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Gene Therapy for Achromatopsia (CNGA3) (CNGA3)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03758404
Recruitment Status : Completed
First Posted : November 29, 2018
Results First Posted : February 11, 2022
Last Update Posted : February 11, 2022
Sponsor:
Collaborator:
Janssen Research & Development, LLC
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Achromatopsia
Intervention Biological: adeno-associated virus vector AAV- CNGA3
Enrollment 11
Recruitment Details Participants were recruited from two medical centers between 12 August 2019 (date first participant signed informed) and 19 November 2020 (date last participant signed informed consent). A total of 11 participants were enrolled in the study.
Pre-assignment Details  
Arm/Group Title Low Dose AAV - CNGA3 Intermediate Dose AAV - CNGA3 High Dose AAV - CNGA3
Hide Arm/Group Description

Subretinal administration of a single low dose adeno-associated virus AAV-CNGA3

AAV-CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single intermediate dose adeno-associated virus AAV-CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single high dose adeno-associated virus AAV-CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Period Title: Overall Study
Started 3 3 5
Completed 3 3 5
Not Completed 0 0 0
Arm/Group Title Low Dose AAV - CNGA3 Intermediate Dose AAV - CNGA3 High Dose AAV - CNGA3 Total
Hide Arm/Group Description

Subretinal administration of a single low dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single intermediate dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single high dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Total of all reporting groups
Overall Number of Baseline Participants 3 3 5 11
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 11 participants
<=18 years
3
 100.0%
3
 100.0%
3
  60.0%
9
  81.8%
Between 18 and 65 years
0
   0.0%
0
   0.0%
2
  40.0%
2
  18.2%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 11 participants
Female
1
  33.3%
0
   0.0%
1
  20.0%
2
  18.2%
Male
2
  66.7%
3
 100.0%
4
  80.0%
9
  81.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 11 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
2
  40.0%
2
  18.2%
Not Hispanic or Latino
3
 100.0%
3
 100.0%
3
  60.0%
9
  81.8%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 11 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
2
  66.7%
1
  33.3%
2
  40.0%
5
  45.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
1
  33.3%
2
  66.7%
3
  60.0%
6
  54.5%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 11 participants
United States 0 0 2 2
United Kingdom 3 3 3 9
1.Primary Outcome
Title Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.
Hide Description

The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone:

  • Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences
  • Severe unresponsive inflammation
  • Infective endophthalmitis
  • Ocular malignancy
  • Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
Time Frame 6 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all enrolled participants.
Arm/Group Title Low Dose AAV - CNGA3 Intermediate Dose AAV - CNGA3 High Dose AAV - CNGA3
Hide Arm/Group Description:

Subretinal administration of a single low dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single intermediate dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single high dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Overall Number of Participants Analyzed 3 3 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
2.Secondary Outcome
Title Improvement in Visual Function
Hide Description Improvements in visual function as assessed by visual acuity
Time Frame 6 Months
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Improvement in Retinal Function
Hide Description Improvements in retinal function as assessed by microperimetry and perimetry
Time Frame 6 Months
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Improvement in Quality of Life
Hide Description Quality of life will be measured by QoL questionnaires
Time Frame 6 Months
Outcome Measure Data Not Reported
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Low Dose AAV - CNGA3 Intermediate Dose AAV - CNGA3 High Dose AAV - CNGA3
Hide Arm/Group Description

Subretinal administration of a single low dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single intermediate dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

Subretinal administration of a single high dose AAV - CNGA3

AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene

All-Cause Mortality
Low Dose AAV - CNGA3 Intermediate Dose AAV - CNGA3 High Dose AAV - CNGA3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/3 (0.00%)   0/5 (0.00%) 
Hide Serious Adverse Events
Low Dose AAV - CNGA3 Intermediate Dose AAV - CNGA3 High Dose AAV - CNGA3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/3 (0.00%)   0/5 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Low Dose AAV - CNGA3 Intermediate Dose AAV - CNGA3 High Dose AAV - CNGA3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/3 (100.00%)   3/3 (100.00%)   5/5 (100.00%) 
Ear and labyrinth disorders       
Ear pain   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Endocrine disorders       
Hyperlipidaemia   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Type 2 diabetes mellitus   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Eye disorders       
Conjunctival haemorrhage   3/3 (100.00%)  3/3 (100.00%)  5/5 (100.00%) 
Visual acuity reduced   3/3 (100.00%)  3/3 (100.00%)  2/5 (40.00%) 
Conjunctival hyperaemia   1/3 (33.33%)  3/3 (100.00%)  2/5 (40.00%) 
Lenticular opacities   1/3 (33.33%)  3/3 (100.00%)  2/5 (40.00%) 
Ocular discomfort   1/3 (33.33%)  1/3 (33.33%)  4/5 (80.00%) 
Eye inflammation   0/3 (0.00%)  0/3 (0.00%)  2/5 (40.00%) 
Eye pain   1/3 (33.33%)  1/3 (33.33%)  0/5 (0.00%) 
Eye pruritus   0/3 (0.00%)  0/3 (0.00%)  2/5 (40.00%) 
Subconjunctival cyst   0/3 (0.00%)  1/3 (33.33%)  1/5 (20.00%) 
Chorioretinal folds   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Foreign body sensation in eyes   0/3 (0.00%)  1/3 (33.33%)  0/5 (0.00%) 
Iridocyclitis   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Photophobia   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Punctate keratitis   0/3 (0.00%)  1/3 (33.33%)  0/5 (0.00%) 
Retinal disorder   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Uveitis   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Vitreous floaters   0/3 (0.00%)  1/3 (33.33%)  0/5 (0.00%) 
Gastrointestinal disorders       
Vomiting   3/3 (100.00%)  0/3 (0.00%)  0/5 (0.00%) 
Diarrhoea   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Nausea   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Immune system disorders       
Seasonal allergy   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Infections and infestations       
Rhinitis   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Injury, poisoning and procedural complications       
Tenon's cyst   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Investigations       
Intraocular pressure increased   1/3 (33.33%)  1/3 (33.33%)  0/5 (0.00%) 
Alanine aminotransferase increased   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Blood glucose increased   0/3 (0.00%)  0/3 (0.00%)  1/5 (20.00%) 
Intraocular pressure decreased   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Slit-lamp tests abnormal   0/3 (0.00%)  1/3 (33.33%)  0/5 (0.00%) 
Metabolism and nutrition disorders       
Hypophosphataemia   1/3 (33.33%)  0/3 (0.00%)  1/5 (20.00%) 
Nervous system disorders       
Syncope   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Epistaxis   1/3 (33.33%)  0/3 (0.00%)  0/5 (0.00%) 
Indicates events were collected by systematic assessment
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: Program Manager
Organization: MeiraGTx
Phone: 020 3866 4320
EMail: CNGA3@meiragtx.com
Layout table for additonal information
Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT03758404    
Other Study ID Numbers: MGT012
First Submitted: November 27, 2018
First Posted: November 29, 2018
Results First Submitted: January 6, 2022
Results First Posted: February 11, 2022
Last Update Posted: February 11, 2022