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NCT05228145 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy Study for Children With CLN5 Batten Disease
A Phase 1 study of Neuronal Ceroid Lipofuscinosis CLN5, sponsored by Neurogene.
- Active
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 2
- Study locations
NCT05228145: Active Phase 1 study of Neuronal Ceroid Lipofuscinosis CLN5, sponsored by Neurogene.
NCT05228145 is a Phase 1 study of Neuronal Ceroid Lipofuscinosis CLN5 that is active but no longer recruiting, run by Neurogene. The registered enrollment target is 6 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05228145, a Phase 1 study of Neuronal Ceroid Lipofuscinosis CLN5, is active but no longer recruiting, sponsored by Neurogene.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 2
- Study locations
Study Summary
This is a prospective, non-randomized, open-label, dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype 5 (CLN5) disease.
Primary Outcome
Incidence, type, severity, and frequency of TEAEs
Conditions Studied
Interventions
- GENETIC NGN-101
Study Locations (2)
New York
- University of Rochester - Rochester
Other
- Great Ormond Street Hospital for Children - London
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2022-01-31 |
| Est. Completion | 2028-11 |
| Phase | Phase 1 |
What the registry record for NCT05228145 still lists
NCT05228145 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower).
The record links to 1 condition, with Neuronal Ceroid Lipofuscinosis CLN5 appearing as the primary indexed condition, and to 1 intervention - of which NGN-101 is the first listed.
NCT05228145 reports a single indexed study location in New York, Other.
Frequently Asked Questions
What is clinical trial NCT05228145 about?
NCT05228145 is a clinical study titled "Gene Therapy Study for Children With CLN5 Batten Disease". This is a prospective, non-randomized, open-label, dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype 5 (CLN5) disease.
What is the current status of trial NCT05228145?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2022-01-31. Estimated completion is 2028-11.
What conditions does trial NCT05228145 study?
This clinical trial studies the following conditions: Neuronal Ceroid Lipofuscinosis CLN5.
What interventions are being tested in trial NCT05228145?
The interventions under investigation include: NGN-101 (GENETIC).
Who is sponsoring clinical trial NCT05228145?
This trial is sponsored by Neurogene, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05228145 being conducted?
This trial has 2 study locations across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05228145's enrollment target sits among peer trials
6 1974th of 2000 higher than 12 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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