Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA to Children With LINCL With Uncommon Genotypes and/or Moderate to Severe Impairment
Phase of Trial: Phase I/II
Latest Information Update: 01 Jun 2017
At a glance
- Drugs CLN2 gene therapy (Primary)
- Indications Neuronal ceroid lipofuscinosis
- Focus Therapeutic Use
- 16 Aug 2012 Planned End Date changed from 1 Dec 2011 to 1 Dec 2022 as reported by ClinicalTrials.gov.
- 16 Aug 2012 Planned number of patients changed from 16 to 8 as reported by ClinicalTrials.gov.
- 15 Aug 2011 New trial record