Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA to Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

Status: Completed

Phase of Trial: Phase I

Latest Information Update: 06 Nov 2021

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At a glance

Drugs CLN2 gene therapy (Primary)
Indications Neuronal ceroid lipofuscinosis
Focus Therapeutic Use

Most Recent Events

31 Jan 2021 Status changed from active, no longer recruiting to completed.
30 Apr 2018 Planned primary completion date changed from 1 Dec 2017 to 1 Dec 2020.
21 Dec 2017 Planned number of patients changed from 16 to 25.

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