There are currently 95 active clinical trials seeking participants for Sickle Cell Disease research studies. The states with the highest number of trials for Sickle Cell Disease participants are New York, North Carolina, California and Georgia.
Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
NCT06689800
Recruiting
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age).
Gender:
ALL
Ages:
8 years and above
Trial Updated:
11/27/2024
Locations: Washington University School of Medicine, Saint Louis, Missouri
Conditions: Hematologic Malignancy, Solid Tumor, Sickle Cell Disease, Aplastic Anemia, Immune Deficiency, Metabolic Disorder
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Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
NCT05405114
Recruiting
This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo. Participants who are on HU treatment before joining the study will get NDec, NDec and placebo, or continue on HU. Which treatment participants g... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
11/25/2024
Locations: University Of South Alabama, Mobile, Alabama +70 locations
Conditions: Sickle Cell Disease
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Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
NCT05810181
Recruiting
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.
Gender:
ALL
Ages:
Between 18 years and 35 years
Trial Updated:
11/20/2024
Locations: St. Jude Children's Research Hospital, Memphis, Tennessee
Conditions: Sickle Cell Disease
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An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
NCT04688411
Recruiting
The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and acceptability of MED-Go app in adolescents and young adults (AYA) with sickle cell disease (SCD). The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
Gender:
ALL
Ages:
Between 12 years and 21 years
Trial Updated:
11/19/2024
Locations: Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois
Conditions: Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
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Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients with Sickle Cell Disease
NCT05861453
Recruiting
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
11/13/2024
Locations: University of Alabama at Birmingham (UAB), Birmingham, Alabama +20 locations
Conditions: Sickle Cell Disease
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FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
NCT06539169
Recruiting
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via elect... Read More
Gender:
ALL
Ages:
All
Trial Updated:
11/12/2024
Locations: xCures, Los Altos, California
Conditions: Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
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Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
NCT06016634
Recruiting
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
Gender:
ALL
Ages:
Between 18 years and 80 years
Trial Updated:
11/08/2024
Locations: UC Davis Comprehensive Cancer Center, Sacramento, California
Conditions: Sickle Cell Disease, Sickle Cell Anemia, Osteonecrosis, Ischemic Necrosis, Avascular Necrosis
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Cutaneous Hydration Assessment in SCD
NCT05210114
Recruiting
This study will validate the diagnostic accuracy of a cutaneous hydration sensor. This sensor will also be evaluated for its feasibility as a point-of-care device for the assessment of hydration status and its potential to guide hydration therapy in patients with sickle cell disease (SCD).
Gender:
ALL
Ages:
12 years and above
Trial Updated:
11/08/2024
Locations: UPMC Sickle Cell Clinic, Pittsburgh, Pennsylvania
Conditions: Sickle Cell Disease
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Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
NCT03121001
Recruiting
The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irrad... Read More
Gender:
ALL
Ages:
Between 16 years and 60 years
Trial Updated:
11/07/2024
Locations: University of Illinois at Chicago, Chicago, Illinois
Conditions: Sickle Cell Disease
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Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease
NCT06477289
Recruiting
This study will examine sleep disordered breathing and sleep quality in participants (ages 12-18) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotely in the homes of participants. Neurocognitive, behavioral, and neuroimaging evaluations will occur on the same day as a routine clinic visit. Primary Objective: Evaluate the relationship between no... Read More
Gender:
ALL
Ages:
Between 12 years and 18 years
Trial Updated:
10/21/2024
Locations: St. Jude Children's Research Hospital, Memphis, Tennessee
Conditions: Sickle Cell Disease
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Intravenous Gammaglobulin for Sickle Cell Pain Crises
NCT01757418
Recruiting
The purpose of this study is to determine whether intravenous immune globulin is safe and effective in the acute treatment of pain crises in sickle cell disease. Funding Source: Food and Drug Administration (FDA), Office of Orphan Products Development (OOPD)
Gender:
ALL
Ages:
Between 6 years and 65 years
Trial Updated:
10/17/2024
Locations: Montefiore Medical Center, Bronx, New York
Conditions: Sickle Cell Disease, Pain
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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
NCT05329649
Recruiting
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
Gender:
ALL
Ages:
Between 2 years and 11 years
Trial Updated:
10/16/2024
Locations: Atrium Health Levine Children's Hospital, Charlotte, North Carolina +6 locations
Conditions: Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
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