Clinical trials: Hemoglobinopathy in Children
Sickle Cell Disease
Angiotensin-converting Enzyme Inhibitors and Early Sickle Cell Renal Disease in Children
Patients with sickle cell anaemia may develop renal disease. In fact, renal disease occurred in 40% of adults patients (macroalbuminuria) with evolution to end-stage renal disease for half of them.
- Trial name
- MADREPIEC (NCT01096121)
- Testing
- Enalapril
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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)
Sickle Cell Disease
Study on Use of Omega-3 Fatty Acids to Improve Outcomes in Individuals With Sickle Cell Disease
Sickle cell disease (SCD) is associated with significant morbidity and mortality. Pain and many adverse outcomes occurring in sickle cell disease are inflammatory driven.
- Trial name
- NCT05758766
- Testing
- Plant-based omega-3-FA
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Alabama at Birmingham
Thalassemia Majors (Beta-Thalassemia Major)
Effect of Omega 3 Fatty Acids on Heart and Liver in Beta Thalassemia Patients
the aim of this interventional study to show the effect of taking omega 3 for about 6 months for patients beta thalassemia major maintained on standard of care and receiving the iron chelator deferasirox on liver, cardiac…
- Trial name
- NCT07863791
- Testing
- Omega 3 Fish Oil supplements, Deferasirox
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Ain Shams University
Anemia, Sickle Cell
Telemedicine for Children With Sickle Cell Disease
The purpose of this study is to learn more about how the use of two different types of telemedicine (distance medical care) can address barriers to receiving comprehensive sickle cell care, and whether care can be improved.
- Trial name
- NCT05087303
- Testing
- Types of Telemedicine Delivery Models
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Indiana University
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease, Vitamin D Deficiency
High Dose Vitamin D Supplementation in Children With Sickle Cell Disease
Suboptimal vitamin D status is well reported in sickle cell disease (SCD) patients and associated with a negative impact on health-related quality of life (HRQL).
- Conditions
- Sickle Cell Disease, Vitamin D Deficiency, Health Related Quality of Life, Hand Grip Strength
- Trial name
- NCT06274203
- Testing
- Vitamin D3
- Where
- Egypt
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Zagazig University
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
Sickle Cell Anemia
Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE)
The primary goal of the Phase III SCATE trial is to compare 30 months of alternative therapy (hydroxyurea) to standard care (observation) in children with sickle cell anemia and conditional (170 - 199cm/sec) Transcranial Doppler…
- Trial name
- SCATE (NCT01531387)
- Testing
- Hydroxyurea
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 38 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Thalassemia, Hematopoietic Stem Cell Transplantation
Low-Dose Liposomal Amphotericin B for Invasive Fungal Infection Prophylaxis in Neutropenic Children
This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (1 mg/kg/day, intravenous, once daily) for the prevention of invasive fungal infections in…
- Conditions
- Thalassemia, Hematopoietic Stem Cell Transplantation, Neutropenia, Invasive Fungal Infections
- Trial name
- NCT07725263
- Testing
- Liposomal Amphotericin B (LAmB)
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
Nursing Caries
Nonpharmacological Approaches and Parental Education in Children With Sickle Cell Disease
Aim: Sickle cell disease (SCD) is a hereditary disease. Approximately 5% of the world's population carries trait genes for haemoglobin disorders, mainly, sickle-cell disease and thalassaemia.
- Trial name
- NCT04121247
- Testing
- nonpharmacological approaches to parents of children with sickle cell disease training program
- Where
- Turkey (Türkiye)
- Volunteers
- 163 people, healthy volunteers welcome
- Sponsor
- Akdeniz University
Sickle Cell Disease, Vaso-occlusive Crisis
Effect of Virtual Reality Technology for Pain Management of Vaso-Occlusive Crisis in Patients With Sickle Cell Disease
Acute vaso-occlusive crisis (VOC) is the most common complication in patients with sickle cell disease (SCD) and pain related to VOC is often inadequately treated.
- Trial name
- NCT03353584
- Testing
- Virtual Reality Therapy
- Where
- 2 sites in United States
- Volunteers
- 76 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Ameliorating Attention Problems in Children With Sickle Cell Disease (SCD)
The purpose of this study is to assess whether methylphenidate is effective in enhancing the cognitive performance of children with the HbSS or HbSC genotype of SCD who have sustained neurological complications on…
- Trial name
- NCT01411280
- Testing
- methylphenidate
- Where
- 2 sites in United States
- Volunteers
- 34 people
- Sponsor
- Temple University
Beta-thalassemia
Complications in Children With B- Thalassemia Major
Worldwide, there are more than 60,000 births annually of serious forms of thalassemia .The World Health Organization considers thalassemia to be a major health burden.
- Trial name
- NCT03462784
- Testing
- Demographic, physical examination, data will becollected .
- Volunteers
- 201 people
- Sponsor
- Assiut University
Sickle Cell Disease
Trial of Zileuton CR in Children and Adults With Sickle Cell Disease
The purpose of this research study is to test the safety of Zileuton and see what effects (good and bad) it has on you, other children and adults with Sickle Cell Disease (SCD).
- Trial name
- Zileuton (NCT01136941)
- Testing
- Zileuton
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Epidemiology and Pathophysiological Mechanisms of HTAP in SS and SC Children in Martinique and Guadeloupe.
pulmonary arterial hypertension (PAH) has been reported with a prevalence of approximately 30% in adult sickle cell disease (SCD) patients, with an increased mortality in SCD patients with PAH, compared with those without PAH.
- Trial name
- SAPOTILLE (NCT03368261)
- Testing
- HTAP/ clinical complications in the sickle cell disease
- Where
- Martinique
- Volunteers
- 185 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease
Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults
The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain.
- Conditions
- Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease, Ketamine Infusion, Pain Management
- Trial name
- NCT07369024
- Testing
- Sub-dissociative Ketamine Infusion
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Mohsen Saidinejad
Sickle Cell Anemia, Sickle Cell Disease
Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas
The Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM) study is the first placebo-controlled randomized clinical trial of hydroxyurea treatment in a malaria endemic region.
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Malaria
- Trial name
- NOHARM-MTD (NCT03128515)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 187 people
- Sponsor
- Indiana University
Sickle Cell Disease
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice…
- Trial name
- KID-BID (NCT06578507)
- Testing
- Hydroxycarbamid
- Where
- 7 sites in France, French Guiana
- Volunteers
- 50 people
- Sponsor
- Theravia
Cognitive Change, Beta-Thalassemia
Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia
This study aims to: Assess the cognitive function in children with beta thalassemia Evaluate the fatigue in beta thalassemic children Assess the health related quality of life measures in children with beta thalassemia.
- Conditions
- Cognitive Change, Beta-Thalassemia, Fatigue, Quality of Life
- Trial name
- NCT05469230
- Where
- Egypt
- Volunteers
- 168 people, healthy volunteers welcome
- Sponsor
- Cairo University