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Gene Therapy for Children With CLN3 Batten Disease

NCT03770572 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2025-08-07

No results posted yet for this study

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.

Conditions

  • CLN3
  • Batten Disease

Interventions

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

Sponsors & Collaborators

  • Alcyone Therapeutics, Inc

    lead INDUSTRY

Principal Investigators

  • Kathrin C Meyer, PhD · Alcyone Therapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-11-13
Primary Completion
2029-09-30
Completion
2029-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03770572 on ClinicalTrials.gov