X-Linked Retinitis Pigmentosa
Conditions
Brief summary
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Interventions
BIOLOGICALGenetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Sponsors
Janssen Research & Development, LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Masking description
No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)
Intervention model description
Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female * 3 years of age or older * Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory
Exclusion criteria
* Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration * Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule * Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze | From Baseline to 52 Weeks | Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52 | From Baseline to Week 52 | Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed. |
| Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52 | From Baseline to Week 52 | Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed. |
| Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52 | From Baseline to Week 52 | Pointwise response in full visual field at Week 52 will be assessed. |
| Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52 | From Baseline to Week 52 | Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed. |
| Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52 | From Baseline to Week 52 | Pointwise response in the central 30 degrees visual field at Week 52 will be assessed. |
| Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52 | From Baseline to Week 52 | Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed. |
| Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52 | From Baseline to Week 52 | Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed. |
| Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52 | From Baseline to Week 52 | Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52. |
| Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52 | From Baseline to Week 52 | Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52. |
| Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52 | From Baseline to Week 52 | Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52. |
| Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52 | From Baseline to Week 52 | Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52. |
| Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52 | From Baseline to Week 52 | Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52. |
| Number of Participants with Ocular and Non-ocular Adverse Events | Day 1 - Week 52 | Number of participants with ocular and non-ocular adverse events will be assessed. |
| Number of Participants With Abnormalities in Laboratory Assessments | Day 1 - 52 Weeks | Number of participants with abnormalities in laboratory assessments will be assessed. |
Countries
Belgium, Canada, Denmark, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States
Contacts
STUDY_DIRECTORJanssen Research & Development, LLC Clinical Trial
Janssen Research & Development, LLC
Outcome results
None listed