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History of Changes for Study: NCT03584165
Long-term Safety and Efficacy Follow-up of AAV2-REP1 for the Treatment of Choroideremia (SOLSTICE) (SOLSTICE)
Latest version (submitted May 5, 2023) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 June 29, 2018 None (earliest Version on record)
2 July 24, 2018 Outcome Measures and Study Status
3 August 7, 2018 Study Status
4 February 4, 2021 Outcome Measures, Study Identification, Arms and Interventions, Eligibility, Study Status, Contacts/Locations, Oversight, Study Design, Conditions and Study Description
5 October 26, 2021 Study Design, Arms and Interventions, Eligibility, Study Status and Oversight
6 January 17, 2022 Study Status and Contacts/Locations
7 April 20, 2022 Study Status and Contacts/Locations
8 February 9, 2023 Study Status, Study Identification, Contacts/Locations and Outcome Measures
9 May 5, 2023 Study Status, Contacts/Locations, Eligibility, Outcome Measures, Study Design and Study Identification
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Study NCT03584165
Submitted Date:  June 29, 2018 (v1)
Open or close this module Study Identification
Unique Protocol ID: NSR-CHM-OS2
Brief Title: Long-term Safety and Efficacy Follow-up of AAV2-REP1 for the Treatment of Choroideremia (SOLSTICE) (SOLSTICE)
Official Title: 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) in an Antecedent Study
Secondary IDs:
Open or close this module Study Status
Record Verification: June 2018
Overall Status: Enrolling by invitation
Study Start: June 4, 2018
Primary Completion: April 2024 [Anticipated]
Study Completion: April 2024 [Anticipated]
First Submitted: June 29, 2018
First Submitted that
Met QC Criteria:		 June 29, 2018
First Posted: July 12, 2018 [Actual]
Last Update Submitted that
Met QC Criteria:		 June 29, 2018
Last Update Posted: July 12, 2018 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: NightstaRx Ltd, a Biogen Company
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: No
Open or close this module Study Description
Brief Summary: This is a long-term follow up evaluating the safety and efficacy of AAV2-REP1 used in antecedent choroideremia studies.
Detailed Description: This is a prospective and part retrospective, multi-centre, observational, follow-up study for subjects who have previously received a sub-retinal injection of AAV2-REP1 for the treatment of choroideremia in an antecedent study. No further gene therapy will be given in this protocol. Subjects will rollover into the current study when they complete the antecedent study. The study will consist of up to 9 visits over a maximum 48-month study period.
Open or close this module Conditions
Conditions: Choroideremia
Keywords: NightstaRx
NSR-REP1
Choroideremia
CHM
Gene Therapy
AAV
REP1
Open or close this module Study Design
Study Type: Observational
Observational Study Model: Other
Time Perspective: Other
Biospecimen Retention:
Biospecimen Description:
Enrollment: 100 [Anticipated]
Number of Groups/Cohorts 0
Open or close this module Groups and Interventions
Intervention Details:
Genetic: AAV2-REP1
Sub-retinal injection of AAV2-REP1 after vitrectomy.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Safety Outcome Measures
[ Time Frame: up to 5 years ]

Incidence of Adverse Events (AEs) and serious adverse events (SAEs) throughout the study period and at each study visit.
Secondary Outcome Measures:
1. Best Corrected Visual Acuity
[ Time Frame: up to 5 years ]

EDTRS Visual Acuity Chart
2. Fundus Autofluorescence (AF)
[ Time Frame: up to 5 years ]

Change in AF (mm2)
3. Optical Coherence Tomography
[ Time Frame: up to 5 years ]

Ellipsoid Zone
4. Microperimetry
[ Time Frame: up to 5 years ]

Change in Sensitivity (dB)
Open or close this module Eligibility
Study Population: Patients previously treated with AAV2-REP1 in an antecedent study.
Sampling Method: Non-Probability Sample
Minimum Age: 18 Years
Maximum Age:
Sex: Male
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • Are willing and able to give informed consent for participation in the study.
  • Have received a sub-retinal injection of AAV2-REP1 for CHM in an antecedent study.

Exclusion Criteria:

  • There are no exclusion criteria for this study.
Open or close this module Contacts/Locations
Locations: United States, Florida
Study Site
Miami, Florida, United States, 33136
Germany
Study Site
Tübingen, Germany
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:
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U.S. National Library of Medicine | U.S. National Institutes of Health | U.S. Department of Health & Human Services