Batten Disease Clinical Trial
Official title:
Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9
This is a phase 1/2, open-label, single dose, dose-escalation clinical trial to evaluate the safety and efficacy of AT-GTX-502 (previous NCH Code: scAAV9.P546.CLN3) delivered intrathecally into the lumbar spinal cord region of subjects with CLN3 Batten disease.
This is a phase 1/2, 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 clinicaltrials@amicusrx.com or +1 609-662-2000. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT02254863 -
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
|
Phase 1 | |
Completed |
NCT01907087 -
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
|
Phase 1/Phase 2 | |
Recruiting |
NCT03307304 -
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
|
||
Recruiting |
NCT06203106 -
NYSCF Scientific Discovery Biobank
|
||
Completed |
NCT00151268 -
Genotype - Phenotype Correlations of LINCL
|
N/A | |
Recruiting |
NCT02435940 -
Inherited Retinal Degenerative Disease Registry
|
||
Completed |
NCT01035424 -
Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
|
||
Completed |
NCT00151216 -
Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
|
Phase 1 | |
Active, not recruiting |
NCT04273243 -
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
|
||
Recruiting |
NCT03285425 -
Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae
|
||
Completed |
NCT00176904 -
Stem Cell Transplant for Inborn Errors of Metabolism
|
Phase 2/Phase 3 | |
Recruiting |
NCT03333200 -
Longitudinal Study of Neurodegenerative Disorders
|
||
Completed |
NCT02678689 -
A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
|
Phase 2 | |
Completed |
NCT02485899 -
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT05174039 -
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease
|
Phase 1/Phase 2 | |
Terminated |
NCT01698229 -
Collection of Cerebrospinal Fluid in Healthy Children
|
N/A | |
Enrolling by invitation |
NCT03862274 -
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
|
||
Recruiting |
NCT01873924 -
Clinical and Neuropsychological Investigations in Batten Disease
|
||
Completed |
NCT01161576 -
Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)
|
Phase 1 | |
Recruiting |
NCT04613089 -
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
|