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Trial record 3 of 3 for:    NSR-REP1

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

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: NCT03496012
Recruitment Status : Active, not recruiting
First Posted : April 12, 2018
Last Update Posted : January 13, 2020
Sponsor:
Collaborator:
Biogen
Information provided by (Responsible Party):
NightstaRx Ltd, a Biogen Company

Tracking Information
First Submitted Date  ICMJE March 7, 2018
First Posted Date  ICMJE April 12, 2018
Last Update Posted Date January 13, 2020
Actual Study Start Date  ICMJE December 11, 2017
Estimated Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 9, 2020)
Best Corrected Visual Acuity (BCVA) [ Time Frame: 12 Months ]
Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart
Original Primary Outcome Measures  ICMJE
 (submitted: April 11, 2018)
Best Corrected Visual Acuity [ Time Frame: 12 Months ]
ETDRS visual acuity chart
Change History Complete list of historical versions of study NCT03496012 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 9, 2020)
  • Fundus autofluorescence (AF) [ Time Frame: 12 Months ]
    Change in AF (mm2)
  • Optical coherence tomography (OCT) [ Time Frame: 12 Months ]
    Ellipsoid Zone
  • Microperimetry [ Time Frame: 12 Months ]
    Change in Sensitivity (dB)
  • Contrast Sensitivity [ Time Frame: 12 Months ]
    Pelli-Robson Chart
  • Color Vision [ Time Frame: 12 Months ]
    Farnsworth-Munsell 100 Hue Test
  • Reading Performance [ Time Frame: 12 Months ]
    International Reading Speed Texts
  • Self Reported Vision-Targeted Health Status [ Time Frame: 12 Months ]
    25-item Visual Function Questionnaire (VFQ-25)
Original Secondary Outcome Measures  ICMJE
 (submitted: April 11, 2018)
  • Fundus autofluorescence (AF) [ Time Frame: 12 Months ]
    Change in AF (mm2)
  • Optical coherence tomography [ Time Frame: 12 Months ]
    Ellipsoid Zone
  • Microperimetry [ Time Frame: 12 Months ]
    Change in Sensitivity (dB)
  • Contrast Sensitivity [ Time Frame: 12 Months ]
    Pelli-Robson Chart
  • Color Vision [ Time Frame: 12 Months ]
    Farnsworth-Munsell 100 Hue Test
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia
Official Title  ICMJE 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)
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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 subject is in the treated or the control group.

Sponsor, subjects and trial centers are masked to dose.

Primary Purpose: Treatment
Condition  ICMJE Choroideremia
Intervention  ICMJE Genetic: AAV2-REP1
Sub-retinal injection of AAV2-REP1 after vitrectomy.
Study Arms  ICMJE
  • Experimental: AAV2-REP1 High Dose
    Subjects will receive a single administration of high-dose AAV2-REP1 in one eye.
    Intervention: Genetic: AAV2-REP1
  • Experimental: AAV2-REP1 Low Dose
    Subjects will receive a single administration of low-dose AAV2-REP1 in one eye.
    Intervention: Genetic: AAV2-REP1
  • No Intervention: Untreated control group
    Observation.
Publications * Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5. Review.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 9, 2020)
169
Original Estimated Enrollment  ICMJE
 (submitted: April 11, 2018)
140
Estimated Study Completion Date  ICMJE November 30, 2020
Estimated Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Are willing and able to give informed consent for participation in the study.
  • Are male and ≥18 years of age.
  • Have a documented 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, or abstain from sexual intercourse, for a period of 3 months, if treated with AAV2-REP1.
  • Have had 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Gender Eligibility Description: Male subjects only.
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Denmark,   Finland,   France,   Germany,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03496012
Other Study ID Numbers  ICMJE NSR-REP-01
2015-003958-41 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party NightstaRx Ltd, a Biogen Company
Study Sponsor  ICMJE NightstaRx Ltd, a Biogen Company
Collaborators  ICMJE Biogen
Investigators  ICMJE Not Provided
PRS Account NightstaRx Ltd, a Biogen Company
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP