Neuronal Ceroid Lipofuscinosis
7
3
3
2
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Treatment Research
5 treatments under study, led by Surgery To Implant Human Cns Stem Cells Hucnssc
Research Leaders
10 sponsors active, led by StemCells, Inc.
Related Conditions
Often studied with 20 related conditions
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 7 trials
100.0%
+13.4% vs benchmark
0%
0 trials in Phase 3/4
0%
0 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 2 completed trials
Clinical Trials (7)
Gene Therapy Trial for CLN6 Batten Disease
Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies
Clinical and Neuropsychological Investigations in Batten Disease
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities
Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
Research Network
Common Treatments
- Surgery To Implant Human Cns Stem Cells Hucnssc1
- Hucnssc1
- Natural History1
- Scaav9cbcln6 Dose 15e14 Vector Genomes1
- Medication To Suppress The Immune System1
Active Sponsors
- StemCells, Inc.2
- The Charlotte and Gwenyth Gray Foundation1
- Bambino Gesù Hospital and Research Institute1
- IRCCS Fondazione Stella Maris1
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta1
Top Investigators
Oregon Health and Science University
IRCCS Fondazione Stella Maris
University of Rochester
Nationwide Children's Hospital
Oregon Health and Science University
Related Conditions
- Batten Disease3
- Battens Disease2
- Neuronal Ceroid Lipofuscinosis Cln62
- Cln4 Disease1
- Neuronal Ceroid Lipofuscinosis Cln11