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MinorStudy completion date: Jul 2029 to Jan 2030v25
Minor4 versionsRe-verified since May 14, 2020, no change to tracked fieldsv21-v24
MinorPublications: 9 to 10v20
MinorRe-verified, no change to tracked fieldsv19
NotableResults first posted: Mar 26, 2018v18Show 6 more changesShow less
Primary endpoint revised
Interventions: AAV2-hRPE65v2 (Biological) to AAV2-hRPE65v2,voretigene neparvovec-rzyl (Biological)
Eligibility criteria shortened, about 9 words
Secondary endpoints: 2 to 3
Publications: 8 to 9
Study description expanded, about 2 words
Show 16 earlier versionsHide 16 earlier versions
MinorRe-verified, no change to tracked fieldsv17
MinorEnrollment target: estimated 24 to confirmed 31v16
Minor2 versionsRe-verified since Feb 16, 2016, no change to tracked fieldsv14-v15
MinorPrimary completion date changed from estimate of Jun 2015 to Jul 2015
Study completion date: Apr 2029 to Jul 2029
v13
MinorTrial status: Recruiting to Active, Not Recruiting
Primary completion date: Apr 2015 to Jun 2015
Site details revised at 2 of 2 sites
v12
NotableLead sponsor: Children's Hospital of Philadelphia to Spark Therapeutics, Inc.
Collaborators: 1 to 2
Eligibility criteria expanded, about 5 words
Study description expanded, about 1 word
v11
MinorSite contacts updated at 1 of 2 sitesv10
MinorSite details revised at 2 of 2 sitesv9
NotableTrial status: Not Yet Recruiting to Recruitingv8Show 7 more changesShow less
Start date: Aug 2012 to Oct 2012
Primary completion date: Dec 2014 to Apr 2015
Study completion date: Dec 2028 to Apr 2029
Eligibility criteria expanded, about 141 words
Study description expanded, about 11 words
Site details revised at 2 of 2 sites
Publications: 7 to 8
NotableStart date: Mar 2012 to Aug 2012v7
NotableEnrollment target: 12 to 24v6Show 8 more changesShow less
Primary completion date: Mar 2018 to Dec 2014
Primary endpoint revised
Study completion date: Sep 2027 to Dec 2028
Eligibility criteria expanded, about 159 words
Secondary endpoints revised (2)
Study description expanded, about 24 words
Publications: 5 to 7
Study arms: 1 to 2
NotableStart date: Sep 2011 to Mar 2012
Primary completion date: Sep 2017 to Mar 2018
Publications: 4 to 5
v5
NotablePrimary completion date: Jan 2017 to Sep 2017
Start date: Jan 2011 to Sep 2011
Study completion date: Jan 2027 to Sep 2027
v4
NotablePrimary completion date: Aug 2012 to Jan 2017
Start date: Aug 2010 to Jan 2011
Study completion date: Aug 2026 to Jan 2027
v3
NotablePrimary completion date: Jan 2012 to Aug 2012
Start date: Jan 2010 to Aug 2010
Study completion date: Jan 2012 to Aug 2026
v2
MinorStudy 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.
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.