Investigational Gene Therapy
Clinical Trial
for Children with
CLN5 Batten Disease

Enrolling Now

For more information call:

 +1 (877) 237-5020

Clinical Trial information can be found on 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 will know they are receiving the investigational gene therapy product and will be followed over time.
Is there an age requirement for participation in the trial?
Yes, the gene therapy clinical trial will enroll children who are 3 to 9 years old, with a genetic diagnosis of Batten disease subtype CLN5.
What is the route of administration into the body?
The investigational gene therapy will be given as a single intracerebroventricular (ICV) dose into the brain and a single intravitreal (IVT) dose into one eye. Both doses will be 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.
Is there a cost to participate?
Trial-related costs and expenses are paid or reimbursed by the clinical trial sponsor, Neurogene. There is a comprehensive travel and expense policy in place for costs and expenses related to the clinical trial. More details of the specific policy can be provided by the clinical trial site.
Is there someone to help with travel arrangements?
Yes, travel and accommodations will be arranged for participants in the clinical trial. The clinical trial staff will provide more details.
Are patients from all geographic areas eligible to enroll in this trial?
Although the clinical trial sites are in the US and the UK, patients from around the world will be considered for enrollment and are encouraged to contact Neurogene for more information +1 (877) 237-5020. Professional translators will be available if needed.

Study Locations

This clinical trial is now enrolling patients at:

  • University of Rochester Medical Center in Rochester, NY, USA.
  • Great Ormond Street Hospital, London, United Kingdom

Patients from around the world will be considered for enrollment and are encouraged to contact Neurogene for more information +1 (877) 237-5020.


Families are encouraged to contact Neurogene at:

Healthcare providers may contact Neurogene Inc.