Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- CLN3
- Sponsor
- Alcyone Therapeutics, Inc
- Enrollment
- 7
- Locations
- 1
- Primary Endpoint
- Safety evaluation based on the development of dose-limiting toxicity (DLT).
- Status
- Active, not recruiting
- Last Updated
- 8 months ago
Overview
Brief 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 info@alcyonetx.com
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Safety evaluation based on the development of dose-limiting toxicity (DLT).
Time Frame: 36 Months
The DLT is defined as any unanticipated AE that is considered related to CLN-301 and is Common Terminology Criteria for Adverse Events Grade 3 or higher.
Efficacy: Change in rating as determined using the Unified Batten Disease Rating Scale (UBDRS) rating scale.
Time Frame: 36 months
The UBDRS is a clinical ratings instrument used specifically to assess motor, seizure, behavioral and functional capabilities. The "Physical Assessment" is a 20 item subscale that measures vision, speech, motor strength, gait, abnormal involuntary movements and balance. Each item has a score range of 0 to 4. The minimum score is 0 and the maximum score is 112. The items are summed up to obtain a total score.The higher the score, the more severe the disability and worse the outcome.
Secondary Outcomes
- QOL: Change in Quality of Life (QOL) as determined using the Pediatric Quality of Life (PedsQL™) scale.(36 months)
- Seizures: Change is seizure subscore as determined using Seizure subscale of the UBDRS scale.(36 months)
- Global impression: Change in disease severity using the UBDRS clinical global impression (CGI) subscale.(36 months)