Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-25 @ 2:11 AM
Ignite Modification Date:
2025-12-25 @ 2:11 AM
NCT ID:
NCT04737460
Brief 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).
Study:
NCT04737460
Study Brief:
Protocol Section:
NCT04737460