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| Name | Class |
|---|---|
| Janssen Research & Development, LLC | INDUSTRY |
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A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia
CNGA3 retinal gene therapy for patients with achromatopsia
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low dose adeno-associated virus (AAV) CNGA3 | Experimental | Subretinal administration of a single low dose AAV CNGA3 |
|
| Intermediate dose adeno-associated virus (AAV) CNGA3 | Experimental | Subretinal administration of a single intermediate dose AAV CNGA3 |
|
| High dose adeno-associated virus (AAV) CNGA3 | Experimental | Subretinal administration of a single high dose AAV CNGA3 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| adeno-associated virus vector AAV- CNGA3 | Biological | Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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:
| 6 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Improvements in Visual Function as Assessed by Visual Acuity | Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | 6 Months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| James Bainbridge | Chief Investigator | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kellogg Eye Center | Ann Arbor | Michigan | 48105 | United States | ||
| Moorfields Eye Hospital NHS Foundation Trust |
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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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Low Dose AAV - CNGA3 | Subretinal administration of a single low dose adeno-associated virus AAV-CNGA3 AAV-CNGA3: AAV gene therapy for defects in CNGA3 gene |
| FG001 | Intermediate Dose AAV - CNGA3 | Subretinal administration of a single intermediate dose adeno-associated virus AAV-CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
| FG002 | High Dose AAV - CNGA3 | Subretinal administration of a single high dose adeno-associated virus AAV-CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Low Dose AAV - CNGA3 | Subretinal administration of a single low dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
| BG001 | Intermediate Dose AAV - CNGA3 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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:
| The safety analysis set included all enrolled participants. | Posted | Count of Participants | Participants | 6 Weeks |
|
6 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Low Dose AAV - CNGA3 | Subretinal administration of a single low dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctival haemorrhage | Eye disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Program Manager | MeiraGTx | 020 3866 4320 | CNGA3@meiragtx.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 16, 2021 | Dec 23, 2021 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 2, 2021 | Dec 23, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D003117 | Color Vision Defects |
| ID | Term |
|---|---|
| D014786 | Vision Disorders |
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
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| Improvements in Retinal Function as Assessed by Static Perimetry |
Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity. |
| 6 Months |
| Quality of Life Measured by QoL Questionnaires in Children and Adolescents | Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening. | 6 Months |
| Quality of Life Measured by QoL Questionnaires in Adults | Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening. | 6 Months |
| London |
| EC1V 2PD |
| United Kingdom |
Subretinal administration of a single intermediate dose AAV - CNGA3
AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene
| BG002 | High Dose AAV - CNGA3 | Subretinal administration of a single high dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
| BG003 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Subretinal administration of a single low dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
| OG001 | Intermediate Dose AAV - CNGA3 | Subretinal administration of a single intermediate dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
| OG002 | High Dose AAV - CNGA3 | Subretinal administration of a single high dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene |
|
|
| Secondary | Improvements in Visual Function as Assessed by Visual Acuity | Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | All of the 11 participants performed the visual acuity assessment at baseline and Week 24. | Posted | Mean | Full Range | number of ETDRS letters | 6 Months |
|
|
|
| Secondary | Improvements in Retinal Function as Assessed by Static Perimetry | Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity. | Six of the 11 participants had mean retinal sensitivity data available at both baseline and Week 24 in the treated eye. | Posted | Mean | Full Range | decibel | 6 Months |
|
|
|
| Secondary | Quality of Life Measured by QoL Questionnaires in Children and Adolescents | Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening. | EQ-VAS data were available for 6 of the 9 children/adolescents enrolled in the study. | Posted | Mean | Full Range | units on a scale | 6 Months |
|
|
|
| Secondary | Quality of Life Measured by QoL Questionnaires in Adults | Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening. | EQ-VAS data were available for both adults enrolled in the study. | Posted | Mean | Full Range | units on a scale | 6 Months |
|
|
|
| 0 |
| 3 |
| 0 |
| 3 |
| 3 |
| 3 |
| EG001 | Intermediate Dose AAV - CNGA3 | Subretinal administration of a single intermediate dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene | 0 | 3 | 0 | 3 | 3 | 3 |
| EG002 | High Dose AAV - CNGA3 | Subretinal administration of a single high dose AAV - CNGA3 AAV- CNGA3: AAV gene therapy for defects in CNGA3 gene | 0 | 5 | 0 | 5 | 5 | 5 |
| Visual acuity reduced | Eye disorders | Systematic Assessment |
|
| Conjunctival hyperaemia | Eye disorders | Systematic Assessment |
|
| Lenticular opacities | Eye disorders | Systematic Assessment |
|
| Ocular discomfort | Eye disorders | Systematic Assessment |
|
| Eye inflammation | Eye disorders | Systematic Assessment |
|
| Eye pain | Eye disorders | Systematic Assessment |
|
| Eye pruritus | Eye disorders | Systematic Assessment |
|
| Subconjunctival cyst | Eye disorders | Systematic Assessment |
|
| Chorioretinal folds | Eye disorders | Systematic Assessment |
|
| Foreign body sensation in eyes | Eye disorders | Systematic Assessment |
|
| Iridocyclitis | Eye disorders | Systematic Assessment |
|
| Photophobia | Eye disorders | Systematic Assessment |
|
| Punctate keratitis | Eye disorders | Systematic Assessment |
|
| Retinal disorder | Eye disorders | Systematic Assessment |
|
| Uveitis | Eye disorders | Systematic Assessment |
|
| Vitreous floaters | Eye disorders | Systematic Assessment |
|
| Intraocular pressure increased | Investigations | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | Systematic Assessment |
|
| Blood glucose increased | Investigations | Systematic Assessment |
|
| Intraocular pressure decreased | Investigations | Systematic Assessment |
|
| Slit-lamp tests abnormal | Investigations | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Hypophosphataemia | Metabolism and nutrition disorders | Systematic Assessment |
|
| Hyperlipidaemia | Endocrine disorders | Systematic Assessment |
|
| Type 2 diabetes mellitus | Endocrine disorders | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | Systematic Assessment |
|
| Seasonal allergy | Immune system disorders | Systematic Assessment |
|
| Rhinitis | Infections and infestations | Systematic Assessment |
|
| Tenon's cyst | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Syncope | Nervous system disorders | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
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| D000077765 |
| Cone Dystrophy |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |