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A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study (SOLSTICE)

Status
Not yet recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-507994-16-00
Acronym
NSR-CHM-OS2 273CH201
Enrollment
54
Registered
2024-10-01
Start date
Unknown
Completion date
Unknown
Last updated
2025-12-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Choroideremia (CHM) X-Linked Retinitis Pigmentosa (XLRP)

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

Biogen Idec Research Limited
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
Male
Age
18 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
Safety assessments will be performed as per protocol

Secondary

MeasureTime 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

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026