Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
- Conditions
- ChoroideremiaX-Linked Retinitis Pigmentosa
- Interventions
- Genetic: BIIB111Genetic: BIIB112
- Registration Number
- NCT03584165
- Lead Sponsor
- NightstaRx Ltd, a Biogen Company
- 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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 330
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
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description BIIB111 BIIB111 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. BIIB112 BIIB112 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.
- Primary Outcome Measures
Name Time Method Ophthalmic Examination Assessment: Lens Opacity Grading Up to 5 years Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation Up to 5 years 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: Indirect Ophthalmoscopy Up to 5 years
- Secondary Outcome Measures
Name Time Method Change from Baseline in Best-Corrected Visual Acuity (BCVA) Up to 5 years 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.
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 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.
Assessment of Microperimetry at Each Visit Up to 5 years Microperimetry will be performed on both eyes to assess changes in retinal sensitivity within the macula. It will be reported in decibels (dB).
Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25) Up to 5 years 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.
Assessment of Fundus Photography at Each Visit Up to 5 years Fundus photography will be performed on both eyes following the dilation of the participant's pupils.
Assessment of Spectral Domain Optical Coherence Tomography (SD-OCT) at Each Visit Up to 5 years SD-OCT will be performed on both eyes to assess the foveal changes and other potential anatomic changes. The measurements were taken after dilation of the participant's pupil and would be reported in micrometres (µm).
Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants 18 Months to 60 Months, Post-Day 0 Visits The LLVA was measured by placing a 2.0-log-unit neutral density filter over the best correction for that eye and having the participant read the normally illuminated ETDRS chart. The assessment was performed prior to dilating the eyes. Initially, letters are read at a distance of 4 metres from the chart. If \<20 letters are read at 4 metres, testing at 1 metre should be performed. BCVA is to be reported as number of letters read correctly by the participant.
Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants Up to 5 years 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.
Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants Up to 5 years 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.
Assessment of Fundus Autofluorescence (AF) at Each Visit Up to 5 years Fundus Autofluoroscence will be performed on both eyes to assess the change in the area of viable retinal tissue. It will be reported in square millimetres (mm\^2).
Change from Baseline in Visual Field Up to 5 years The outcome measure will be assessed in BIIB112-treated participants.
Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112-treated Participants 18 Months to 60 Months, Post-Day 0 Visits The LLVA was measured by placing a 2.0-log-unit neutral density filter over the best correction for that eye and having the participant read the normally illuminated ETDRS chart. The assessment was performed prior to dilating the eyes. Initially, letters are read at a distance of 4 metres from the chart. If \<20 letters are read at 4 metres, testing at 1 metre should be performed. BCVA is to be reported as number of letters read correctly by the participant.
Trial Locations
- Locations (25)
Instituto Genetica Ocular
🇧🇷Sao Paulo, Brazil
Vitreo Retinal Associates PA - The Millennium Center
🇺🇸Gainesville, Florida, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Cincinnati Eye Institute - Blue Ash
🇺🇸Cincinnati, Ohio, United States
Retina Foundation of the Southwest
🇺🇸Dallas, Texas, United States
University of Wisconsin School of Medicine
🇺🇸Madison, Wisconsin, United States
The University of British Columbia - Eye Care Centre
🇨🇦Vancouver, British Columbia, Canada
The Northern Alberta Clinical Trials and Research Centre
🇨🇦Edmonton, Alberta, Canada
McGill University Health Centre
🇨🇦Montreal, Quebec, Canada
CHU Montpellier - Saint Eloi
🇫🇷Montpellier, France
Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
🇫🇷Paris, France
Universitäts-Augenklinik Bonn
🇩🇪Bonn, Germany
Radboudumc
🇳🇱Nijmegen, Netherlands
Universitats Klinikum Tubingen - Institute for Ophthalmic Research
🇩🇪Tübingen, Germany
Moorfields Eye Hospital
🇬🇧London, United Kingdom
Manchester Royal Eye Hopsital
🇬🇧Manchester, United Kingdom
John Radcliffe Hospital
🇬🇧Oxford, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom
Helsinki University Central Hospital (HUCH)
🇫🇮Helsinki, Finland
Rigshospitalet-Glostrup, Oejenafdelingen
🇩🇰Glostrup, Denmark
UCLA - Jules Stein Eye Institute
🇺🇸Los Angeles, California, United States
Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
MEEI Massachusets Eye and Ear Infirmary
🇺🇸Boston, Massachusetts, United States
OHSU - Casey Eye Institute
🇺🇸Portland, Oregon, United States
University of Miami
🇺🇸Miami, Florida, United States