Where
- 4 sites in United States
- Volunteers
- 50 people
- Sponsor
- Columbia University
Anemia, Sickle Cell
An Assessment of Prasugrel on Healthy Adults and Sickle Cell Adults
The purpose of this study is to measure the exposure to prasugrel's active metabolite and the pharmacodynamic effects of prasugrel treatment in people with Sickle Cell Disease (SCD).
- Trial name
- NCT01178099
- Testing
- Prasugrel
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease
Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization
- Trial name
- NCT03943615
- Testing
- Voxelotor
- Where
- 18 sites in United States
- Sponsor
- Pfizer
Sickle Cell Disease
An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
- Trial name
- NCT03041909
- Testing
- GBT440
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Global Blood Therapeutics
Sickle Cell Disease, Thalassemia
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemoglobinopathy
- Trial name
- NCT06313398
- Testing
- Biotin-labeled red blood cells
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Sickle Cell Anemia
Stroke Prevention in Young Adults With Sickle Cell Anemia
Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide. Approximately 150,000 Nigerian children are born each year with sickle cell disease (SCD), making it the country with the…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Stroke, Ischemic, Silent Stroke
- Trial name
- SPIYA (NCT04808778)
- Testing
- Hydroxyurea therapy per standard care
- Where
- 2 sites in United States, Nigeria
- Volunteers
- 250 people
- Sponsor
- Vanderbilt University Medical Center
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
- Trial name
- NCT05777993
- Testing
- Mitapivat
- Where
- 23 sites in Belgium, Brazil and 6 more
- Volunteers
- 55 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Vitamin D Deficiency, Stem Cell Transplant Complications
A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT
Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as…
- Conditions
- Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder
- Trial name
- NCT03176849
- Testing
- Vitamin D3, Standard Vitamin D3 Supplementation
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Phoenix Children's Hospital
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
Genetic Disorders, Sickle Cell Anemia
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
The purpose of this study is to determine and confirm the role of bone marrow transplantation in the treatment of disorders of the red cell and hemoglobin including sickle cell anemia, thalassemia and diamond blackfan anemia.
- Trial name
- NCT00578435
- Testing
- Busulfan, Cyclophosphamide, BMD
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Disease, Solid Tumor
Validating Pain Scales in Children and Young Adults
Background: Assessing pain levels is important to improve treatments for different illnesses. Most pain rating scales are used to determine pain levels in adults. Pain is also a common symptom among children who have cancer.
- Conditions
- Sickle Cell Disease, Solid Tumor, Leukemia, Neurofibromatosis
- Trial name
- NCT01639950
- Where
- United States
- Volunteers
- 252 people
- Sponsor
- National Cancer Institute (NCI)
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