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Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

NCT04273243 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 10

Last updated 2025-10-02

No results posted yet for this study

Summary

This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501.

Conditions

  • CLN6
  • Batten Disease

Interventions

GENETIC

AT-GTX-501

No study drug is administered in this study. Subjects who received AT-GTX-501 in a previous trial will be evaluated in this trial for long-term safety and efficacy.

Sponsors & Collaborators

  • Emily de los Reyes

    lead OTHER

Principal Investigators

  • Emily de los Reyes, MD · Nationwide Children's Hospital

Eligibility

Min Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-24
Primary Completion
2026-12-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT04273243 on ClinicalTrials.gov