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Gene Therapy Study for Children With CLN5 Batten Disease

NCT05228145 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-08-12

No results posted yet for this 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.

Conditions

  • Neuronal Ceroid Lipofuscinosis CLN5

Interventions

GENETIC

NGN-101

Participants with confirmed mutations in the CLN5 gene who meet all the inclusion and none of the exclusion criteria will be treated with a single intracerebroventricular (ICV) dose and a single intravitreal (IVT) dose of the study treatment.

Sponsors & Collaborators

  • Neurogene Inc.

    lead INDUSTRY

Principal Investigators

  • Effie Albanis, MD · Neurogene Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Years
Max Age
9 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-31
Primary Completion
2028-11-30
Completion
2028-11-30
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05228145 on ClinicalTrials.gov