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Efficacy and Safety of BIIB111 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 : Completed
First Posted : April 12, 2018
Last Update Posted : February 13, 2023
Sponsor:
Information provided by (Responsible Party):
Biogen ( NightstaRx Ltd, a Biogen Company )

Study Description
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Brief Summary:
The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

Condition or disease Intervention/treatment Phase
Choroideremia Genetic: BIIB111 Phase 3

Detailed Description:
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 170 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
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
Actual Primary Completion Date : December 1, 2020
Actual Study Completion Date : December 1, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: BIIB111 High Dose
Participants will receive a single administration of high dose BIIB111 in one eye through sub-retinal injection after vitrectomy.
 Genetic: BIIB111
Administered as specified in the treatment arm.
Other Name: AAV2-REP1
Experimental: BIIB111 Low Dose
Participants will receive a single administration of low dose BIIB111 in one eye through sub-retinal injection after vitrectomy.
 Genetic: BIIB111
Administered as specified in the treatment arm.
Other Name: AAV2-REP1
No Intervention: Untreated Control Group
Participants will receive no sham surgery or study medication.


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of Participants with a ≥15-Letter Improvement from Baseline in Best Corrected Visual Acuity (BCVA) at Month 12 as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart [ Time Frame: Month 12 ]
    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.


Secondary Outcome Measures :
  1. Change from Baseline in BCVA at Month 12 Measured by the ETDRS Chart [ Time Frame: Month 12 ]
    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.

  2. Percentage of Participants with a ≥10-Letter Improvement from Baseline in BCVA at Month 12 Measured by the ETDRS Chart [ Time Frame: Month 12 ]
    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.

  3. Percentage of Participants with No Decrease from Baseline in BCVA or a Decrease from Baseline in BCVA of <5 ETDRS Letters at Month 12 Measured by the EDRS Chart [ Time Frame: Month 12 ]
    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.

  4. Change from Baseline in BCVA at Months 4 and 8 [ Time Frame: Months 4 and 8 ]
    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.

  5. Change from Baseline in Total Area of Preserved Autofluorescence (AF) at Month 12 [ Time Frame: Month 12 ]
  6. Change from Baseline in the Area of Preserved Ellipsoid Zone Using Spectral Domain Optical Coherence Tomography (SD-OCT) at Month 12 [ Time Frame: Months 12 ]
  7. Change from Baseline in Microperimetry at Month 12 [ Time Frame: Month 12 ]
  8. Change from Baseline in Contrast Sensitivity Score at Month 12 [ Time Frame: Month 12 ]
    Change from baseline in contrast sensitivity in the study eye is measured using a Pelli Robson contrast sensitivity chart at 1 meter. The contrast sensitivity chart contains letters that are darkest at the top and then get progressively lighter. Scores range from 0 to 48 and are based on the number of letters read correctly. A negative change from baseline indicates a worsening in contrast sensitivity and a positive change from baseline indicates an improvement.

  9. Change from Baseline in Colour Vision at Month 12 [ Time Frame: Month 12 ]
  10. Change from Baseline in Reading Speed Test at Month 12 [ Time Frame: Month 12 ]
  11. Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25) at Month 12 [ Time Frame: Month 12 ]
    VFQ-25 questionnaire measures dimensions of self-reported vision-targeted health status that are most important to persons with eye disease. Total score ranges from 0-100, where a score of 0 represents the worst outcome and 100 represents the best outcome.


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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

Key Inclusion Criteria:

  • Are willing and able to give informed consent for participation in the study.
  • Have a documented genetically-confirmed diagnosis of CHM.
  • Have active disease clinically visible within the macular region in the study eye.
  • Have a BCVA of 34-73 ETDRS letters (equivalent to worse than or equal to 6/12 or 20/40 Snellen acuity, but better than or equal to 6/60 or 20/200 Snellen acuity) in the study eye.

Key Exclusion Criteria:

  • Have a history of amblyopia in the eligible eye.
  • 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 participants at risk because of participation in the study, or may influence the results of the study, or the participant'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.
  • Are unwilling to use barrier contraception methods, or abstain from sexual intercourse, for a period of 3 months, if treated with AAV2-REP1.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Contacts and Locations
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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


Locations
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United States, California
Research Site
Los Angeles, California, United States, 90095
United States, Florida
Research Site
Miami, Florida, United States, 33136
United States, Maryland
Research Site
Baltimore, Maryland, United States, 21287
United States, New York
Research Site
New York, New York, United States, 10032
United States, Ohio
Research Site
Cincinnati, Ohio, United States, 45242
United States, Oregon
Research Site
Portland, Oregon, United States, 97232
United States, Texas
Research Site
Dallas, Texas, United States, 75231
United States, Wisconsin
Research Site
Madison, Wisconsin, United States, 53705
Canada
Research Site
Montréal, Canada, H3A 0E7
Research Site
Vancouver, Canada, V5Z 3N9
Denmark
Research Site
Glostrup, Denmark
Finland
Research Site
Helsinki, Finland, 00290
France
Research Site
Montpellier, France
Germany
Research Site
Bonn, Germany, 53127
Research Site
Tübingen, Germany
Netherlands
Research Site
Nijmegen, Netherlands
United Kingdom
Research Site
Manchester, United Kingdom, M13 9WL
Research Site
Oxford, United Kingdom, OX3 9DU
Sponsors and Collaborators
NightstaRx Ltd, a Biogen Company
Investigators
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Study Director: Medical Director Biogen
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: NightstaRx Ltd, a Biogen Company
ClinicalTrials.gov Identifier: NCT03496012    
Other Study ID Numbers: 273CH301
2015-003958-41 ( EudraCT Number )
NSR-REP-01 ( Other Identifier: NightstaRx Ltd, a Biogen Company )
First Posted: April 12, 2018    Key Record Dates
Last Update Posted: February 13, 2023
Last Verified: February 2023
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 Biogen ( NightstaRx Ltd, a Biogen Company ):
NightstaRx
NSR-REP1
Gene Therapy
 AAV
REP1
Timrepigene Emparvovec
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