🏛️ Verified NIH Record • Updated 2024-08-09
Gene Therapy Study for Children With CLN5 Batten Disease (NCT05228145)
A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease
🔵 Active, Not Recruiting Phase1 / Phase2 📋 Clinical Research Protocol Sponsor: Neurogene Inc.
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of Rochester — Rochester, New York
Age range: 3 Years to 9 Years
🧪 Interventions in This Study
- NGN-101 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (2 Sites)
- University of Rochester — Rochester, New York
- Great Ormond Street Hospital for Children — London,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
Explore by condition: All Neuronal Ceroid Lipofuscinosis CLN5 clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.