Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2016-002290-35 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Janssen Research & Development, LLC | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
CNGB3 retinal gene therapy for patients with achromatopsia
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Biological-Low dose AAV - CNGB3 | Experimental | Subretinal administration of a single low dose of AAV - CNGB3 |
|
| Biological-Medium dose AAV - CNGB3 | Experimental | Subretinal administration of a single intermediate dose of AAV - CNGB3 |
|
| Biological-High dose AAV - CNGB3 | Experimental | Subretinal administration of a single high dose of AAV - CNGB3 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV - CNGB3 | Biological | Comparison of different dosages of AAV-CNGB3 |
|
| 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 in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | 6 months |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| James Bainbridge, Prof | Chief Investigator | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Michigan Kellog Eye Centre | Ann Arbor | Michigan | MI 48105 | United States | ||
| Moorfields Eye Hospital NHS Foundation Trust |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37172884 | Derived | Michaelides M, Hirji N, Wong SC, Besirli CG, Zaman S, Kumaran N, Georgiadis A, Smith AJ, Ripamonti C, Gottlob I, Robson AG, Thiadens A, Henderson RH, Fleck P, Anglade E, Dong X, Capuano G, Lu W, Berry P, Kane T, Naylor S, Georgiou M, Kalitzeos A, Ali RR, Forbes A, Bainbridge J. First-in-Human Gene Therapy Trial of AAV8-hCARp.hCNGB3 in Adults and Children With CNGB3-associated Achromatopsia. Am J Ophthalmol. 2023 Sep;253:243-251. doi: 10.1016/j.ajo.2023.05.009. Epub 2023 May 11. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Participants were recruited from medical centers in the United Kingdom (UK) and the United States (US). A total of 23 participants were enrolled in the study.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Low Dose AAV - CNGB3 | Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| FG001 | Intermediate Dose AAV-CNGB3 | Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| FG002 | Other Dose - AAV - CNGB3 | Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| FG003 | High Dose AAV - CNGB3 | Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Low Dose AAV - CNGB3 | Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| BG001 | Intermediate Dose AAV-CNGB3 |
| 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:
| Posted | Count of Participants | Participants | 6 weeks |
|
6 months
Not provided
Not provided
| 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 - CNGB3 | Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Uveitis | Eye disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctival haemorrhage | Eye disorders | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Program Manager | MeiraGTx | 0044 (0)20 3866 4320 | CNGB3@meiragtx.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 29, 2019 | Jan 3, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 18, 2019 | Jan 3, 2023 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D003117 | Color Vision Defects |
| ID | Term |
|---|---|
| D014786 | Vision Disorders |
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 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-5D-Y 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 change from baseline reflects worsening. | 6 months |
| Quality of Life Measured by QoL Questionnaires in Adults | Change from baseline to Week 24 in EuroQol-5D-5L 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 change from baseline reflects worsening. | 6 Months |
| London |
| United Kingdom |
Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene
| BG002 | Other Dose - AAV - CNGB3 | Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| BG003 | High Dose AAV - CNGB3 | Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| BG004 | 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 of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene
| OG001 | Intermediate Dose AAV-CNGB3 | Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| OG002 | Other Dose - AAV - CNGB3 | Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene |
| OG003 | High Dose AAV - CNGB3 | Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 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 in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | 22 of the 23 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. | 20 of the 23 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-5D-Y 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 change from baseline reflects worsening. | EQ-VAS data were available for 11 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-5D-5L 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 change from baseline reflects worsening. | EQ-VAS data were available for 11 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-CNGB3 | Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene | 0 | 12 | 1 | 12 | 12 | 12 |
| EG002 | Other Dose - AAV - CNGB3 | Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene | 0 | 3 | 0 | 3 | 3 | 3 |
| EG003 | High Dose AAV - CNGB3 | Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene | 0 | 5 | 1 | 5 | 5 | 5 |
| EG004 | Total | Safety Analysis Set | 0 | 23 | 2 | 23 | 23 | 23 |
| Ectopic pregnancy | Pregnancy, puerperium and perinatal conditions | Systematic Assessment |
|
| Lenticular opacities | Eye disorders | Systematic Assessment |
|
| Visual acuity reduced | Eye disorders | Systematic Assessment |
|
| Subretinal fluid | Eye disorders | Systematic Assessment |
|
| Foreign body sensation in eyes | Eye disorders | Systematic Assessment |
|
| Ocular discomfort | Eye disorders | Systematic Assessment |
|
| Chorioretinal folds | Eye disorders | Systematic Assessment |
|
| Retinal haemorrhage | Eye disorders | Systematic Assessment |
|
| Conjunctival hyperaemia | Eye disorders | Systematic Assessment |
|
| Eye inflammation | Eye disorders | Systematic Assessment |
|
| Maculopathy | Eye disorders | Systematic Assessment |
|
| Retinal depigmentation | Eye disorders | Systematic Assessment |
|
| Retinal oedema | Eye disorders | Systematic Assessment |
|
| Retinal tear | Eye disorders | Systematic Assessment |
|
| Eye pain | Eye disorders | Systematic Assessment |
|
| Photophobia | Eye disorders | Systematic Assessment |
|
| Vitreous floaters | Eye disorders | Systematic Assessment |
|
| Conjunctival oedema | Eye disorders | Systematic Assessment |
|
| Corneal disorder | Eye disorders | Systematic Assessment |
|
| Macular fibrosis | Eye disorders | Systematic Assessment |
|
| Retinal disorder | Eye disorders | Systematic Assessment |
|
| Retinal pigment epitheliopathy | Eye disorders | Systematic Assessment |
|
| Uveitis | Eye disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Visual field defect | Nervous system disorders | Systematic Assessment |
|
| Dizziness | Nervous system disorders | Systematic Assessment |
|
| Rhinitis | Infections and infestations | Systematic Assessment |
|
| Postoperative wound infection | Infections and infestations | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Intraocular pressure increased | Investigations | Systematic Assessment |
|
| Fatigue | General disorders | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
Not provided
Not provided
| D000077765 |
| Cone Dystrophy |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |