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NCT04273243 · ClinicalTrials.gov registry record
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
A clinical trial of Batten Disease and CLN6, sponsored by Emily de los Reyes.
- Active
- Registry status
- 10
- Enrollment target
- 1
- Study location
NCT04273243: Active study of Batten Disease and CLN6, sponsored by Emily de los Reyes.
NCT04273243 is a study of Batten Disease and CLN6 that is active but no longer recruiting, run by Emily de los Reyes. The registered enrollment target is 10 participants, below the 2,829-participant average among 11 other Batten Disease trials with a reported enrollment target (100% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04273243, a study of Batten Disease and CLN6, is active but no longer recruiting, sponsored by Emily de los Reyes.
- ACTIVE NOT RECRUITING
- Registry status
- 10 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
All AEs that occur during this study will be classified as treatment-emergent adverse events (TEAEs), as AT-GTX-501 was previously received by all subjects in this study.
Conditions Studied
Interventions
- GENETIC AT-GTX-501
Study Locations (1)
Ohio
- Nationwide Children's Hosptial - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2020-01-24 |
| Est. Completion | 2027-12 |
What the registry record for NCT04273243 still lists
NCT04273243 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 2,829-participant average among 11 other Batten Disease trials with a reported enrollment target (100% lower).
The record links to 2 conditions, with Batten Disease appearing as the primary indexed condition, and to 1 intervention - of which AT-GTX-501 is the first listed.
NCT04273243 reports a single indexed study location in Ohio.
Frequently Asked Questions
What is clinical trial NCT04273243 about?
NCT04273243 is a clinical study titled "Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer". 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.
What is the current status of trial NCT04273243?
This trial is currently active not recruiting. The enrollment target is 10 participants. The study started on 2020-01-24. Estimated completion is 2027-12.
What conditions does trial NCT04273243 study?
This clinical trial studies the following conditions: Batten Disease, CLN6.
What interventions are being tested in trial NCT04273243?
The interventions under investigation include: AT-GTX-501 (GENETIC).
Who is sponsoring clinical trial NCT04273243?
This trial is sponsored by Emily de los Reyes, which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04273243 being conducted?
This trial has 1 study location across Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04273243's enrollment target sits among peer trials
10 11th of 11 higher than 1 of 11 other Batten Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Batten Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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