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Long-term Safety and Efficacy Follow-up of AAV2-REP1 for the Treatment of Choroideremia (SOLSTICE) (SOLSTICE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03584165
Recruitment Status : Enrolling by invitation
First Posted : July 12, 2018
Last Update Posted : August 8, 2018
Sponsor:
Information provided by (Responsible Party):
Nightstar Therapeutics

Brief Summary:
This is a long-term follow up evaluating the safety and efficacy of AAV2-REP1 used in antecedent choroideremia studies.

Condition or disease Intervention/treatment
Choroideremia Genetic: AAV2-REP1

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.

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Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Other
Time Perspective: Other
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
Actual Study Start Date : June 4, 2018
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : April 2024

Resource links provided by the National Library of Medicine



Intervention Details:
  • Genetic: AAV2-REP1
    Sub-retinal injection of AAV2-REP1 after vitrectomy.


Primary Outcome Measures :
  1. Adverse events as a measure of safety and tolerability [ Time Frame: up to 5 years ]
    Incidence of treatment emergent adverse events (%)


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)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients previously treated with AAV2-REP1 in an antecedent study.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03584165


Locations
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United States, Florida
Study Site
Miami, Florida, United States, 33136
Germany
Study Site
Tübingen, Germany
Sponsors and Collaborators
Nightstar Therapeutics

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Responsible Party: Nightstar Therapeutics
ClinicalTrials.gov Identifier: NCT03584165     History of Changes
Other Study ID Numbers: NSR-CHM-OS2
First Posted: July 12, 2018    Key Record Dates
Last Update Posted: August 8, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Nightstar Therapeutics:
NightstaRx
NSR-REP1
Choroideremia
CHM
Gene Therapy
AAV
REP1

Additional relevant MeSH terms:
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Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked