Table II.
Human Clinical Trials for Neuronal Ceroid Lipofuscinoses (Observational clinical studies)1
| NCL/Study Title | Treatment | Phase | Number Enrolled | Status | NCT Number2 |
|---|---|---|---|---|---|
| All CLN types | |||||
| Clinical and Neuropsychological Investigations in Batten Disease | Natural history only, no treatment | none (observational) | 400 | Recruiting | NCT01873924 |
| Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities | Natural history only, no treatment | none (observational) | 57 | Completed | NCT01966757 |
| Genetic Characterization of Movement Disorders and Dementias | Natural history only, no treatment | none (observational) | 12,000 | Recruiting | NCT02014246 |
| Inherited Retinal Degenerative Disease Registry | Natural history only, no treatment | none (observational) | 20,000 | Recruiting | NCT02435940 |
| Longitudinal Study of Neurodegenerative Disorders | Hematopoietic Stem Cell Transplantation | none (observational) | 1,500 | Recruiting | NCT03333200 |
| CLN2 | |||||
| Genotype - Phenotype Correlations of LINCL | Natural history only, no treatment | none (observational) | 18 | Completed | NCT00151268 |
| Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis | Natural history only, no treatment | none (observational) | 75 | Active, not recruiting | NCT01035424 |
| Collection of Cerebrospinal Fluid in Healthy Children | none (normal controls) | none (observational) | 32 | Terminated | NCT01698229 |
| CLN3 | |||||
| Investigations of Juvenile Neuronal Ceroid Lipofuscinosis | Natural history only, no treatment | none (observational) | 50 | Recruiting | NCT03307304 |
| CLN6 | |||||
| Natural History of Neuronal Ceroid Lipofuscinosis, Batten’s CLN6 Disease | Natural history only, no treatment | none (observational) | 15 | Recruiting | NCT03285425 |
1
Abbreviations: neuronal ceroid lipofuscinosis (NCL); late infantile neuronal ceroid lipofuscinosis (LINCL)
2
Clinical identifier numbers obtained from clinicaltrials.gov website