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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

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Leber Congenital Amaurosis
Intervention Biological: AAV RPE65
Enrollment 15
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Low Dose AAV-RPE65 Intermediate Dose AAV-RPE65 High Dose AAV-RPE65
Hide Arm/Group Description Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at low dose (1 x 10^11 vg/mL) Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL) Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL)
Period Title: Overall Study
Started 9 3 3
Completed 9 3 3
Not Completed 0 0 0
Arm/Group Title Low Dose AAV-RPE65 Intermediate Dose AAV-RPE65 High Dose AAV-RPE65 Total
Hide Arm/Group Description Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at low dose (1 x 10^11 vg/mL) Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL) Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL) Total of all reporting groups
Overall Number of Baseline Participants 9 3 3 15
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants 3 participants 3 participants 15 participants
12.8  (5.52) 19.0  (2.65) 21.0  (3.00) 15.7  (5.79)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 3 participants 3 participants 15 participants
Female
7
  77.8%
2
  66.7%
0
   0.0%
9
  60.0%
Male
2
  22.2%
1
  33.3%
3
 100.0%
6
  40.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 3 participants 3 participants 15 participants
Hispanic or Latino
0
   0.0%
1
  33.3%
0
   0.0%
1
   6.7%
Not Hispanic or Latino
9
 100.0%
2
  66.7%
3
 100.0%
14
  93.3%
Black or African American
0
   0.0%
0
   0.0%
1
  33.3%
1
   6.7%
Asian
2
  22.2%
0
   0.0%
0
   0.0%
2
  13.3%
White
7
  77.8%
3
 100.0%
2
  66.7%
12
  80.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 9 participants 3 participants 3 participants 15 participants
United States 2 0 0 2
United Kingdom 7 3 3 13
1.Primary Outcome
Title Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Hide Description

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).
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Low Dose AAV-RPE65 Intermediate Dose AAV-RPE65 High Dose AAV-RPE65
Hide Arm/Group Description:
Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at low dose (1 x 10^11 vg/mL)
Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL)
Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL)
Overall Number of Participants Analyzed 9 3 3
Measure Type: Count of Participants
Unit of Measure: Participants
Number of subjects with dose limiting events.
0
   0.0%
0
   0.0%
1
  33.3%
Reduction in visual acuity by 15 ETDRS
0
   0.0%
0
   0.0%
1
  33.3%
Severe unresponsive inflammation
0
   0.0%
0
   0.0%
0
   0.0%
Infective endophthalmitis
0
   0.0%
0
   0.0%
0
   0.0%
Ocular malignancy
0
   0.0%
0
   0.0%
0
   0.0%
Grade III or above non-ocular SUSAR
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame Within 6 months of ATIMP administration.
Adverse Event Reporting Description A serious adverse event that started on or after the start of the administration of ATIMP. Any serious adverse events that occurred after the last administration of ATIMP + 6 months were not considered treatment emergent.
 
Arm/Group Title Low Dose AAV-RPE65 Intermediate Dose AAV-RPE65 High Dose AAV-RPE65
Hide Arm/Group Description Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at low dose (1 x 10^11 vg/mL) Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL) Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL)
All-Cause Mortality
Low Dose AAV-RPE65 Intermediate Dose AAV-RPE65 High Dose AAV-RPE65
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/9 (0.00%)      0/3 (0.00%)      0/3 (0.00%)    
Hide Serious Adverse Events
Low Dose AAV-RPE65 Intermediate Dose AAV-RPE65 High Dose AAV-RPE65
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/9 (0.00%)      2/3 (66.67%)      1/3 (33.33%)    
Eye disorders       
Uveitis  [1]  0/9 (0.00%)  0 2/3 (66.67%)  2 1/3 (33.33%)  1
Visual acuity reduced  [1]  0/9 (0.00%)  0 0/3 (0.00%)  0 1/3 (33.33%)  1
Indicates events were collected by systematic assessment
[1]
ATIMP Related
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Low Dose AAV-RPE65 Intermediate Dose AAV-RPE65 High Dose AAV-RPE65
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/9 (55.56%)      2/3 (66.67%)      3/3 (100.00%)    
Eye disorders       
Uveitis  [1]  3/9 (33.33%)  3 1/3 (33.33%)  1 1/3 (33.33%)  2
Visual acuity reduced  [1]  2/9 (22.22%)  2 0/3 (0.00%)  0 1/3 (33.33%)  1
Anterior chamber inflammation  [1]  0/9 (0.00%)  0 0/3 (0.00%)  0 1/3 (33.33%)  1
Hypotony of eye  [1]  1/9 (11.11%)  1 0/3 (0.00%)  0 0/3 (0.00%)  0
Blurred vision  [1]  0/9 (0.00%)  0 1/3 (33.33%)  1 0/3 (0.00%)  0
Investigations       
Intraocular pressure increased  [1]  1/9 (11.11%)  1 0/3 (0.00%)  0 0/3 (0.00%)  0
Nervous system disorders       
Visual field defect  [1]  0/9 (0.00%)  0 1/3 (33.33%)  1 0/3 (0.00%)  0
Indicates events were collected by systematic assessment
[1]
ATIMP Related
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
 
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Chief Medical Officer
Organization: MeiraGTx
Phone: +1 973 609-2156
EMail: robert.zeldin@meiragtx.com
Layout table for additonal information
Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT02781480    
Other Study ID Numbers: MGT003
First Submitted: April 28, 2016
First Posted: May 24, 2016
Results First Submitted: March 10, 2021
Results First Posted: April 8, 2021
Last Update Posted: July 12, 2021