Clinical trials: Sickle Cell Anemia in Children
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
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
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
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
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
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
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
Sickle Cell Anemia in Children
The Effect of Voxelotor on Cerebral Hemodynamic Response in Children With Sickle Cell Anemia
Voxelotor is a new drug for adolescents and adults with sickle cell disease that improves hemoglobin levels and reduces the incidence of worsening anemia.
- Trial name
- VoxSCAN (NCT05018728)
- Testing
- Voxelotor
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Emory University
Vitamin D Deficiency, Stem Cell Transplant Complications
A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT
Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as…
- Conditions
- Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder
- Trial name
- NCT03176849
- Testing
- Vitamin D3, Standard Vitamin D3 Supplementation
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Phoenix Children's Hospital
Nocturnal Enuresis, Anemia, Sickle Cell
Desmopressin as a Therapy for Bedwetting in Children With Sickle Cell Disease
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
- Trial name
- NCT02636387
- Testing
- Desmopressin
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Montefiore Medical Center
Preventing Stroke in Sickle Cell Anaemia
Preventing Stroke Triggers in Children With Sickle Cell Anaemia in Mulago Hospital, Kampala (PREST ): a Randomized Control Trial
Sickle cell anaemia (SCA) is a common hereditary haemoglobin disorder in Africa. World wide it is estimated that about 300,000 newborns are born every year. Of which 75% of them live in Sub-saharan Africa (SSA).
- Trial name
- PREST (NCT03666806)
- Testing
- Influenza vaccine
- Where
- Uganda
- Volunteers
- 150 people
- Sponsor
- Makerere University
Sickle Cells Patients
Study of Vitamin B12 Metabolism in Children With Sickle Cell Disease Exposed to MEOPA
Short description of the protocol intended for the lay public. Include a brief statement of the study hypothesis (Limit : 5000 characters) The sickle cells anemia is a monogenic disease linked to the presence of Hemoglobin S due…
- Trial name
- DREPAM (NCT06581900)
- Testing
- Vitamin B12
- Where
- France
- Volunteers
- 29 people
- Sponsor
- CHU de Reims
Anemia; Drepanocytic
Prognostic Factors of Acute Splenic Sequestration
Acute splenic sequestration is a frequent and life threatening complication occurring in approximately 10 % of homozygous children. Maximal incidence is between 6 and 18 months.
- Trial name
- SSADREPA (NCT01207037)
- Testing
- blood samples and scintigraphy
- Where
- France
- Volunteers
- 58 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
Children with sickle cell disease (SCD) are at risk for neurobehavioral problems because of the impact the disease can have on the central nervous system.
- Trial name
- NCT02227472
- Testing
- Evaluation of cognitive and pre-academic skills, Parent questionnaires
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- St. Jude Children's Research Hospital
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