Clinical trials: Sickle Cell Anemia in Children
Sickle Cell Disease
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical…
- Trial name
- NYMC-571 (NCT02675959)
- Testing
- Defibrotide
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
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
Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics.
- Trial name
- NCT05725902
- Testing
- Etavopivat
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Novo Nordisk A/S
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
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
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
Smoking, Cancer
Parent Willingness to Participate in Tobacco Trials in the Pediatric Clinical Setting
Little is known about what factors influence parental decisions to participate or to decline participation in tobacco trials offered in the pediatric clinical setting.
- Conditions
- Smoking, Cancer, Sickle Cell Disease
- Trial name
- NCT01673243
- Testing
- Questionnaire
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell…
- Trial name
- NCT05470270
- Testing
- Hydroxycarbamide
- Where
- 3 sites in France
- Volunteers
- 33 people
- Sponsor
- Theravia
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 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 Anemia
Adherence to Hydroxyurea in Children With Sickle Cell Anemia
Medication non-adherence is a true public health problem. Despite advancements in the molecular understanding of disease and improvements in therapy, patient health outcomes will not improve unless patients take prescribed…
- Trial name
- NCT00672789
- Testing
- Blood smear education, standard education
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Duke 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
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 Trait
A Program to Increase Sickle Cell Trait Knowledge Among Parent of Young Children Identified in Newborn Screening
This is a study for parents of young children with Sickle Cell Trait (SCT) identified by newborn screening who are referred and present for in person SCT education at the Institution.
- Trial name
- SCTaware (NCT03984500)
- Testing
- Education
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Susan Creary
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
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 Anemia in Children
Low Systemic/High Local Exercise Load in Peds SCD
This research study wants to learn about what kind of exercise is best for kids with sickle cell disease. Participating children will have a small amount of blood drawn one time at the beginning of the study.
- Trial name
- NCT06387758
- Testing
- Low systemic strength training, Moderate systemic exercise
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Mississippi Medical Center