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Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)

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ClinicalTrials.gov Identifier: NCT02781480
Recruitment Status : Completed
First Posted : May 24, 2016
Results First Posted : April 8, 2021
Last Update Posted : July 12, 2021
Sponsor:
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Tracking Information
First Submitted Date  ICMJE April 28, 2016
First Posted Date  ICMJE May 24, 2016
Results First Submitted Date  ICMJE March 10, 2021
Results First Posted Date  ICMJE April 8, 2021
Last Update Posted Date July 12, 2021
Study Start Date  ICMJE April 2016
Actual Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 9, 2021)
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event [ Time Frame: 6 months ]
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related:
  • Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more.
  • Severe unresponsive inflammation.
  • Infective endophthalmitis.
  • Ocular malignancy.
  • Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
Original Primary Outcome Measures  ICMJE
 (submitted: May 23, 2016)
Number of participants with treatment-related ocular adverse events graded as III & IV in CTCAE Version 4.0 grading scale. [ Time Frame: 1 year ]
The primary outcome is safety of subretinal administration of AAV2/5-OPTIRPE65. Safety is defined as the absence of ocular Grade III (sustained reduction of visual acuity to counting fingers or less, severe unresponsive inflammation, infective endophthalmitis) or Grade IV (loss of light perception, ocular malignancy) adverse events and severe non-ocular Suspected Unexpected Serious Adverse Reaction. Safety will be assessed for 6 months after the intervention.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: May 23, 2016)
  • Number of participants with improvement in visual function as shown in several visual assessments including Visual Acuity & Visual Mobility & also retinal function as shown in electroretinography (ERG) assessments. [ Time Frame: 1 year ]
    The secondary outcomes are measures of the efficacy of the intervention, which will be performed on an individual participant basis and will be descriptive in nature. Any improvement in patient's visual function from baseline that is greater that the test-retest variation for that test and is sustained for at least two consecutive assessments.
  • Number of participants with improvement in visual function as shown in several visual assessments including Visual Acuity & Visual Mobility & also retinal function as shown in electroretinography (ERG) assessments. [ Time Frame: 1 year ]
    Any improvement in patient's retinal function from baseline that is measurable by electroretinography (ERG).
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Official Title  ICMJE An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
Brief Summary A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Detailed Description A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:
AAV2/5-OPTIRPE65
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Leber Congenital Amaurosis
Intervention  ICMJE Biological: AAV RPE65
Comparison of different dosages of AAV RPE65
Study Arms  ICMJE
  • Experimental: Low dose AAV-RPE65
    Subretinal administration of a single low dose of range AAV-RPE65
    Intervention: Biological: AAV RPE65
  • Experimental: Intermediate dose AAV-RPE65
    Subretinal administration of a single intermediate dose of range AAV-RPE65
    Intervention: Biological: AAV RPE65
  • Experimental: High dose AAV-RPE65
    Subretinal administration of a single high dose of range AAV-RPE65
    Intervention: Biological: AAV RPE65
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 15, 2019)
15
Original Estimated Enrollment  ICMJE
 (submitted: May 23, 2016)
27
Actual Study Completion Date  ICMJE December 2018
Actual Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Aged 3 years or older
  • Early-onset severe retinal dystrophy consistent with RPE65 deficiency

Key Exclusion Criteria:

  • Females who are pregnant or breastfeeding
  • Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02781480
Other Study ID Numbers  ICMJE MGT003
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party MeiraGTx UK II Ltd
Study Sponsor  ICMJE MeiraGTx UK II Ltd
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account MeiraGTx UK II Ltd
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP