Gene Therapy Study for Children With CLN5 Batten Disease
Public ClinicalTrials.gov record NCT05228145. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05228145
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 6 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 3 Years to 9 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 30, 2022
- Primary completion
- Oct 31, 2028
- Completion
- Oct 31, 2028
- Last update posted
- Aug 11, 2024
2022 – 2028
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Rochester | Rochester | New York | 14642 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
Recently updated Neuronal Ceroid Lipofuscinosis CLN5 trials
Other public records listing Neuronal Ceroid Lipofuscinosis CLN5. Sorted by last update posted; newest first. Not a recommendation.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05228145, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 11, 2024 · Synced Jul 27, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05228145 live on ClinicalTrials.gov.