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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Study for the Treatment for CLN7 Disease
NCTID
NCT04737460
(View at clinicaltrials.gov)
Description
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).
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Development Status
Active
Indication
CLN7 Batten Disease
Disease Ontology Term
DOID:0110722
Compound Name
AAV9/CLN7
Sponsor
Benjamin Greenberg
Funder Type
Other
Recruitment Status
Active not recruiting
Enrollment Count
4
Results Posted
Not Available
Therapy Information
Target Gene/Variant
MFSD8
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intrathecal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV9
Editor Type
none
Dose 1
5E14 vg
Dose 2
1E15 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2021-01-15
Completion Date
2029-02-01
Last Update
2024-12-13
Participation Criteria
Eligible Age
1 Year - 18 Years
Standard Ages
Child, Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease