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

Trial Profile

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

Recruiting
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
  • Most Recent Events

    • 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
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