Track this trial free. DataLookout checks ClinicalTrials.gov daily and emails you which fields changed on NCT04737460 (status, enrollment, completion dates, endpoints, sites) when this record is updated. Free accounts track up to 5 trials.

Track this trial

Study for the Treatment for CLN7 Disease

Study acronym: CLN7
StatusActive, Not Recruiting
PhasePhase 1
Started
View on ClinicalTrials.gov ↗

Record History

See every version on ClinicalTrials.gov ↗

  1. 4 versions Re-verified since Jul 14, 2023, no change to tracked fieldsv7-v10
  2. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised
    v6
  3. 2 versions Re-verified since May 16, 2021, no change to tracked fieldsv4-v5
  4. Trial status: Not Yet Recruiting to Recruiting
    • Start date: May 1, 2021 to May 4, 2021
    • Site details revised
    • Eligibility criteria expanded, about 1 word
    v3
  5. Start date: Feb 1, 2021 to May 1, 2021v2
  6. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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).
Trial Details
NCT Number NCT04737460
Lead Sponsor Benjamin Greenberg
Conditions
(from sponsor)		 CLN7
Related terms Neuronal Ceroid-Lipofuscinoses
Enrollment 4 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov