Clinical trials: Hemoglobinopathy in Children
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.
- Trial name
- HOPE Kids 2 (NCT04218084)
- Testing
- Voxelotor
- Where
- 35 sites in United States, Egypt and 7 more
- Volunteers
- 236 people
- Sponsor
- Pfizer
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Beta Thalassemia Major
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
the aim of this study is to evaluate the effect of Spirulina compared to Amlodipine on cardiac iron overload and cardiac functions in multi-transfused children with beta thalassemia major
- Trial name
- NCT02671695
- Testing
- Spirulina, Amlodipine
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Tanta University
Vitamin D Deficiency, Sickle Cell Disease
Vitamin D Status in Children With Sickle Cell Disease Living in Lyon, France
Vitamin D deficiency may be under-diagnosed in sickle cell disease French children. Therefore, the investigator need an epidemiologic study about the prevalence of vitamin D deficiency in this specific population.
- Trial name
- EVADDREP (NCT04694976)
- Testing
- 25-OH Vitamin D measure
- Where
- France
- Volunteers
- 130 people
- Sponsor
- Hospices Civils de Lyon
Sickle Cell Disease
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
- Trial name
- PRECISE (NCT06923111)
- Testing
- Xromi
- Where
- 12 sites in Germany, United Kingdom
- Volunteers
- 180 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Anemia
SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell Anemia
Prospective screening and treatment study for children with Sickle Cell Anemia and increased stroke risk living in the Dominican Republic.
- Trial name
- SACRED (NCT02769845)
- Testing
- Hydroxyurea, TCD examination
- Where
- Dominican Republic
- Volunteers
- 283 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease
Propanolol is a beta blocker which has been found to inhibit the ability of epinephrine to upregulate sickle red cell adhesion to laminin and endothelial cells in vitro.
- Trial name
- NCT02012777
- Testing
- propranolol
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Miami
Acute Chest Syndrome, Sickle Cell Disease
Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death.
- Trial name
- INNOSTAPED (NCT01089439)
- Testing
- Nitric oxide by inhalation INOMAX
- Where
- France
- Volunteers
- 21 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Thalassemia
Assessment of Cognitive Function and Quality of Life in Thalassemic Children at Sohag University Hospital
Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world .
- Trial name
- NCT06058260
- Testing
- Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth edition
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Sohag University
Sickle Cell Disease
Interest of Famotidine in Children With Sickle Cell Disease
The purpose of this study is to determine whether oral famotidine, a histamine type 2 receptor antagonist already widely used with very few side effects in other indications in children, is effective in reducing endothelial…
- Trial name
- FAMODREP (NCT05084521)
- Testing
- Famotidine 400 mg/50 mL
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Crisis, Acute Pain
Low-dose Ketamine vs Morphine for Vaso-occlusive Crisis in Sicklers
This clinical trial will inform of the role of Low dose ketamine in the acute treatment of severe painful sickle cell crisis in children in a day-case sickle cell centre.
- Trial name
- KEM-VOC (NCT02434939)
- Testing
- Low dose ketamine, Morphine
- Where
- Uganda
- Volunteers
- 240 people
- Sponsor
- Makerere University
Bone Marrow Transplantation, Sickle Cell Disease
Sickle Cell Disease Conditioning for Bone Marrow Transplant
Most bone marrow transplants for children with sickle cell disease are performed using high doses of two chemotherapy agents: busulfan and cyclophosphamide for the pre-transplant conditioning.
- Trial name
- NCT00968162
- Testing
- fludarabine
- Where
- 10 sites in United States
- Volunteers
- 8 people
- Sponsor
- Emory University
Thalassemia Major
Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital
Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha \& Beta thalassemia.
- Trial name
- NCT03161899
- Where
- Egypt
- Volunteers
- 246 people
- Sponsor
- Assiut University
Sickle Cell Disease
Patient and Public Involvement and Engagement in Research With Children and Young People With Sickle Cell Disorder and Their Families
Aim: To co-produce resources for inclusive and equitable Patient and Public Involvement and Engagement in research on life-limiting conditions, with children and young people with sickle cell disorder and their families.
- Trial name
- NCT06293222
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- King's College London
Vaso-occlusive Crisis, Sickle Cell Disease
Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia
The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease.
- Conditions
- Vaso-occlusive Crisis, Sickle Cell Disease, Sickle Cell Anemia in Children
- Trial name
- NCT04301336
- Testing
- Omega 3, Vit D, Zinc sulfate
- Where
- 4 sites in Egypt, Saudi Arabia
- Volunteers
- 350 people
- Sponsor
- Beni-Suef University
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Beta-Thalassemia
Detection Of β-thalassemia Carriers In Assiut
Thalassemia is different in kids with microcytic hypochromic anemia than general population because there is a confusion between symptoms of thalassemia and iron deficiency anemia in kids and both of them differ in management and…
- Trial name
- NCT03822585
- Testing
- CBC, Iron Study, Serum Ferrittin, HPLC,Genitic Study
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assiut University
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids that would otherwise be started during a sickle cell pain crisis…
- Conditions
- Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children
- Trial name
- NCT07799571
- Testing
- Buprenorphine Buccal Film [Belbuca]
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Natasha Archer
Thalassemia
L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia
- Trial name
- NCT03402191
- Testing
- L-arginine, Sildenafil
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Sherief Abd-Elsalam
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