Track this trial free. DataLookout checks ClinicalTrials.gov daily and emails you which fields changed on NCT00481546 (status, enrollment, completion dates, endpoints, sites) when this record is updated. Free accounts track up to 5 trials.

Track this trial

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations

Study acronym: LCA
StatusCompleted
PhasePhase 1
Started
View on ClinicalTrials.gov ↗

Record History

  1. Trial status: Active, Not Recruiting to Completed Primary and study completion dates changed from estimate of Jun 2026 to Dec 2016v21 Show all 2 changesShow less
    • Primary and study completion dates changed from estimate of Jun 2026 to Dec 2016
  2. 8 versions Re-verified since Aug 23, 2017, no change to tracked fieldsv13-v20
  3. Lead sponsor: Samuel G. Jacobson to University of Pennsylvaniav12
  4. Lead sponsor: University of Pennsylvania to Samuel G. Jacobson Study arms: 0 to 1v11 Show all 2 changesShow less
    • Study arms: 0 to 1
  5. Trial status: Recruiting to Active, Not Recruiting Site details revised at 2 of 2 sites · Publications: details revised at 1 of 17 entriesv10 Show all 3 changesShow less
    • Site details revised at 2 of 2 sites
    • Publications: details revised at 1 of 17 entries
Show 8 earlier versionsHide 8 earlier versions
  1. Enrollment target: 9 to 15 Primary and study completion dates: Jul 2022 to Jun 2026 · Secondary endpoints: details revised at 1 of 1 entry · Publications: 15 to 17 · 1 morev9 Show all 5 changesShow less
    • Primary and study completion dates: Jul 2022 to Jun 2026
    • Secondary endpoints: details revised at 1 of 1 entry
    • Publications: 15 to 17
    • Eligibility criteria expanded, about 1 word

    See this version in full on ClinicalTrials.gov ↗

  2. Re-verified, no change to tracked fieldsv8
  3. Publications: 13 to 15v7 Show moreShow less

    See this version in full on ClinicalTrials.gov ↗

  4. Publications: 9 to 13v6 Show moreShow less

    See this version in full on ClinicalTrials.gov ↗

  5. Re-verified, no change to tracked fieldsv5
  6. Primary completion date: Jul 2022v4
  7. Completion date: Jul 2022 Publications: 8 to 9v3 Show all 2 changesShow less
    • Publications: 8 to 9

    See this version in full on ClinicalTrials.gov ↗

  8. Trial status: Not Yet Recruiting to Recruiting Start date: Jul 2007 · Site details revised at 2 of 2 sitesv2 Show all 3 changesShow less
    • Start date: Jul 2007
    • Site details revised at 2 of 2 sites
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber congenital amaurosis (LCA), an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-CBSB-hRPE65 administration to individuals with RPE65-associated retinal disease. Five cohorts will be included in this trial. Cohorts 1, 2 and 4 will consist of individuals 18 years of age and older. Cohorts 3 and 5 will consist of individuals between the ages of 8 and 17, inclusive. Enrollment in Cohorts 3 and 5 will begin only after confirming the safety of rAAV2-CBSB-hRPE65 administration in the older groups of participants. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in RPE65-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention. The goal of this clinical trial is to determine the safety of uniocular subretinal administration of rAAV2-CBSB-hRPE65 in individuals with RPE65-associated retinal disease. Ocular and systemic toxicity will be assessed prior to and following vector administration to determine if there are adverse changes that may be associated with vector administration.
Trial Details
NCT Number NCT00481546
Lead Sponsor University of Pennsylvania
Collaborators: National Eye Institute (NEI)
Conditions Amaurosis of Leber, Retinal Diseases
Enrollment 15 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov