Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell Disease
This study aims to develop an effective, brief, family-based intervention targeting quality of life and school functioning for youth with sickle cell disease.
- Trial name
- FTC (NCT02273310)
- Testing
- Problem-Solving Skills Training for Disease Management
- Volunteers
- 83 people
- Sponsor
- University of Pennsylvania
Sickle Cell Disease
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of…
- Trial name
- HIBISCUS (NCT04624659)
- Testing
- Etavopivat Tablets Low dose, Etavopivat Tablets High dose, Etavopivat Tablets
- Where
- 142 sites in United States, Canada and 15 more
- Volunteers
- 450 people
- Sponsor
- Forma Therapeutics, Inc.
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 Anemia
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or…
- Trial name
- NCT03020615
- Testing
- Hydroxyurea
- Where
- 4 sites in United States
- Volunteers
- 58 people
- Sponsor
- St. Jude Children's Research Hospital
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
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
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.
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness
This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea and are receiving care at one of the study sites.
- Trial name
- ADHERE (NCT06264700)
- Testing
- Video Directly Observed Therapy (VDOT), Health Reminder Tip Alerts
- Where
- 3 sites in United States
- Volunteers
- 60 people
- Sponsor
- Nationwide Children's Hospital
Sickle Cell Disease
Sickle Cell Anemia in an Arab Bedouin Village in the Northern Israel
Sickle cell anemia and sickle cell thalassemia are frequent diseases among the israeli arab population. The purpose of this study is to assess the clinical characteristics of the patients in one arab village and the laboratory…
- Trial name
- NCT00481039
- Testing
- Medical history and basic laboratory analysis
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease
The Link Between Anemia and Deficits in Memory and Attention in Individuals With Sickle Cell Disease
Sickle cell disease is an inherited blood disorder that affects red blood cells (RBCs). People with sickle cell disease frequently experience anemia, or a low number of RBCs.
- Trial name
- NCT00451919
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Sickle Cell Disease, Acute Chest Syndrome
A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease
Acute chest syndrome (ACS), a lung complication in sickle cell disease (SCD), is the second most common cause of hospitalization and leading cause of death in SCD.
- Trial name
- NCT02630394
- Testing
- Azithromycin
- Where
- 2 sites in United States
- Sponsor
- University of Mississippi Medical Center
SNAKEBITE, THROMBOTIC MICROANGIOPATHY
Incidence of Snakebite Associated Thrombotic Microangiopathy & Role of Peripheral Blood Smear as a Predictor of Clinical Outcome
The aim of this study is to find the overall incidence of thrombotic microangiopathy in snakebite victims. As we know snakebite is a common in tropical regions.
- Conditions
- SNAKEBITE, THROMBOTIC MICROANGIOPATHY, SCHISTOCYTES
- Trial name
- NCT04845022
- Where
- India
- Volunteers
- 81 people
- Sponsor
- Jubilee Mission Medical College and Research Institute
Thrombosis
Sickle Cell Trait and the Risk of Venous Thromboembolism
The purpose of this trial is to investigate D-Dimer levels, a surrogate marker of venous thromboembolism, in pregnant/postpartum white women as compared to pregnant/postpartum black women, and pregnant/postpartum women with…
- Trial name
- SCT&DVT (NCT01148940)
- Where
- United States
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Sickle Cell Disease, Sickle Cell Anemia
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease.
- Trial name
- NCT05169580
- Testing
- Pociredir oral capsule(s)
- Where
- 18 sites in United States, Nigeria and 1 more
- Volunteers
- 45 people
- Sponsor
- Fulcrum Therapeutics
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
Thalassemia, Sickle Cell Anemia
Blood Transfusions in Thalassemia Patients, Complications and Adverse Effects
Patients suffering from Thalassemia or another hemoglobinopathies required regular blood transfusions. The complications and adverse effects of blood transfusions can be classified as immediate and late.
- Trial name
- NCT00971880
- Testing
- Clinical medical record summary
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease
Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease
The research proposed is a pilot study of pediatric and adolescent/young adult patients who have received the curative intervention (MSD-SCT), disease-modifying interventions (HU or CT) or SCC (control), with respect to three…
- Trial name
- SCD_Cross (NCT01369160)
- Testing
- Quality of Life measures
- Where
- 2 sites in United States
- Volunteers
- 33 people
- Sponsor
- Emory University
Anemia, Sickle Cell
Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
- Trial name
- NCT00067080
- Testing
- ICL670, deferoxamine
- Where
- 34 sites in United States
- Volunteers
- 195 people
- Sponsor
- Novartis Pharmaceuticals
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