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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-003104-42 | EudraCT Number |
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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.
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
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| 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. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BIIB111 | Genetic | Administered as specified in the treatment arm. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Best-Corrected Visual Acuity (BCVA) | 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. |
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA - Jules Stein Eye Institute | Los Angeles | California | 90095-7065 | United States | ||
| Vitreo Retinal Associates PA - The Millennium Center |
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| BIIB112 | Genetic | Administered as specified in the treatment arm. |
|
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| 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 | 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. | Up to 5 years |
| Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants | 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. | Up to 5 years |
| Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants | 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. | Up to 5 years |
| Assessment of Fundus Autofluorescence (AF) at Each Visit | 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). | Up to 5 years |
| Assessment of Fundus Photography at Each Visit | Fundus photography will be performed on both eyes following the dilation of the participant's pupils. | Up to 5 years |
| Assessment of Spectral Domain Optical Coherence Tomography (SD-OCT) at Each Visit | 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). | Up to 5 years |
| Assessment of Microperimetry at Each Visit | Microperimetry will be performed on both eyes to assess changes in retinal sensitivity within the macula. It will be reported in decibels (dB). | Up to 5 years |
| Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25) | 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. | Up to 5 years |
| Change from Baseline in Visual Field | The outcome measure will be assessed in BIIB112-treated participants. | Up to 5 years |
| Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants | 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. | 18 Months to 60 Months, Post-Day 0 Visits |
| Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112-treated Participants | 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. | 18 Months to 60 Months, Post-Day 0 Visits |
| Gainesville |
| Florida |
| 32607 |
| United States |
| University of Miami | Miami | Florida | 33136 | United States |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287-0005 | United States |
| MEEI Massachusets Eye and Ear Infirmary | Boston | Massachusetts | 02114-3002 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Cincinnati Eye Institute - Blue Ash | Cincinnati | Ohio | 45242-5664 | United States |
| OHSU - Casey Eye Institute | Portland | Oregon | 97239 | United States |
| Retina Foundation of the Southwest | Dallas | Texas | 75231-5080 | United States |
| University of Wisconsin School of Medicine | Madison | Wisconsin | 53705-3644 | United States |
| Instituto Genetica Ocular | São Paulo | 04039 | Brazil |
| The Northern Alberta Clinical Trials and Research Centre | Edmonton | Alberta | T5H 3V9 | Canada |
| The University of British Columbia - Eye Care Centre | Vancouver | British Columbia | V5Z 39N | Canada |
| McGill University Health Centre | Montreal | Quebec | H4A 3JI | Canada |
| Rigshospitalet-Glostrup, Oejenafdelingen | Glostrup Municipality | 2600 | Denmark |
| Helsinki University Central Hospital (HUCH) | Helsinki | 00290 HUS | Finland |
| CHU Montpellier - Saint Eloi | Montpellier | 34295 | France |
| Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts | Paris | 75012 | France |
| Universitäts-Augenklinik Bonn | Bonn | 53127 | Germany |
| Universitats Klinikum Tubingen - Institute for Ophthalmic Research | Tübingen | 72076 | Germany |
| Radboudumc | Nijmegen | 6525 GA | Netherlands |
| Moorfields Eye Hospital | London | EC1V 2PD | United Kingdom |
| Manchester Royal Eye Hopsital | Manchester | M13 9WL | United Kingdom |
| John Radcliffe Hospital | Oxford | OX3 9DU | United Kingdom |
| Southampton General Hospital | Southampton | SO16 6YD | United Kingdom |
| ID | Term |
|---|---|
| D015794 | Choroideremia |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D015862 | Choroid Diseases |
| D014603 | Uveal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
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| ID | Term |
|---|---|
| C000730043 | Cotoretigene Toliparvovec |
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