Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa

NCT03584165 | Enrolling By Invitation Phase 3 FDA Drug

• 2 other identifiers: 273CH2012017-003104-42
• Study Type: interventional
• Target: 330
• Locations: 9 countries
• Sites: 25

Brief Summary

The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.

Conditions

Choroideremia; X-Linked Retinitis Pigmentosa

Keywords

NightstaRx; Biogen; NSR-REP1; CHM; Gene Therapy; AAV; REP1; AAV8; RPGR

Outcome Measures

Primary Outcomes (6)

• Percentage of Participants with Adverse Events (AEs)

  Up to 5 years

• Ophthalmic Examination Assessment: Intraocular Pressure (IOP)

  Up to 5 years

• Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination

  Up to 5 years

• Ophthalmic Examination Assessment: Lens Opacity Grading

  Up to 5 years

• Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation

  Up to 5 years

• Ophthalmic Examination Assessment: Indirect Ophthalmoscopy

  Up to 5 years

Secondary Outcomes (12)

• Change from Baseline in Best-Corrected Visual Acuity (BCVA)

  Up to 5 years

• Percentage of Participants with no Decrease from Baseline in BCVA or a Decrease from Baseline in BCVA of <5 ETDRS Letters in Choroideremia (CHM) Participants

  Up to 5 years

• Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants

  Up to 5 years

• Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants

  Up to 5 years

• Assessment of Fundus Autofluorescence (AF) at Each Visit

  Up to 5 years

Study Arms (3)

BIIB111

EXPERIMENTAL

Participants previously treated with sub-retinal injection of BIIB111 in antecedent studies 273CH301 (NCT03496012) and 273CH203 (NCT03507686) will be enrolled. Participants previously treated with this same sub-retinal injection (rAAV2-REP1) in antecedent studies 20150371 (NCT02553135), Pro00028599 (NCT02077361), THOR-TUE-01 (NCT02671539), CHM09/01 (NCT01461213) and REGEN2015 (NCT02407678) will also be invited for enrollment. This is a follow-up study, investigational product was administered in the previous study.

Genetic: BIIB111

BIIB112

EXPERIMENTAL

Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled. This is a follow-up study, investigational product was administered in the previous study.

Genetic: BIIB112

Untreated

NO INTERVENTION

Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled. This is a follow-up study, participants will not be administered study medication nor receive a sham surgery.

Interventions

BIIB111 GENETIC

Administered as specified in the treatment arm.

Also known as: AAV2-REP1, rAAV2-REP1

BIIB111

BIIB112 GENETIC

Administered as specified in the treatment arm.

Also known as: AAV8-RPGR

BIIB112

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• CHM Participants
• a. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of BIIB111 for CHM.
• XLRP Participants
• a. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.

You may not qualify if:

• a. In the opinion of the Investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.

Sponsors & Collaborators

• NightstaRx Ltd, a Biogen Company: lead

Study Sites (25)

UCLA - Jules Stein Eye Institute

Los Angeles, California, 90095-7065, United States

Location

Vitreo Retinal Associates PA - The Millennium Center

Gainesville, Florida, 32607, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287-0005, United States

Location

MEEI Massachusets Eye and Ear Infirmary

Boston, Massachusetts, 02114-3002, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Cincinnati Eye Institute - Blue Ash

Cincinnati, Ohio, 45242-5664, United States

Location

OHSU - Casey Eye Institute

Portland, Oregon, 97239, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75231-5080, United States

Location

University of Wisconsin School of Medicine

Madison, Wisconsin, 53705-3644, United States

Location

Instituto Genetica Ocular

São Paulo, 04039, Brazil

Location

The Northern Alberta Clinical Trials and Research Centre

Edmonton, Alberta, T5H 3V9, Canada

Location

The University of British Columbia - Eye Care Centre

Vancouver, British Columbia, V5Z 39N, Canada

Location

McGill University Health Centre

Montreal, Quebec, H4A 3JI, Canada

Location

Rigshospitalet-Glostrup, Oejenafdelingen

Glostrup Municipality, 2600, Denmark

Location

Helsinki University Central Hospital (HUCH)

Helsinki, 00290 HUS, Finland

Location

CHU Montpellier - Saint Eloi

Montpellier, 34295, France

Location

Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts

Paris, 75012, France

Location

Universitäts-Augenklinik Bonn

Bonn, 53127, Germany

Location

Universitats Klinikum Tubingen - Institute for Ophthalmic Research

Tübingen, 72076, Germany

Location

Radboudumc

Nijmegen, 6525 GA, Netherlands

Location

Moorfields Eye Hospital

London, EC1V 2PD, United Kingdom

Location

Manchester Royal Eye Hopsital

Manchester, M13 9WL, United Kingdom

Location

John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom

Location

Southampton General Hospital

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Choroideremia

Interventions

Cotoretigene Toliparvovec

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Choroid Diseases; Uveal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2018
• First Posted: July 12, 2018
• Study Start: June 4, 2018
• Primary Completion (Estimated): June 4, 2026
• Study Completion (Estimated): June 4, 2026
• Last Updated: June 12, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

GB (4); FR (2); DK (1); CA (3); FI (1); BR (1); NL (1); US (10); DE (2)