A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Treated Previously 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

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Treated Previously 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 Study

Status

Active, not recruiting

Phases

Phase 3

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON54647

Enrollment

Registered

2021-04-09

Start date

2022-01-10

Completion date

Unknown

Last updated

2025-08-18

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

Conditions

choroideremia (CHM) retinal degeneration

Interventions

None listed

Eligibility

Age

18 Years to 64 Years

Inclusion criteria

Inclusion criteria: Inclusion Criteria - Participants are eligible for study participation if they meet all of the following inclusion criteria: CHM Participants: a. Are willing and able to give informed consent for participation in the study b. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of AAV2-REP1 for CHM XLRP Participants: a. Are willing and able to give informed consent for participation in the study b. Have received a sub-retinal injection of AAV8-RPGR for XLRP and have exited an antecedent study

Exclusion criteria

Exclusion criteria: Participants are not eligible for study participation if they meet the following exclusion criterion. a. In the opinion of the investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.

Design outcomes

Primary

Measure	Time frame
The primary endpoint of this study is safety of AAV2-REP1 and AAV8-RPGR, which will be evaluated through adverse event (AE) reporting and full ophthalmic examinations.	—

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 = 10 ETDRS letters (in CHM participants only) • Proportion of participants with an increase from Baseline in BCVA of >= 15 ETDRS letters (in CHM participants 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-RPGR-treated 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.	—

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 = 10 ETDRS letters (in CHM participants only) • Proportion of participants with an increase from Baseline in BCVA of >= 15 ETDRS letters (in CHM participants 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-RPGR-treated 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.

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Countries

Netherlands

Outcome results