Clinical trials: Hemoglobinopathy (Disorder)
Sickle Cell Disease
Arterial Function Parameters and Transcranial Doppler Velocity in Paediatric Patients With Sickle Cell Disease
Structural and functional changes in arteries are increasingly being recognized as significant features of sickle cell disease. This study aims to determine whether there are differences in arterial function parameters between…
- Trial name
- AFAT (NCT05748717)
- Where
- Jamaica
- Volunteers
- 34 people
- Sponsor
- The University of The West Indies
Cardiovascular Diseases, Sickle Cell Disease
Fitness Trial in Adults With Sickle Cell Disease (SCD Fit): A Feasibility Study
The purpose of this project is to develop novel approaches to promote health and longevity while enhancing quality of life among persons with Sickle cell disease (SCD).
- Conditions
- Cardiovascular Diseases, Sickle Cell Disease, Exercise
- Trial name
- NCT04772391
- Testing
- Exercise Sessions
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Memorial Healthcare System
Sickle Cell Disease
Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care
Background: Sickle cell disease (SCD) is caused by a genetic defect that affects how hemoglobin is made. Due to this, people with SCD have abnormally-shaped red blood cells, which can result in poor oxygen transport in the body…
- Trial name
- NCT03167450
- Where
- United States
- Volunteers
- 109 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Beta-Thalassemia, Iron Overload
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients
This is a randomized, open label, two arms superiority trial of a representative population of patients with a primary diagnosis of transfusion dependent thalassemia with evidence of moderate cardiac iron overload, defined as an…
- Trial name
- CANALI (NCT02474420)
- Testing
- Amlodipine, Deferasirox
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- Kevin H.M. Kuo, MD, MSc, FRCPC
Pain Crisis
Natural History of Sickle Cell Disease
This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.
- Trial name
- NCT00081523
- Where
- 3 sites in United States
- Volunteers
- 3,500 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hepatitis C, Thalassemia
Pegasys® Plus Ribavirin in Thalassemic Patients With Hepatitis C Virus Infection
Antiviral treatment of HCV in thalassemia has raised concerns of ribavirin-induced hemolysis and increased iron loading. Blood Transfusion in Thalassemic patients are a known high risk for acquiring hepatitis C.
- Trial name
- NCT00707850
- Testing
- PEGASYS® (Peginterferon Alfa-2a (40KD)) Plus COPEGUS® (Ribavirin)
- Where
- Iran
- Volunteers
- 300 people
- Sponsor
- Baqiyatallah Medical Sciences University
Red Cell Alloantibodies, Blood Transfusion-Dependent
Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients
Transfusion-dependent patients, particularly those with β-thalassemia major, require lifelong regular red blood cell (RBC) transfusions to maintain adequate hemoglobin levels and prevent severe anemia.
- Conditions
- Red Cell Alloantibodies, Blood Transfusion-Dependent, Beta Thalassemia
- Trial name
- NCT07480980
- Volunteers
- 100 people
- Sponsor
- Assiut University
Sickle Cell Anemia, Children, Only
Shared-Decision Making for Hydroxyurea
The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent…
- Trial name
- ENGAGE-HU (NCT03442114)
- Testing
- Hydroxyurea SDM Toolkit, Clinician Pocket Guide
- Where
- 11 sites in United States
- Volunteers
- 176 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
Osteopathic Manipulation in the Management of Pain Associated With Sickel Cell Disease
Background: Sickle cell disease is the most common monogenic disease in the world caused by a mutation in the β-globin gene which creates abnormal hemoglobin called HbS.
- Trial name
- OSTEODREP (NCT05632289)
- Testing
- osteopathic manipulative therapy
- Where
- France
- Volunteers
- 37 people
- Sponsor
- College Osteopathique de Provence Aix-Marseille
Sickle Cell Anemia
The Efficacy of Jobelyn (Sorghum Bicolor Extract)in the Treatment of Sickle Cell Anemia
The primary objective is to determine if there is a significant increase in the haematocrit value of patients on Jobelyn and standard therapy compared to those on standard therapy alone.
- Trial name
- NCT01703104
- Testing
- Paludrine + Folic Acid, Paludrine + Folic Acid + Jobelyn
- Where
- Nigeria
- Volunteers
- 150 people
- Sponsor
- Lagos State University
Sickle Cell Disease
Improving Self-Management in Adolescents With Sickle Cell Disease
The objective of this study is to determine the feasibility and acceptability of SCThrive, a an innovative, technology-enhanced, group self-management intervention that uses a mixed in-person and online format and supported by a…
- Trial name
- SCThrive (NCT02851615)
- Testing
- SCThrive Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Sickle Cell Anemia, Pulmonary Hypertension
Heart Disease in Sickle Cell Anemia
This study will explore what may cause people with sickle cell anemia to have heart problems and an increased risk of sudden death. People 18 years of age and older with sickle cell anemia may be eligible for this study.
- Trial name
- NCT00113152
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Sickle Cell Disease, Sickle Cell Anemia
Hydroxyurea Optimization Through Precision Study
Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for…
- Trial name
- HOPS (NCT03789591)
- Testing
- Hydroxyurea
- Where
- 13 sites in United States
- Volunteers
- 104 people
- Sponsor
- Lifespan
Sickle Cell Disease, Cognitive Impairment
Cognitive Rehabilitation in Sickle Cell Disease
The majority of school-age children with sickle cell disease (SCD) experience neurocognitive deficits, even in the absence of stroke.
- Trial name
- NCT01793740
- Testing
- Cogmed
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Duke University
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis
Sickle Cell Disease
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can…
- Trial name
- SUN (NCT03587272)
- Testing
- Alemtuzumab, low dose total body irradiation, Sirolimus
- Where
- 7 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Robert Nickel
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver.
- Trial name
- NCT01186419
- Testing
- SPD602 (FBS0701, SSP-004184)
- Where
- 9 sites in United States, Italy and 3 more
- Volunteers
- 51 people
- Sponsor
- Shire
Covid19
Comorbidities and Risk Score in COVID-19 Patients
Retrospective multi-center cohort study. Consecutive patients hospitalized for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) up to October 2020 will be included.
- Trial name
- Comorbidities (NCT04670094)
- Where
- 5 sites in Italy
- Volunteers
- 4,555 people
- Sponsor
- University of Milano Bicocca