2 recruiting. .
Follow Kufor-Rakeb syndrome: new trials by email Literature Review of Kufor-Rakeb syndrome trials Research Report on Kufor-Rakeb syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database Natural History - Neuronal Ceroid Lipofuscinosis, Batten Disease | Recruiting | Universitätsklinikum Hamburg-Eppendorf | 500 | 2020-04-08 | 2021-10-29 | |
| TIRCON International NBIA Registry Neurodegeneration With Brain Iron Accumulation (NBIA), Pantothenate Kinase-associated Neurodegeneration (PKAN) | Recruiting | LMU Klinikum | 2000 | 2012-06-14 | 2025-06-05 |
Ceroid lipofuscinosis, neuronal 5 (1) · Familial apoceruloplasmin deficiency (1) · Fatty Acid Hydroxylase-Associated Neurodegeneration (1) · Lysosomal Storage Diseases (1) · Metabolic Diseases (1) · Neuroaxonal Dystrophies (1) · Neurodegenerative Diseases (1) · Neuroferritinopathy (1) · Neuronal Ceroid-Lipofuscinoses (1) · Pantothenate Kinase-Associated Neurodegeneration (1) · Woodhouse Sakati syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Heredodegenerative Disorders, Nervous System (2) · Nervous System Diseases (2) · Basal Ganglia Diseases (1) · Brain Diseases (1) · Central Nervous System Diseases (1) · Lipid Metabolism Disorders (1) · Lipid Metabolism, Inborn Errors (1) · Lipidoses (1) · Metabolism, Inborn Errors (1) · Movement Disorders (1) · Neurodegenerative Diseases (1) · Nutritional and Metabolic Diseases (1)
LMU Klinikum (1) · Universitätsklinikum Hamburg-Eppendorf (1)
Germany (2) · Canada (1) · Czechia (1) · Italy (1) · Netherlands (1) · Poland (1) · Serbia (1) · Spain (1)
Angela Schulz, MD, PhD (1) · Thomas Klopstock, Prof. Dr. (1)