Study Locations
- University of Rochester Medical Center in Rochester, NY, USA.
Great Ormond Street Hospital, London, United Kingdom
Investigational Gene Therapy
Clinical Trial
for Children with
CLN5 Batten Disease
Contact Information:
Clinical Trial information can be found on
www.ClinicalTrials.gov search #NCT05228145
About the Clinical Trial
What is the title of the gene therapy trial?
This investigational gene therapy clinical trial is titled: A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis Subtype 5 (CLN5) Disease. This gene therapy clinical trial is a first of its kind, designed to address the neurodegeneration and vision loss associated with CLN5 Batten disease.
What is the purpose of the clinical trial?
The purpose of this gene therapy clinical trial is to assess the safety and potential for efficacy of an investigational gene therapy in children 3-9 years old with genetically confirmed Neuronal Ceroid Lipofuscinosis subtype 5 (CLN5) Batten disease.Clinical Trial FAQ
What type of clinical trial is this?
This is a prospective, open-label clinical trial.
- This is a clinical trial where all participants know they are receiving the investigational gene therapy product and will be followed over time.
What is the route of administration into the body?
The investigational gene therapy is given as a single intracerebroventricular (ICV) dose into the brain and a single intravitreal (IVT) dose into one eye. Both doses are given during the same procedure.
How long does the clinical trial last?
Each participant will be followed for safety and efficacy for 5 years after dosing.
- For approximately the first 6 months after the gene therapy is given, the participant will reside close to the clinical trial site to enable the clinical trial doctor to monitor and care for the participant.
- After the initial safety monitoring period, there will be telephone and in-person visits with the gene therapy clinical trial site in decreasing frequency for regular assessments over a 5-year period.
Contact
Families are encouraged to contact Neurogene at:
Healthcare providers may contact Neurogene Inc.