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
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
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
Chronic Iron Overload
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasirox (DFX) at dose of 20-40…
- Trial name
- NCT01825512
- Testing
- Deferiprone, Deferasirox
- Where
- 22 sites in Albania, Cyprus and 5 more
- Volunteers
- 435 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
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 Disease
Adenosine 2A Agonist Lexiscan in Children and Adults With Sickle Cell Disease
Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape.
- Trial name
- NCT01085201
- Testing
- Lexiscan
- Where
- 7 sites in United States
- Volunteers
- 39 people
- Sponsor
- Dana-Farber Cancer Institute
Sickle Cell Disease
Measurement of Cerebral Blood Flow Using Transcranial Doppler Ultrasound in Children With Sickle Cell Disease
Sickle cell disease (SCD) affects haemoglobin - the molecule in blood cells which carries oxygen. It causes red blood cells to become abnormal crescent (or sickle)- shaped.
- Trial name
- NCT02090881
- Testing
- Ultrasound scan
- Where
- United Kingdom
- Volunteers
- 25 people
- Sponsor
- Imperial College Healthcare NHS Trust
Sickle Cell Anemia, Severe Acute Malnutrition
Management of Severe Acute Malnutrition in SCD, in Northern Nigeria
Except for children with HIV, all recommendations for treatment of childhood malnutrition are for children < 5 years of age. The overall goal of this randomized controlled nutrition feasibility trial is to identify whether…
- Trial name
- NCT03634488
- Testing
- hydroxyurea (20mg/kg/day), Ready-to-use therapeutic food
- Where
- 3 sites in United States, Nigeria
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
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 Disease
Study of Vitamin D in Children With Sickle Cell Disease
This pilot study aims to answer the question whether monthly oral vitamin D3 supplementation, 100,000 IU, will be safe and effective in raising serum 25-hydroxyvitamin D (form of vitamin D measured in the blood) to levels…
- Trial name
- NCT01276587
- Testing
- Vitamin D3
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Gary M Brittenham, MD
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 Disease
Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes.
- Trial name
- SCURT (NCT00745420)
- Testing
- Hematopoietic Stem Cell Transplantation
- Where
- 19 sites in United States
- Volunteers
- 30 people
- Sponsor
- Medical College of Wisconsin
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 Anemia, Children, Only
Shared-Decision Making for Hydroxyurea
The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent…
- Trial name
- ENGAGE-HU (NCT03442114)
- Testing
- Hydroxyurea SDM Toolkit, Clinician Pocket Guide
- Where
- 11 sites in United States
- Volunteers
- 176 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Anemia, Sickle Cell
Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA.
- Trial name
- NCT00890396
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 163 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Omega-3 Fatty Acids in Sickle Cell Disease
The purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain…
- Trial name
- NCT02947100
- Testing
- SCD-Omegatex™
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Robin E. Miller
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
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
Anemia, Sickle Cell
Trial of Oral Glutamine in Patients With Sickle Cell Anemia
Children with sickle cell anemia (SCA) seem to have higher energy needs than children who do not have the disease. This may be the reason why children and teenagers with sickle cell anemia tend to be smaller, weigh less, and have…
- Trial name
- NCT00131508
- Testing
- Glutamine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- St. Jude Children's Research Hospital