Clinical trials: Hemoglobinopathy in Children
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
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
Thalassemia Major
Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload…
- Trial name
- NCT05303506
- Testing
- serum ferritin and AST
- Volunteers
- 50 people
- Sponsor
- Assiut University
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
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
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
Beta-Thalassemia
Iron Status in BTM With Blood Transfusion
To assess the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.
- Trial name
- NCT04947540
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assiut 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
Sickle Cell Disease
Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease
The proposed research study is a cross-sectional study enrolling young children with sickle cell disease between 5 and 12 years of age.
- Trial name
- NCT01895998
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Duke 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
Anesthesia, Pediatric Cancer
the Efficacy and Safety of Sugammadex in Children 0-2 Years Old
Sugammadex is a selective antidote to muscle relaxants rocuronium bromide and vecuronium bromide. Sugammadex is a modified gamma-cyclodextrin, a compound that selectively binds rocuronium bromide and vecuronium bromide.
- Conditions
- Anesthesia, Pediatric Cancer, Pediatric Tumor, Pediatric Hepatoblastoma
- Trial name
- NCT03728543
- Testing
- Sugammadex
- Where
- Russia
- Volunteers
- 62 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Sickle Cell Anemia
Bone Marrow Transplantation in Treating Children With Sickle Cell Disease
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ…
- Trial name
- NCT00004485
- Testing
- cyclosporine, fludarabine, mycophenolate mofetil
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease, Sickle Cell Anemia in Children
Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study
NOHARM MTD is an extension of a previous study for children with Sickle Cell Anemia (SCA) who were enrolled in the NOHARM study.
- Trial name
- NOHARM MTD (NCT07708714)
- Testing
- hydroxycarbamide
- Where
- Uganda
- Volunteers
- 250 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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, Sickle Cell Anemia in Children
PINPOINT: Gaming Technology for SCD Pain
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Thalassemia, Sickle Cell SC Disease
- Trial name
- Pinpoint II (NCT04579926)
- Testing
- Pinpoint app
- Where
- 2 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Sickle Cell Disease
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice…
- Trial name
- KID-BID (NCT06578507)
- Testing
- Hydroxycarbamid
- Where
- 7 sites in France, French Guiana
- Volunteers
- 50 people
- Sponsor
- Theravia
Sickle Cell Disease
Low-Dose Ketamine Infusion for Children With Sickle Cell Disease-Related Pain
Acute pain episodes associated with sickle cell disease (SCD) are very difficult to manage effectively. Opioid tolerance and side effects have been major roadblocks in our ability to provide these patients with adequate pain…
- Trial name
- NCT00595530
- Testing
- ketamine
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Connecticut Children's Medical Center
Sickle Cell Anemia
Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia
The purpose of this study is to use comprehensive exercise testing to examine longitudinal changes in exercise capacity over a 2 year period in children and young adults with sickle cell anemia.
- Trial name
- NCT01558076
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Conditions
- Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders
- Trial name
- FAM BMT (NCT03128996)
- Testing
- RIC regimen, GVHD prophylaxis regimen
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications…
- Trial name
- R34 pK/PD (NCT02447874)
- Testing
- Arginine, Arginine (Loading), Arginine (Continuous)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Emory University