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Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia (STAR)

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ClinicalTrials.gov Identifier: NCT03496012
Recruitment Status : Recruiting
First Posted : April 12, 2018
Last Update Posted : April 12, 2018
Sponsor:
Information provided by (Responsible Party):
Nightstar Therapeutics

Brief Summary:
The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with Choroideremia.

Condition or disease Intervention/treatment Phase
Choroideremia Genetic: AAV2-REP1 Phase 3

Detailed Description:
This is a phase 3 clinical trial of a gene therapy vector made from adeno-associated virus (AAV) called AAV2-REP1 for the treatment of Choroideremia. Participating subjects will be required to attend a screening visit during which their suitability for the study will be assessed, and eligible subjects will be randomized to either AAV2-REP1 treatment or the control group.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group Study.
Masking: Single (Outcomes Assessor)
Masking Description:

Outcomes-assessors will be masked to whether the subjects is in the treated or the control group.

Subjects and trial centres will be masked to whether high or low dose has been administered in the treated group.

Primary Purpose: Treatment
Official Title: A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)
Actual Study Start Date : December 11, 2017
Estimated Primary Completion Date : March 31, 2020
Estimated Study Completion Date : March 31, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: AAV2-REP1 High Dose
Subjects will receive a single administration of high-dose AAV2-REP1 in one eye.
Genetic: AAV2-REP1
Sub-retinal injection of AAV2-REP1 after vitrectomy.

Experimental: AAV2-REP1 Low Dose
Subjects will receive a single administration of low-dose AAV2-REP1 in one eye.
Genetic: AAV2-REP1
Sub-retinal injection of AAV2-REP1 after vitrectomy.

No Intervention: Untreated control group
Untreated control subjects will be observed for 12 months.



Primary Outcome Measures :
  1. Best Corrected Visual Acuity [ Time Frame: 12 Months ]
    ETDRS visual acuity chart


Secondary Outcome Measures :
  1. Fundus autofluorescence (AF) [ Time Frame: 12 Months ]
    Change in AF (mm2)

  2. Optical coherence tomography [ Time Frame: 12 Months ]
    Ellipsoid Zone

  3. Microperimetry [ Time Frame: 12 Months ]
    Change in Sensitivity (dB)

  4. Contrast Sensitivity [ Time Frame: 12 Months ]
    Pelli-Robson Chart

  5. Color Vision [ Time Frame: 12 Months ]
    Farnsworth-Munsell 100 Hue Test



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Are willing and able to give informed consent for participation in the study.
  • Are male and ≥18 years of age.
  • Have a genetically-confirmed diagnosis of CHM.
  • Have active disease clinically visible within the macular region in the study eye.
  • Fulfill pre-defined visual acuity criteria.

Exclusion Criteria:

  • Have a history of amblyopia in the eligible eye.
  • Are unwilling to use barrier contraception methods, for a period of 3 months, if treated with AAV2-REP1.
  • Previous intraocular surgery performed in the study eye within 3 months of Visit 1.
  • Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the subjects at risk because of participation in the study, or may influence the results of the study, or the subject's ability to participate in the study.
  • Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously.

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): NCT03496012


Contacts
Contact: Nightstar Therapeutics +44 (0)20 7611 2077 researchenquiries@nightstartx.com

Locations
United States, California
Jules Stein Eye Institute, University of California Recruiting
Los Angeles, California, United States, 90095
United States, Florida
Bascom Palmer Eye Institute Recruiting
Miami, Florida, United States, 33136
United States, Maryland
The Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21287
United States, New York
Columbia University Medical Center, Edward S Harkness Eye Inst Recruiting
New York, New York, United States, 10032
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97232
United States, Texas
Retina Foundation of the Southwest, Recruiting
Dallas, Texas, United States, 75231
United States, Wisconsin
University of Wisconsin Recruiting
Madison, Wisconsin, United States, 53705
Canada
The Research Institute of the McGill University Health Centre Recruiting
Québec, Canada, H3A 0E7
University of British Columbia, Eye Care Centre Recruiting
Vancouver, Canada, V5Z 3N9
Finland
Helsinki University Central Hospital Recruiting
Helsinki, Finland, 00290
Germany
University Eye Clinic Bonn Recruiting
Bonn, Germany, 53127
University of Tübingen Recruiting
Tübingen, Germany
Netherlands
Radboud University Medical Center Recruiting
Nijmegen, Netherlands
United Kingdom
Manchester Centre for Genomic Medicine, St Mary's Hospital Recruiting
Manchester, United Kingdom, M13 9WL
Oxford Eye Hospital, John Radcliffe Hospital Recruiting
Oxford, United Kingdom, OX3 9DU
Sponsors and Collaborators
Nightstar Therapeutics

Responsible Party: Nightstar Therapeutics
ClinicalTrials.gov Identifier: NCT03496012     History of Changes
Other Study ID Numbers: NSR-REP-01
2015-003958-41 ( EudraCT Number )
First Posted: April 12, 2018    Key Record Dates
Last Update Posted: April 12, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked