Ignite Creation Date:
2024-05-06 @ 12:30 PM
Last Modification Date:
2024-10-26 @ 12:59 PM
Study NCT ID:
NCT03770572
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-08-29
First Post:
2018-12-07
Brief Title:
Gene Therapy for Children With CLN3 Batten Disease
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
Phase IIIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis Delivering the CLN3 Gene by Self-Complementary AAV9
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a phase 12 open-label single dose dose-escalation clinical trial to evaluate the safety and efficacy of AT-GTX-502 previous NCH Code scAAV9P546CLN3 delivered intrathecally into the lumbar spinal cord region of subjects with CLN3 Batten disease
Detailed Description:
This is a phase 12 open-label single-dose dose-escalation study of AT-GTX-502 administered intrathecally into the lumbar spinal cord region of pediatric patients with CLN3 Batten disease
This study consists of a one-time injection of AT-GTX-502 with follow-up visits on Day 7 14 21 and 30 followed by every 3 months through 1 year post-dose and then every 6 months through the fifth year There are two Cohorts with a low dose and a high dose
The primary outcome for this clinical study is to evaluate safety The co-primary objective is to determine the efficacy of AT-GTX-502 as measured by United Batten Disease Rating Scale UBDRS physical subscale
The secondary outcome measures include Pediatric Quality of Life PedsQL inventory seizure subscale of the UBDRS and global impression subscale of the UBDRS
The exploratory outcome measures include visual impairment assessment cognitive evaluations Brain magnetic resonance imaging MRI electroencephalogram EEG electrocardiogram ECG and echocardiogram ECHO
For more information about this study please contact Amicus Therapeutics Patient Advocacy at clinicaltrialsamicusrxcom or 1 609-662-2000
This study consists of a one-time injection of AT-GTX-502 with follow-up visits on Day 7 14 21 and 30 followed by every 3 months through 1 year post-dose and then every 6 months through the fifth year There are two Cohorts with a low dose and a high dose
The primary outcome for this clinical study is to evaluate safety The co-primary objective is to determine the efficacy of AT-GTX-502 as measured by United Batten Disease Rating Scale UBDRS physical subscale
The secondary outcome measures include Pediatric Quality of Life PedsQL inventory seizure subscale of the UBDRS and global impression subscale of the UBDRS
The exploratory outcome measures include visual impairment assessment cognitive evaluations Brain magnetic resonance imaging MRI electroencephalogram EEG electrocardiogram ECG and echocardiogram ECHO
For more information about this study please contact Amicus Therapeutics Patient Advocacy at clinicaltrialsamicusrxcom or 1 609-662-2000
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None