Clinical trials: Hemoglobinopathy in Children
Beta Thalassemia Major
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
- Trial name
- NCT03947632
- Testing
- serum ferritin and hemoglobin test
- Volunteers
- 367 people
- Sponsor
- Aga Khan University
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 Disease (SCD), Invasive Bacterial Infections
Epidemiology of Invasive Bacterial Infections in Children With Sickle Cell Disease in France Between 2020 and 2025
Children with sickle cell disease are at high risk of invasive bacterial infections, which may lead to serious complications. Preventive measures, including antibiotic prophylaxis and vaccination, have changed the epidemiology of…
- Trial name
- DREPIBAC (NCT07639008)
- Where
- 31 sites in France
- Volunteers
- 350 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Pain
The Effect of Acupressure on Pain in Children With Thalassemia
This randomized controlled trial evaluates the effect of acupressure on reducing the pain of blood collection in children with Thalassemia. The hypothesis of this study is that acupressure application reduces acute pain.
- Trial name
- acupressure (NCT06163196)
- Testing
- acupressure
- Where
- Turkey (Türkiye)
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Mersin 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
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle-cell Disease (SCD)
Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve…
- Trial name
- DREPAtient (NCT04413539)
- Where
- France
- Volunteers
- 1,088 people
- Sponsor
- Argo Sante
Haematological Malignancy, Acute Leukemia
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts
To date, allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes, haemoglobinopathies…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT04331483)
- Testing
- Physical activity program
- Where
- France
- Sponsor
- Centre Leon Berard
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
Anemia, Sickle Cell
Folic Acid Supplementation in Children With Sickle Cell Disease
Folic acid supplementation (1mg/d) is the standard recommendation for Canadian children with Sickle cell disease (SCD), even though it can provide up to six times the recommended intake amount for healthy children.
- Trial name
- NCT04011345
- Testing
- Folic Acid Supplement
- Where
- Canada
- Volunteers
- 31 people
- Sponsor
- University of British Columbia
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
Hemoglobinopathies
Transcranial Doppler in Children With Hemoglobinopathies
Transcranial Doppler ultrasonography (TCD) is a noninvasive, portable technique for evaluating the intracranial vasculature. TCD ultrasonography is performed placing a low frequency (≤ 2 MHz) transducer on the scalp of the…
- Trial name
- NCT04798157
- Testing
- transcranial doppler
- Volunteers
- 50 people
- Sponsor
- Sohag University
Sickle Cell Disease
Biotin-RBC Transfusion in SCD
This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion…
- Trial name
- NCT04426591
- Testing
- Biotin Labeled Red Blood Cells, Pathogen-reduced Biotin Labeled Red Blood Cells
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Marianne Yee
Hemoglobinopathy (Disorder), Severe Aplastic Anemia
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition.
- Conditions
- Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
- Trial name
- NCT04356469
- Testing
- Haploidentical Hematopoietic Cell Transplantation
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Johns Hopkins All Children's Hospital
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Beta Thalassemia Major
Effect of Spirulina on Serum Hyaluronic Acid in Beta Thalassemic Children With Hepatitis C
Thalassemics can develop liver fibrosis because of iron overload and hepatitis C infection. The latter is the main risk factor for liver fibrosis in transfusion dependent thalassemics.
- Trial name
- NCT02744547
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Tanta University
Sickle Cell Anemia in Children
Effect of Aquatic Exercises on Muscle Strength and Quality of Life in Children With Sickle Cell Anemia
Sickle cell anemia is a global public health disorder that affects millions of people across the globe. It is an inherited autosomal recessive hemoglobin condition caused by a beta-globin gene mutation that results in an aberrant…
- Trial name
- NCT06939933
- Testing
- aquatic exercise in addition to physical exercise, designed physical exercises only
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Kafrelsheikh University
Beta Thalassemia
Effects of Spirulina on Cardiac Functions in Children With Beta Thalassemia Major
The aim of this study is to evaluate the cardioprotective effect of spirulina in children with beta thalassemia.
- Trial name
- NCT02597595
- Testing
- spirulina
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Tanta University
Sickle Cell Disease, Stroke
Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease
This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease.
- Trial name
- HUPrevent (NCT01389024)
- Testing
- Hydroxyurea
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Johns Hopkins University
Beta Thalassemia Major
Effect of Spirulina on Liver Iron Concentration in Beta Thalassemic Children With Hepatitis C
Thalassemics can develop liver fibrosis because of iron overload and hepatitis C infection. The latter is the main risk factor for liver fibrosis in transfusion dependent thalassemics.
- Trial name
- NCT02744560
- Testing
- spirulina
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Tanta University