A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with 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 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 CTR

Registry ID

EUCTR2017-003104-42-GB

Enrollment

440

Registered

2018-02-06

Start date

2018-06-26

Completion date

Unknown

Last updated

2019-12-10

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) MedDRA version: 20.1 Level: LLT Classification code 10008791 Term: Choroideremia System Organ Class: 100000004853 MedDRA version: 20.0 Level: PT Classification code 10038914 Term: Retinitis pigmentosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: AAV2-REP1 Product Code: AAV2-REP1 Pharmaceutical Form: Suspension for injection INN or Proposed INN: timrepigene emparvovec CAS Number: 1905415-02-2 Current Sponsor code: AAV2-REP1 Other

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: CHM Subjects: a. Are willing and able to give informed consent for participation in the study, and b. Have received a sub-retinal injection of AAV2-REP1 for CHM and have exited an antecedent study XLRP Subjects: a. Are willing and able to give informed consent (or legal guardian consent and subject assent, if applicable) for participation in the study b. Have received a sub-retinal injection of AAV8-RPGR for XLRP and have exited an antecedent study Are the trial subjects under 18? yes Number of subjects for this age range: 9 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 167 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: In the opinion of the investigator and/or the Sponsor, it is not in the subject’s best interest to participate in the study.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the long-term safety and efficacy of a sub retinal injection of: • AAV2-REP1 in subjects with choroideremia (CHM) who have been previously treated with AAV2-REP1 and who have exited an antecedent study. • AAV8-RPGR in subjects with X-linked retinitis pigmentosa (XLRP) who have been previously treated with AAV8-RPGR and who have exited an antecedent study.;Secondary Objective: Not applicable;Primary end point(s): Long-term safety of AAV2-REP1 and AAV8-RPGR;Timepoint(s) of evaluation of this end point: Safety assessments will be performed as per protocol	—

Measure

Time frame

Main Objective: To evaluate the long-term safety and efficacy of a sub retinal injection of: • AAV2-REP1 in subjects with choroideremia (CHM) who have been previously treated with AAV2-REP1 and who have exited an antecedent study. • AAV8-RPGR in subjects with X-linked retinitis pigmentosa (XLRP) who have been previously treated with AAV8-RPGR and who have exited an antecedent study.;Secondary Objective: Not applicable;Primary end point(s): Long-term safety of AAV2-REP1 and AAV8-RPGR;Timepoint(s) of evaluation of this end point: Safety assessments will be performed as per protocol

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Secondary

Measure	Time frame
Secondary end point(s): • Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart • Change from Baseline in fundus autofluorescence • Change from Baseline in fundus photography • Change from Baseline in spectral-domain optical coherence tomography (SD-OCT) • Change from Baseline in microperimetry • Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25) • Change from Baseline in visual field (in AAV8-RPGR-treated subjects only);Timepoint(s) of evaluation of this end point: Visual assessments will be performed as per protocol	—

Measure

Time frame

Secondary end point(s): • Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart • Change from Baseline in fundus autofluorescence • Change from Baseline in fundus photography • Change from Baseline in spectral-domain optical coherence tomography (SD-OCT) • Change from Baseline in microperimetry • Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25) • Change from Baseline in visual field (in AAV8-RPGR-treated subjects only);Timepoint(s) of evaluation of this end point: Visual assessments will be performed as per protocol

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Countries

Canada, Denmark, Finland, France, Germany, Netherlands, United Kingdom, United States

Contacts

Public ContactClinical Operations Department

NightstaRx Ltd

enquiries@nightstarx.com+441628501000

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026