An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Ketogenic Diet in Children With Malignant or Recurrent/Refractory Brain Tumor

NCT03328858 ·Status: TERMINATED ·Phase: NA

Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3

NCT06184503 ·Status: RECRUITING

Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

NCT02187809 ·Status: TERMINATED ·Phase: PHASE3

Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

NCT02174094 ·Status: WITHDRAWN ·Phase: PHASE3

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

NCT01399047 ·Status: COMPLETED ·Phase: PHASE2

Natural History Study in Pediatric Patients with STXBP1 Encephalopathy with Epilepsy

NCT05462054 ·Status: WITHDRAWN

Surveillance of Zika-related Microcephaly in Sub-Saharan Africa and Asia

NCT03922594 ·Status: TERMINATED

Management of Myelomeningocele Study (MOMS)

NCT00060606 ·Status: COMPLETED ·Phase: NA

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome

NCT02036853 ·Status: COMPLETED ·Phase: PHASE2

Management of Drug-resistant Epilepsy in Infants and Children: a Real-life Study of the Use of the Ketogenic Diet in France and the KetoCal® Range in Its Maintenance

NCT06075485 ·Status: RECRUITING

Evaluating Dietary Intervention Before surgicaL Treatment for Epilepsy

NCT02261753 ·Status: TERMINATED ·Phase: NA

Valproic Acid-associated Hypoalbuminemia in Medically Fragile Patients

NCT00723762 ·Status: COMPLETED

Gadopiclenol Pharmacokinetics, Safety and Efficacy in Children < 2 Years of Age

NCT05590884 ·Status: COMPLETED ·Phase: PHASE2

Non-interventional Study in Pediatric Patients Treated With NOVOCART Inject in the Knee

NCT05391841 ·Status: WITHDRAWN

A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

NCT06422377 ·Status: TERMINATED ·Phase: PHASE3

Predictors of Drug Resistant Epilepsy Among Pediatric Patients

NCT07241754 ·Status: NOT_YET_RECRUITING

Effectiveness and Impact on the Quality of Life of Ketogenic Diet in Pediatric Patients

NCT06309251 ·Status: RECRUITING

Ultra High Field MRI of Focal Pediatric Epilepsy

NCT03318237 ·Status: TERMINATED

A Multicenter Selective Screening Study to Investigate the Frequency of Neuronal Ceroid Lipofuxinosis Type 2 (CLN2)

NCT06128226 ·Status: RECRUITING

A Phase 2 Study of Radiprodil in Subjects With Drug-resistant Infantile Spasms (IS)

NCT02829827 ·Status: TERMINATED ·Phase: PHASE2

Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial

NCT01619644 ·Status: COMPLETED ·Phase: PHASE2

An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome

NCT02023424 ·Status: UNKNOWN ·Phase: PHASE1

Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome

NCT00272844 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma

NCT04448873 ·Status: COMPLETED ·Phase: PHASE4

A Study to Investigate Efficacy and Safety of KP-001 Compared With Placebo in Patients Aged ≥2 Years With Common VM, Common LM, or KTS/CLOVES Syndrome

NCT07285005 ·Status: NOT_YET_RECRUITING ·Phase: PHASE3