Clinical trials: Hemoglobinopathies
Transfusion Dependent Beta-Thalassaemia
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia.
- Trial name
- ET-01 (NCT04925206)
- Testing
- ET-01
- Where
- 4 sites in China
- Volunteers
- 8 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Iron Overload, Beta-Thalassemia
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Trial name
- NCT02274233
- Testing
- SP-420
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 24 people
- Sponsor
- Sideris Pharmaceuticals
Non Malignant Diseases, Immunodeficiencies
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with…
- Conditions
- Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- RIC (NCT01050855)
- Testing
- Reduced-Intensity Conditioning
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Hospital of Philadelphia
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center
Sickle Cell Disease Vaso-occlusive Crisis
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with…
- Trial name
- NCT06699849
- Testing
- CSL889
- Where
- 30 sites in United States, Turkey (Türkiye) and 1 more
- Volunteers
- 70 people
- Sponsor
- CSL Behring
Sickle Cell Disease
Dissemination and Implementation of Stroke Prevention Looking at the Care Environment
The Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) study is a multi-center, national, National Heart, Lung and Blood Institute (NHLBI)-funded grant to look at the real-world…
- Trial name
- DISPLACE (NCT04173026)
- Testing
- Provider-level intervention, Patient-level intervention
- Where
- 15 sites in United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease
Medication Adherence in Youth With Sickle Cell Disease (SCD)
Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD.
- Trial name
- NCT02143076
- Testing
- Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Vaso-occlusive Pain Episode
Arginine Therapy for Sickle Cell Disease Pain
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores…
- Trial name
- NCT02536170
- Testing
- L-arginine, L-arginine Loading Dose
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Emory University
Preterm Birth, Oxygen Toxicity
Fetal Haemoglobin and Cerebral and Peripheral Oxygenation.
The aim of this study is to investigate the relationship between cerebral and peripheral oxygenation and oxygen extraction, as measured by NIRS (near-infrared spectroscopy ), and the FHbF (fraction of fetal hemoglobin) and…
- Conditions
- Preterm Birth, Oxygen Toxicity, Fetal Hemoglobin
- Trial name
- HbFIN (NCT04802629)
- Testing
- Near-infrared spectroscopy
- Where
- Austria
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
New-borns Screening, Sickle Cell Disease
Quality of Care of Children With Sickle Cell Disease (SCD) Screened at Birth in France
The main objective of this study is to evaluate at the national level (France) the early healthcare practices for children with sickle cell disease screened at birth, the adequacy of theses practices with the national…
- Trial name
- EVADREP (NCT03119922)
- Testing
- No intervention
- Volunteers
- 1,750 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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, Thalassaemia
Integrative Diagnosis for SCD and Other RADs
INTEGRA aims at enabling personalized medicine for RHADs patients by the establishment of an integrative diagnostic approach based on deep phenotypic and genetic characterization through combining new generation methodologies.
- Conditions
- Sickle Cell Disease, Thalassaemia, Congenital Dyserythropoietic Anemia (CDA), Enzyme Disorder; Anemia
- Trial name
- INTEGRA (NCT07206095)
- Testing
- Analysis of genetic modifiers, Disease phenotyping
- Where
- 9 sites in Spain
- Volunteers
- 200 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Sickle Cell Disease and G6PD Deficiency
Screening of Coexistence Between Sickle Cell Anaemia and G6PD Deficiency
G6PD deficiency may have a subtle effect on the severity of hemolysis and also worsen the degree of anaemia in SCD when the two disorders coexist.
- Trial name
- NCT06615024
- Testing
- G6pd enzyme sickling test, G6PD enzyme and sickling test, G6PD enzyme and HPLC
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Fatma Hussein Mahmoud
Thalassemia, Beta, Thalassemia
Impact of Virtual Reality on Pain and Anxiety During Intravenous Cannulation Procedures Among Thalassemia Patients in UAE.
The goal of this trial is to evaluate the effects of therapeutic Virtual Reality (VR) on pain, anxiety, fatigue and boredom during intravenous (IV) cannulations in comparison to standard of care (SOC).The main question it aims to…
- Conditions
- Thalassemia, Beta, Thalassemia, Pain, Anxiety
- Trial name
- NCT07099196
- Testing
- Headset-based Virtual Reality Simulation
- Where
- United Arab Emirates
- Volunteers
- 115 people
- Sponsor
- Mohammed Bin Rashid University of Medicine and Health Sciences
Sickle Cell Disease
HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease
multicentric interventional biomedical research phase II, prospective, non-randomized evaluating a haploidentical marrow transplants after reduced-intensity conditioning and prevention of GvHD based on cyclophosphamide…
- Trial name
- DREPHAPLO (NCT03240731)
- Testing
- bone marrow transplant
- Where
- 7 sites in France
- Volunteers
- 25 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Sickle Cell Disease
European Sickle Cell Disease Cohort - Hydroxyurea
In the context of the Risk Management Plan (RMP), as requested from Addmedica by the EMEA, to collect information about long-term safety of Siklos® (hydroxycarbamide) when used in patients with Sickle Cell Disease.
- Trial name
- ESCORT-HU (NCT02516579)
- Testing
- Siklos
- Where
- 60 sites in France, French Guiana and 5 more
- Volunteers
- 1,906 people
- Sponsor
- Theravia
Thalassemia
Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal Events
The evaluation of efficacy is performed by laboratory monitoring of bone density and resorption markers and clinical monitoring of bone density improvement.
- Trial name
- NCT00346242
- Testing
- Zoledronic Acid
- Where
- Greece
- Volunteers
- 60 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Predictors and Outcomes in Patients With Sickle Cell Disease
Children with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea.
- Trial name
- NCT03431935
- Where
- 2 sites in United States
- Volunteers
- 94 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Renal Disease
Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia
The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients
- Conditions
- Sickle Cell Disease, Renal Disease, Glomerular Disease
- Trial name
- NCT04380610
- Testing
- iohexol GFR
- Where
- 4 sites in United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease
Cannabidiol in Sickle Cell Disease
Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.
- Trial name
- SPICE (NCT06930703)
- Testing
- Cannabidiol
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Icahn School of Medicine at Mount Sinai