Clinical trial NCT03584165: Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
Official title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Choroideremia
X-Linked Retinitis Pigmentosa
Keywords: NightstaRx
Biogen
NSR-REP1
CHM
Gene Therapy
AAV
REP1
AAV8
RPGR
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2018-07-12
Enrollment: 330
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.

Detailed description: This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Eligibility criteria: Key Inclusion Criteria:

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.

Key Exclusion Criteria:

Participants are not eligible for study participation if they meet the following exclusion criterion.

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.

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

Primary outcomes:
measure: Percentage of Participants with Adverse Events (AEs)
timeFrame: Up to 5 years
measure: Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
timeFrame: Up to 5 years
measure: Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination
timeFrame: Up to 5 years
measure: Ophthalmic Examination Assessment: Lens Opacity Grading
timeFrame: Up to 5 years
measure: Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation
timeFrame: Up to 5 years
measure: Ophthalmic Examination Assessment: Indirect Ophthalmoscopy
timeFrame: Up to 5 years
Secondary outcomes:
measure: Change from Baseline in Best-Corrected Visual Acuity (BCVA)
timeFrame: Up to 5 years
measure: 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
timeFrame: Up to 5 years
measure: Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants
timeFrame: Up to 5 years
measure: Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants
timeFrame: Up to 5 years
measure: Assessment of Fundus Autofluorescence (AF) at Each Visit
timeFrame: Up to 5 years
measure: Assessment of Fundus Photography at Each Visit
timeFrame: Up to 5 years
measure: Assessment of Spectral Domain Optical Coherence Tomography (SD-OCT) at Each Visit
timeFrame: Up to 5 years
measure: Assessment of Microperimetry at Each Visit
timeFrame: Up to 5 years
measure: Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25)
timeFrame: Up to 5 years
measure: Change from Baseline in Visual Field
timeFrame: Up to 5 years
measure: Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants
timeFrame: 18 Months to 60 Months, Post-Day 0 Visits
measure: Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112-treated Participants
timeFrame: 18 Months to 60 Months, Post-Day 0 Visits
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA - Jules Stein Eye Institute
state: California
city: Gainesville
country: United States
facility: Vitreo Retinal Associates PA - The Millennium Center
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: MEEI Massachusets Eye and Ear Infirmary
state: Massachusetts
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Eye Institute - Blue Ash
state: Ohio
city: Portland
country: United States
facility: OHSU - Casey Eye Institute
state: Oregon
city: Dallas
country: United States
facility: Retina Foundation of the Southwest
state: Texas
city: Madison
country: United States
facility: University of Wisconsin School of Medicine
state: Wisconsin
city: Sao Paulo
country: Brazil
facility: Instituto Genetica Ocular
city: Edmonton
country: Canada
facility: The Northern Alberta Clinical Trials and Research Centre
state: Alberta
city: Vancouver
country: Canada
facility: The University of British Columbia - Eye Care Centre
state: British Columbia
city: Montreal
country: Canada
facility: McGill University Health Centre
state: Quebec
city: Glostrup
country: Denmark
facility: Rigshospitalet-Glostrup, Oejenafdelingen
city: Helsinki
country: Finland
facility: Helsinki University Central Hospital (HUCH)
city: Montpellier
country: France
facility: CHU Montpellier - Saint Eloi
city: Paris
country: France
facility: Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
city: Bonn
country: Germany
facility: Universitäts-Augenklinik Bonn
city: Tübingen
country: Germany
facility: Universitats Klinikum Tubingen - Institute for Ophthalmic Research
city: Nijmegen
country: Netherlands
facility: Radboudumc
city: London
country: United Kingdom
facility: Moorfields Eye Hospital
city: Manchester
country: United Kingdom
facility: Manchester Royal Eye Hopsital
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: