A Phase 3 interventional study of BIIB111 in Choroideremia, sponsored by Biogen. Completed at 18 sites in 8 countries. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-12-07.
Sponsored by Biogen · Phase 3, Interventional, and Treatment
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).
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
34 studies on the registry are indexed under Choroideremia; 7 are open to participants now.
This study's enrollment of 169 is above the median of 15 across 22 interventional studies indexed under Choroideremia.
Browse Choroideremia studies →Biogen is the lead sponsor of 511 studies on the registry; 24 are open to participants now.
Of its 100 completed or terminated interventional studies of FDA-regulated products, 50 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive a single administration of high dose BIIB111 in one eye through sub-retinal injection after vitrectomy.
Genetic: BIIB111
Participants will receive a single administration of low dose BIIB111 in one eye through sub-retinal injection after vitrectomy.
Genetic: BIIB111
Participants will receive no sham surgery or study medication.
Administered as specified in the treatment arm.
Also known as: AAV2-REP1
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.
Time frame: Month 12
Change From Baseline in BCVA at Month 12 Reported as Letters Measured by the ETDRS Chart in Study Eye
BCVA was assessed for study eye using the ETDRS 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 was 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 VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate decline in BCVA.
Time frame: Baseline, Month 12
Percentage of Participants With a ≥10 -Letter Improvement From Baseline in BCVA at Month 12 Measured by the ETDRS Chart
BCVA was assessed for both eyes using the ETDRS 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 ≥10 -letter improvement from baseline in BCVA at Month 12 was reported for both eyes.
Time frame: 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
BCVA was assessed for both eyes using the ETDRS 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 no decrease or a decrease of \<5 letters from baseline in BCVA at Month 12 was reported for both eyes.
Time frame: Month 12
Change From Baseline in BCVA at Months 4 and 8 Reported as Letters Measured by the ETDRS Chart
BCVA was assessed for both eyes using the ETDRS 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 was reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in both the study and fellow eyes. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate decline in BCVA.
Time frame: Baseline, Months 4 and 8
Change From Baseline in Total Area of Preserved Autofluorescence (AF) at Month 12 in Study Eye
Fundus AF was used to assess change in total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper AF compared with the background areas of surrounding atrophy. A negative change from baseline indicate decline in total area of preserved AF.
Time frame: Baseline, Month 12
Change From Baseline in Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 in Study Eye
Fundus AF was used to assess change in distance from foveal center (FC) to nearest border of preserved AF. A negative change from baseline indicate decline in distance from FC to nearest border of AF.
Time frame: Baseline, Month 12
Change From Baseline in the Foveal Subfield Thickness Using Spectral Domain Optical Coherence Tomography (SD-OCT) at Month 12 in Study Eye
SD-OCT was used to assess change in foveal subfield thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in foveal subfield thickness.
Time frame: Baseline, Month 12
Change From Baseline in the Total Macular Volume Using SD-OCT at Month 12 in Study Eye
SD-OCT was used to assess change in total macular volume. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in total macular volume.
Time frame: Baseline, Month 12
Change From Baseline in the Central Horizontal Ellipsoid Width Using SD-OCT at Month 12 in Study Eye
SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central horizontal ellipsoid width.
Time frame: Baseline, Month 12
Change From Baseline in the Central Ellipsoid Area Using SD-OCT at Month 12 in Study Eye
SD-OCT was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central ellipsoid area.
Time frame: Baseline, Month 12
Change From Baseline in the Choroidal Thickness Using SD-OCT at Month 12 in Study Eye
SD-OCT was used to assess change in choroidal thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in choroidal thickness.
Time frame: Baseline, Month 12
Change From Baseline in the Mean Retinal Sensitivity Using Microperimetry at Month 12 in Study Eye
Microperimetry was conducted to assess change in mean retinal sensitivity within the macula. Retinal mean sensitivity to light was measured in decibels (dBs) in multiple spots across the central and peripheral retina (entire visual field). Higher numbers (dBs) indicate higher retinal sensitivity. A negative change from baseline indicates decline in retinal sensitivity.
Time frame: Baseline, Month 12
Change From Baseline in the Bivariate Contour Ellipse Area 63% Using Microperimetry at Month 12 in Study Eye
Microperimetry was conducted to assess change in bivariate contour ellipse area 63%. A negative change from baseline indicates decline in bivariate contour ellipse area 63%.
Time frame: Baseline, Month 12
Change From Baseline in the Bivariate Contour Ellipse Area 95% Using Microperimetry at Month 12 in Study Eye
Microperimetry was conducted to assess change in bivariate contour ellipse area 95%. A negative change from baseline indicates decline in bivariate contour ellipse area 95%.
Time frame: Baseline, Month 12
Change From Baseline in Contrast Sensitivity Score at Month 12 in Study Eye
Change in contrast sensitivity was assessed by Pelli-Robson chart which uses a single large letter size (20/60 optotype), with contrast varying across groups of letters (6 per line), whose contrast varies from high to low. Participants read letters, starting with highest contrast, until they are unable to read 2 or 3 letters in a single group. Each group had three letters of the same contrast level, so there were three trials per contrast level. Score is assigned based on contrast of last group in which 2 or 3 letters were correctly read. Score is a measure of participant's log contrast sensitivity ranging from 0 (no letters read) to 2.25 (all letters read). Total CS score=\[(total letters correct-3) x 0.05\]. A negative change from baseline indicates worsening in contrast sensitivity score.
Time frame: Baseline, Month 12
Change From Baseline in Colour Vision Total Error Score at Month 12 in Study Eye
Colour vision total error scores were assessed by the Farnsworth Munsell 100 Hue Sort Test. Farnsworth Munsell 100 Hue Test requires placing 100 colour palettes in the correct order based upon colour hue. Scores are determined by the frequency and severity of any displacement in the correct order. One error equates to one misplaced hue, by one step or position. An error score of 0 indicates no errors in ordering the hues while error score greater than 500 indicates virtually no color discrimination. A Total Error Score of 0 to 128 could be seen in a normal population.
Time frame: Baseline, Month 12
Change From Baseline in Reading Speed Test at Month 12 in Study Eye
The reading speed (words per minute) was calculated using the following formula: \[number of words in the text - number of misread words\] / reading time x 60. The number of misread words and reading time is collected. A negative change from baseline indicates decline in reading speed.
Time frame: Baseline, Month 12
Change From Baseline in the 25-Item Visual Function Questionnaire (VFQ-25) Composite Scores at Month 12 in Study Eye
VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. A score of 0 represents the worst outcome and 100 represents the best outcome. A negative change from baseline indicates decline in VFQ-25 score.
Time frame: Baseline, Month 12
Participants were enrolled at the investigative sites in the United States, Germany, Finland, France, the United Kingdom, Canada, Netherlands, and Denmark from 11 December 2017 to 01 December 2020.
| Milestone | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Started | 66 | 34 | 69 |
| Completed | 62 | 34 | 65 |
| Not completed | 4 | 0 | 4 |
| Withdrew: Withdrawal by subject | 2 | 0 | 2 |
| Withdrew: Adverse event | 0 | 0 | 1 |
| Withdrew: Reason not specified | 2 | 0 | 1 |
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.
| percentage of participants | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Study Eye | 0 (0 to 5.8) | 2.9 (0.1 to 15.3) | 4.6 (1.0 to 12.9) |
| Fellow Eye | 0 (0 to 5.8) | 0 (0 to 10.3) | 3.1 (0.4 to 10.7) |
BCVA was assessed for study eye using the ETDRS 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 was 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 VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate decline in BCVA.
| letters | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in BCVA at Month 12 Reported as Letters Measured by the ETDRS Chart in Study Eye | -2.3 (-5.31 to 0.61) | -1.5 (-5.47 to 2.50) | -0.3 (-3.18 to 2.64) |
BCVA was assessed for both eyes using the ETDRS 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 ≥10 -letter improvement from baseline in BCVA at Month 12 was reported for both eyes.
| percentage of participants | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Study Eye | 1.6 (0.0 to 8.7) | 17.6 (6.8 to 34.5) | 13.8 (6.5 to 24.7) |
| Fellow Eye | 0 (0.0 to 5.8) | 8.8 (1.9 to 23.7) | 6.2 (1.7 to 15.0) |
BCVA was assessed for both eyes using the ETDRS 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 no decrease or a decrease of \<5 letters from baseline in BCVA at Month 12 was reported for both eyes.
| percentage of participants | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Study Eye | 67.7 (54.7 to 79.1) | 70.6 (52.5 to 84.9) | 83.1 (71.7 to 91.2) |
| Fellow Eye | 75.8 (63.3 to 85.8) | 85.3 (68.9 to 95.0) | 90.8 (81.0 to 96.5) |
BCVA was assessed for both eyes using the ETDRS 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 was reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in both the study and fellow eyes. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate decline in BCVA.
| letters | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline at Month 4: Study Eye | 0.2 ± 4.34 | -0.4 ± 14.16 | 0.6 ± 11.11 |
| Change From Baseline at Month 4: Fellow Eye | -1.1 ± 8.45 | 0.9 ± 6.67 | 2.0 ± 6.63 |
| Change From Baseline at Month 8: Study Eye | -0.8 ± 4.93 | -1.1 ± 13.13 | 0.1 ± 11.71 |
| Change From Baseline at Month 8: Fellow Eye | -0.4 ± 8.31 | 1.9 ± 7.06 | 1.6 ± 5.91 |
Fundus AF was used to assess change in total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper AF compared with the background areas of surrounding atrophy. A negative change from baseline indicate decline in total area of preserved AF.
| square millimeters (mm^2) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in Total Area of Preserved Autofluorescence (AF) at Month 12 in Study Eye | -0.3872 ± 0.0389 | -0.5413 ± 0.0501 | -0.5136 ± 0.0371 |
Fundus AF was used to assess change in distance from foveal center (FC) to nearest border of preserved AF. A negative change from baseline indicate decline in distance from FC to nearest border of AF.
| micrometers (µm) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 in Study Eye | 21.7796 ± 9.6234 | 23.6171 ± 12.3765 | 21.1660 ± 9.1863 |
SD-OCT was used to assess change in foveal subfield thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in foveal subfield thickness.
| µm | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Foveal Subfield Thickness Using Spectral Domain Optical Coherence Tomography (SD-OCT) at Month 12 in Study Eye | -6.3152 ± 2.7779 | -19.8654 ± 3.6998 | -15.7993 ± 2.7233 |
SD-OCT was used to assess change in total macular volume. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in total macular volume.
| cubic millimeters (mm^3) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Total Macular Volume Using SD-OCT at Month 12 in Study Eye | -0.0895 ± 0.0485 | -0.2876 ± 0.0646 | -0.2088 ± 0.0476 |
SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central horizontal ellipsoid width.
| µm | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Central Horizontal Ellipsoid Width Using SD-OCT at Month 12 in Study Eye | -81.9178 ± 20.6192 | -116.3811 ± 27.8213 | -170.7214 ± 20.4062 |
SD-OCT was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central ellipsoid area.
| mm^2 | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Central Ellipsoid Area Using SD-OCT at Month 12 in Study Eye | -0.2382 ± 0.0364 | -0.4229 ± 0.0527 | -0.3440 ± 0.0332 |
SD-OCT was used to assess change in choroidal thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in choroidal thickness.
| µm | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Choroidal Thickness Using SD-OCT at Month 12 in Study Eye | -5.4897 ± 2.1873 | -7.9579 ± 2.9207 | -4.6371 ± 2.1446 |
Microperimetry was conducted to assess change in mean retinal sensitivity within the macula. Retinal mean sensitivity to light was measured in decibels (dBs) in multiple spots across the central and peripheral retina (entire visual field). Higher numbers (dBs) indicate higher retinal sensitivity. A negative change from baseline indicates decline in retinal sensitivity.
| dBs | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Mean Retinal Sensitivity Using Microperimetry at Month 12 in Study Eye | -0.3443 ± 0.1134 | -0.2965 ± 0.1478 | -0.5015 ± 0.1112 |
Microperimetry was conducted to assess change in bivariate contour ellipse area 63%. A negative change from baseline indicates decline in bivariate contour ellipse area 63%.
| square degrees (deg^2) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Bivariate Contour Ellipse Area 63% Using Microperimetry at Month 12 in Study Eye | -3.2161 ± 1.2623 | -3.3771 ± 1.6405 | -2.5555 ± 1.2403 |
Microperimetry was conducted to assess change in bivariate contour ellipse area 95%. A negative change from baseline indicates decline in bivariate contour ellipse area 95%.
| deg^2 | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the Bivariate Contour Ellipse Area 95% Using Microperimetry at Month 12 in Study Eye | -10.1986 ± 4.1004 | -14.6926 ± 5.3134 | -11.6863 ± 4.0207 |
Change in contrast sensitivity was assessed by Pelli-Robson chart which uses a single large letter size (20/60 optotype), with contrast varying across groups of letters (6 per line), whose contrast varies from high to low. Participants read letters, starting with highest contrast, until they are unable to read 2 or 3 letters in a single group. Each group had three letters of the same contrast level, so there were three trials per contrast level. Score is assigned based on contrast of last group in which 2 or 3 letters were correctly read. Score is a measure of participant's log contrast sensitivity ranging from 0 (no letters read) to 2.25 (all letters read). Total CS score=\[(total letters correct-3) x 0.05\]. A negative change from baseline indicates worsening in contrast sensitivity score.
| score on a scale | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in Contrast Sensitivity Score at Month 12 in Study Eye | -0.0595 ± 0.0315 | -0.0130 ± 0.0417 | -0.0386 ± 0.0304 |
Colour vision total error scores were assessed by the Farnsworth Munsell 100 Hue Sort Test. Farnsworth Munsell 100 Hue Test requires placing 100 colour palettes in the correct order based upon colour hue. Scores are determined by the frequency and severity of any displacement in the correct order. One error equates to one misplaced hue, by one step or position. An error score of 0 indicates no errors in ordering the hues while error score greater than 500 indicates virtually no color discrimination. A Total Error Score of 0 to 128 could be seen in a normal population.
| score on a scale | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in Colour Vision Total Error Score at Month 12 in Study Eye | 35.0003 ± 23.5973 | 43.5915 ± 31.7720 | 54.3777 ± 24.0358 |
The reading speed (words per minute) was calculated using the following formula: \[number of words in the text - number of misread words\] / reading time x 60. The number of misread words and reading time is collected. A negative change from baseline indicates decline in reading speed.
| words per minute | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in Reading Speed Test at Month 12 in Study Eye | -149.5590 ± 10.9857 | -122.1333 ± 14.3276 | -127.4706 ± 9.9110 |
VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. A score of 0 represents the worst outcome and 100 represents the best outcome. A negative change from baseline indicates decline in VFQ-25 score.
| score on a scale | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Change From Baseline in the 25-Item Visual Function Questionnaire (VFQ-25) Composite Scores at Month 12 in Study Eye | -2.6971 ± 1.2786 | 1.7236 ± 1.6624 | 0.7964 ± 1.2308 |
Collected over From signing of informed consent through end of study (up to 12 months). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Untreated Control Group | 0/65 (0%) | 10/65 (15.4%) | 9/65 (13.8%) |
| BIIB111 Low Dose | 0/34 (0%) | 9/34 (26.5%) | 30/34 (88.2%) |
| BIIB111 High Dose | 0/65 (0%) | 11/65 (16.9%) | 54/65 (83.1%) |
| Event | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Visual acuity reducedEye disorders | 8/65 | 5/34 | 5/65 |
| Angina pectorisCardiac disorders | 0/65 | 1/34 | 0/65 |
| Myocardial infarctionCardiac disorders | 0/65 | 1/34 | 0/65 |
| CataractEye disorders | 0/65 | 1/34 | 0/65 |
| Retinal vascular occlusionEye disorders | 0/65 | 1/34 | 0/65 |
| UreterolithiasisRenal and urinary disorders | 0/65 | 1/34 | 0/65 |
| DiverticulitisInfections and infestations | 0/65 | 1/34 | 0/65 |
| Pilonidal cystInfections and infestations | 0/65 | 1/34 | 0/65 |
| SepsisInfections and infestations | 0/65 | 1/34 | 0/65 |
| Angina unstableCardiac disorders | 0/65 | 0/34 | 1/65 |
| Event | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose |
|---|---|---|---|
| Anterior chamber cellEye disorders | 0/65 | 14/34 | 24/65 |
| Conjunctival haemorrhageEye disorders | 0/65 | 13/34 | 26/65 |
| VitritisEye disorders | 0/65 | 10/34 | 16/65 |
| Eye painEye disorders | 0/65 | 6/34 | 11/65 |
| Eye irritationEye disorders | 0/65 | 5/34 | 8/65 |
| CataractEye disorders | 3/65 | 3/34 | 9/65 |
| Foreign body sensation in eyesEye disorders | 0/65 | 3/34 | 9/65 |
| Cataract subcapsularEye disorders | 0/65 | 4/34 | 4/65 |
| Conjunctival hyperaemiaEye disorders | 0/65 | 4/34 | 7/65 |
| Ocular hyperaemiaEye disorders | 0/65 | 4/34 | 7/65 |
Safety population included all randomized participants who either received study treatment (BIIB111) or a post-randomization study visit (control group).
| Age, Continuous(years) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose | Total |
|---|---|---|---|---|
| Mean | 49.4 ± 13.73 | 49.8 ± 12.62 | 47.5 ± 12.91 | 48.7 ± 13.15 |
| Sex: Female, Male(Participants) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose | Total |
|---|---|---|---|---|
| Female | 0 | 0 | 0 | 0 |
| Male | 65 | 34 | 65 | 164 |
| Ethnicity (NIH/OMB)(Participants) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose | Total |
|---|---|---|---|---|
| Hispanic or Latino | 3 | 1 | 4 | 8 |
| Not Hispanic or Latino | 51 | 26 | 54 | 131 |
| Unknown or Not Reported | 11 | 7 | 7 | 25 |
| Race/Ethnicity, Customized(Participants) | Untreated Control Group | BIIB111 Low Dose | BIIB111 High Dose | Total |
|---|---|---|---|---|
| Race — Asian | 0 | 0 | 1 | 1 |
| Race — American Indian or Alaska Native | 1 | 0 | 0 | 1 |
| Race — Black or African American | 1 | 0 | 0 | 1 |
| Race — White | 55 | 30 | 59 | 144 |
| Race — Other | 1 | 0 | 0 | 1 |
| Race — Not Reported | 7 | 4 | 5 | 16 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Data sharing details ↗ · vivli.org
This study is completed, as verified in Nov 2023. You cannot join it, but the record below documents what was studied.
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