NCT03496012

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).

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
169

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Dec 2017

Typical duration for phase_3

Geographic Reach
8 countries

18 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 11, 2017

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

March 7, 2018

Completed
1 month until next milestone

First Posted

Study publicly available on registry

April 12, 2018

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2020

Completed
3 years until next milestone

Results Posted

Study results publicly available

December 7, 2023

Completed
Last Updated

December 7, 2023

Status Verified

November 1, 2023

Enrollment Period

3 years

First QC Date

March 7, 2018

Results QC Date

November 15, 2023

Last Update Submit

November 15, 2023

Conditions

Choroideremia

Keywords

NightstaRxNSR-REP1Gene TherapyAAVREP1Timrepigene Emparvovec

Outcome Measures

Primary Outcomes (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

    BCVA was assessed for both eyes using the 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. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of participants with a ≥15 -letter improvement from baseline in BCVA at Month 12 was reported for both eyes.

    Month 12

Secondary Outcomes (18)

  • Change From Baseline in BCVA at Month 12 Reported as Letters Measured by the ETDRS Chart in Study Eye

    Baseline, Month 12

  • Percentage of Participants With a ≥10 -Letter Improvement From Baseline in BCVA at Month 12 Measured by the ETDRS Chart

    Month 12

  • 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

    Month 12

  • Change From Baseline in BCVA at Months 4 and 8 Reported as Letters Measured by the ETDRS Chart

    Baseline, Months 4 and 8

  • Change From Baseline in Total Area of Preserved Autofluorescence (AF) at Month 12 in Study Eye

    Baseline, Month 12

  • +13 more secondary outcomes

Study Arms (3)

BIIB111 High Dose

EXPERIMENTAL

Participants will receive a single administration of high dose BIIB111 in one eye through sub-retinal injection after vitrectomy.

Genetic: BIIB111

BIIB111 Low Dose

EXPERIMENTAL

Participants will receive a single administration of low dose BIIB111 in one eye through sub-retinal injection after vitrectomy.

Genetic: BIIB111

Untreated Control Group

NO INTERVENTION

Participants will receive no sham surgery or study medication.

Interventions

BIIB111GENETIC

Administered as specified in the treatment arm.

Also known as: AAV2-REP1
BIIB111 High DoseBIIB111 Low Dose

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

Research Site

Los Angeles, California, 90095, United States

Location

Research Site

Miami, Florida, 33136, United States

Location

Research Site

Baltimore, Maryland, 21287, United States

Location

Research Site

New York, New York, 10032, United States

Location

Research Site

Cincinnati, Ohio, 45242, United States

Location

Research Site

Portland, Oregon, 97232, United States

Location

Research Site

Dallas, Texas, 75231, United States

Location

Research Site

Madison, Wisconsin, 53705, United States

Location

Research Site

Montreal, H3A 0E7, Canada

Location

Research Site

Vancouver, V5Z 3N9, Canada

Location

Research Site

Glostrup Municipality, Denmark

Location

Research Site

Helsinki, 00290, Finland

Location

Research Site

Montpellier, France

Location

Research Site

Bonn, 53127, Germany

Location

Research Site

Tübingen, Germany

Location

Research Site

Nijmegen, Netherlands

Location

Research Site

Manchester, M13 9WL, United Kingdom

Location

Research Site

Oxford, OX3 9DU, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 30194931DERIVED

MeSH Terms

Conditions

Choroideremia

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesChoroid DiseasesUveal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Results Point of Contact

Title
US Biogen Clinical Trial Center
Organization
Biogen
clinicaltrials@biogen.com
866-633-4636

Study Officials

  • Medical Director

    Biogen

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 7, 2018

First Posted

April 12, 2018

Study Start

December 11, 2017

Primary Completion

December 1, 2020

Study Completion

December 1, 2020

Last Updated

December 7, 2023

Results First Posted

December 7, 2023

Record last verified: 2023-11

Data Sharing

IPD Sharing
Will share

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

More information

Locations

FR(1)DK(1)CA(2)FI(1)GB(2)US(8)NL(1)DE(2)