NCT03770572

Gene Therapy for Children With CLN3 Batten Disease

Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (estimated)
• Sponsor: Alcyone Therapeutics, Inc · Industry
• Sex: All
• Age: 3 Years – 10 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 1/2, open-label, single dose, dose-escalation clinical trial to evaluate the safety and efficacy of CLN-301 (previous NCH Code: scAAV9.P546.CLN3) delivered intrathecally into the lumbar spinal cord region of subjects with CLN3 Batten disease.

Detailed description

This is a phase 1/2, open-label, single-dose, dose-escalation study of CLN-301 administered intrathecally into the lumbar spinal cord region of pediatric patients with CLN3 Batten disease. This study consists of a one-time injection of CLN-301 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 CLN-301 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 Alcyone Therapeutics at [e-mail]

Conditions

• CLN3
• Batten Disease

Interventions

Type	Name	Description
GENETIC	Low dose CLN-301	Subjects with diagnosis of CLN3 Batten disease will receive a single dose of CLN-301 at low dose
GENETIC	High dose CLN-301	Subjects with diagnosis of CLN3 Batten disease will receive a single dose of CLN-301 at high dose

Timeline

• Start date: 2018-11-13
• Primary completion: 2029-09-30
• Completion: 2029-09-30
• First posted: 2018-12-10
• Last updated: 2025-08-07

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study