Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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. The trial reports 1 study location across 1 state.
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).
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04737460
The ClinicalTrials.gov registry entry for NCT04737460 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Benjamin Greenberg, which has 1 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.