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NCT04737460 · ClinicalTrials.gov registry record · Phase 1
Study for the Treatment for CLN7 Disease
A Phase 1 study of CLN7, sponsored by Benjamin Greenberg.
- Active
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
- 1
- Study location
NCT04737460: Active Phase 1 study of CLN7, sponsored by Benjamin Greenberg.
NCT04737460 is a Phase 1 study of CLN7 that is active but no longer recruiting, run by Benjamin Greenberg. The registered enrollment target is 4 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04737460, a Phase 1 study of CLN7, is active but no longer recruiting, sponsored by Benjamin Greenberg.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a phase 1 open-label, single-administration of gene therapy agent AAV9/CLN7, administered intrathecally into the lumbar spinal cord region of pediatric patients with CLN7 Batten disease. This study consists of a one-time injection of AAV9/CLN7. There are two Cohorts with a low dose and a high dose. The primary objective for this clinical study is to evaluate safety. The secondary objective is to determine the efficacy of AAV9/CLN7. The secondary outcome measures include motor, cognition and intelligence assessments. The exploratory outcome measures include visual impairment assessment, cognitive evaluations, Brain magnetic resonance imaging (MRI), electroencephalogram (EEG), electrocardiogram (ECG) and echocardiogram (ECHO).
Primary Outcome
Adverse event collection at scheduled visits. Lab results at scheduled visits. MRI Brain at Days 90, 180, 360, 720. Lumbar Puncture with Cerebral Spinal Fluid analysis at Days 90, 180, 360 and 720.
Conditions Studied
Interventions
- GENETIC AAV9/CLN7
Study Locations (1)
Texas
- Children' Health - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2021-05-04 |
| Est. Completion | 2029-02-01 |
| Phase | Phase 1 |
What the registry record for NCT04737460 still lists
NCT04737460 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower).
The record links to 1 condition, with CLN7 appearing as the primary indexed condition, and to 1 intervention - of which AAV9/CLN7 is the first listed.
NCT04737460 reports a single indexed study location in Texas.
Frequently Asked Questions
What is clinical trial NCT04737460 about?
NCT04737460 is a clinical study titled "Study for the Treatment for CLN7 Disease". This is a phase 1 open-label, single-administration of gene therapy agent AAV9/CLN7, administered intrathecally into the lumbar spinal cord region of pediatric patients with CLN7 Batten disease. This study consists of a one-time injection of AAV9/CLN7. There are two Cohorts with a low dose and a hi...
What is the current status of trial NCT04737460?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2021-05-04. Estimated completion is 2029-02-01.
What conditions does trial NCT04737460 study?
This clinical trial studies the following conditions: CLN7.
What interventions are being tested in trial NCT04737460?
The interventions under investigation include: AAV9/CLN7 (GENETIC).
Who is sponsoring clinical trial NCT04737460?
This trial is sponsored by Benjamin Greenberg, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04737460 being conducted?
This trial has 1 study location across Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04737460's enrollment target sits among peer trials
4 1995th of 2000 higher than 2 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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