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CLN3 clinical trials

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NCT ID: NCT03770572 Active, not recruiting - Batten Disease Clinical Trials

Gene Therapy for Children With CLN3 Batten Disease

Start date: November 13, 2018
Phase: Phase 1/Phase 2
Study type: Interventional

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.