Choroideremia (CHM) X-Linked Retinitis Pigmentosa (XLRP)
Conditions
Brief summary
Safety assessments will be performed as per protocol
Detailed description
Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart, Proportion of participants with no decrease from Baseline in BCVA or a decrease from Baseline in BCVA of <5 ETDRS letters (in CHM subjects only), Proportion of participants with an increase from Baseline in BCVA of ≥ 10 ETDRS letters (in CHM subjects only), Proportion of participants with an increase from Baseline in BCVA of ≥ 15 ETDRS letters (in CHM subjects only), Available assessments of fundus autofluorescence at each visit, Available assessments of fundus photography at each visit, Available assessments of spectral-domain optical coherence tomography (SD-OCT) at each visit, Available assessments of microperimetry at each visit, Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25), Change from Baseline in visual field (in AAV8-RPGR-treated participants only), Proportion of participants with an increase from Baseline in low luminance visual acuity (LLVA) of ≥10 ETDRS letters (in AAV8-RPGRtreated participants only), Proportion of participants with an increase from Baseline in LLVA of ≥ 15 ETDRS letters (in AAV8-RPGR-treated participants only), Datasets from the 2 disease populations will be analyzed separately.
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety assessments will be performed as per protocol | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart, Proportion of participants with no decrease from Baseline in BCVA or a decrease from Baseline in BCVA of <5 ETDRS letters (in CHM subjects only), Proportion of participants with an increase from Baseline in BCVA of ≥ 10 ETDRS letters (in CHM subjects only), Proportion of participants with an increase from Baseline in BCVA of ≥ 15 ETDRS letters (in CHM subjects only), Available assessments of fundus autofluorescence at each visit, Available assessments of fundus photography at each visit, Available assessments of spectral-domain optical coherence tomography (SD-OCT) at each visit, Available assessments of microperimetry at each visit, Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25), Change from Baseline in visual field (in AAV8-RPGR-treated participants only), Proportion of participants with an increase from Baseline in lo | — |
Countries
France, Germany