Trial · NCT03507686
A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
NCT03507686, Phase 2, Completed, Biogen; results first posted 2023-09-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111. Trials, record NCT03507686. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03507686
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
- NCT number
- NCT03507686
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 66 participants, actual
- Lead sponsor
- Biogen
- Conditions
- Choroideremia
- Interventions
- BIIB111
- First posted
- 2018-04-25
- Last updated
- 2024-02-23
- Other registries' IDs
- eudract:2017-002395-75
- Results first posted
- 2023-09-18
Source ClinicalTrials.gov
Registered outcomes42
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | Month 12 | 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. BCVA is reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eyes. 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. |
| Primary | Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | Month 12 | IOP, the fluid pressure inside the eye was measured and reported in millimeters mercury (mmHg). |
| Primary | Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination | Baseline, Month 12 | Slit lamp examinations of study eyes included examination of Cornea, Conjunctiva, Iris, Lens, and Anterior Segment. |
| Primary | Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Dilated Ophthalmoscopy | Baseline, Month 12 | Dilated Ophthalmoscopy examination of study eyes included examination of Vitreous, Macula, Peripheral retina, Choroid, and Optic nerve. |
| Primary | Ophthalmic Examination Assessment: Number of Participants With Lens Opacity Grading | Month 12 | The following lens opacity grades are reported for categories 1-4: Nuclear Opalescence grade, Nuclear Color grade, Cortical Cataract grade, and Posterior Cataract grade. Opacification severity is graded on a decimal scale, scores can range from 0.1 (no opacity) to 6.9 (maximum opacity) for the Nuclear Opalescence and Nuclear Color grades; and scores can range from 0.1 (lens clear) to 5.9 (lens unclear) for the Cortical and Posterior Cataract grades. Category 1 includes values 1, 1.0 and 0.x, Category 2 includes values 2, 2.0 and 1.x, Category 3 includes values 3, 3.0 and 2.x, and Category 4 includes values 4, 4.0, 3.x and any values above 4. For each opacification type the higher grading scores indicate greater severity. |
| Primary | Spectral Domain Optical Coherence Tomography (SD-OCT): Foveal Subfield Thickness at Month 12 | Month 12 | SD-OCT was used to assess change in foveal subfield thickness. The measurements were taken after dilation of the participant's pupil. |
| Primary | SD-OCT: Total Macular Volume at Month 12 | Month 12 | SD-OCT was used to assess change in total macular volume. The measurements were taken after dilation of the participant's pupil. |
| Primary | SD-OCT: Central Horizontal Ellipsoid Width at Month 12 | Month 12 | SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. |
| Primary | SD-OCT: Central Ellipsoid Area at Month 12 | Month 12 | SD-OCT was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. |
| Primary | SD-OCT: Square Root of Central Ellipsoid Area at Month 12 | Month 12 | SD-OCT was used to assess change in square root of central ellipsoid area. The measurements were taken after dilation of the participant's pupil. |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 registered outcomesOutcomes42
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | letters | MEAN |
| PRIMARY | Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | mmHg | MEAN |
| PRIMARY | Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination | Baseline, Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified category at specified timepoint. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Dilated Ophthalmoscopy | Baseline, Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified category at specified timepoint. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Ophthalmic Examination Assessment: Number of Participants With Lens Opacity Grading | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified category of specified parameter. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Spectral Domain Optical Coherence Tomography (SD-OCT): Foveal Subfield Thickness at Month 12 | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | microns (µ) | MEAN |
| PRIMARY | SD-OCT: Total Macular Volume at Month 12 | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | cubic millimeters (mm^3) | MEAN |
| PRIMARY | SD-OCT: Central Horizontal Ellipsoid Width at Month 12 | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | microns (μ) | MEAN |
| PRIMARY | SD-OCT: Central Ellipsoid Area at Month 12 | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | square millimeters (mm^2) | MEAN |
| PRIMARY | SD-OCT: Square Root of Central Ellipsoid Area at Month 12 | Month 12 | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | mm | MEAN |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 outcomesResults by group346
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | Treatment Period 1: BIIB111 | Study Eye 1 | 68.0 | Standard Deviation 25.18 | letters |
| Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | Treatment Period 2: BIIB111 | Study Eye 1 | 76.5 | Standard Deviation 13.78 | letters |
| Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | Treatment Period 1: BIIB111 | Study Eye 2 | 82.3 | Standard Deviation 8.67 | letters |
| Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | Treatment Period 2: BIIB111 | Study Eye 2 | 81.2 | Standard Deviation 10.82 | letters |
| Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | Treatment Period 1: BIIB111 | Study Eye 1 | 13.5 | Standard Deviation 3.09 | mmHg |
| Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | Treatment Period 2: BIIB111 | Study Eye 1 | 13.5 | Standard Deviation 3.12 | mmHg |
| Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | Treatment Period 1: BIIB111 | Study Eye 2 | 14.9 | Standard Deviation 2.50 | mmHg |
| Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | Treatment Period 2: BIIB111 | Study Eye 2 | 13.5 | Standard Deviation 3.55 | mmHg |
| Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination | Treatment Period 1: BIIB111 | Cornea: Study Eye 1: Baseline | 0 | Participants | |
| Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination | Treatment Period 2: BIIB111 | Cornea: Study Eye 1: Baseline | 0 | Participants |
1 to 10 of 346Source ClinicalTrials.gov
See all 346 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period 1(Day 1 up to Month 12) | STARTED | Treatment Period 1: BIIB111 | 60 | |
| Treatment Period 1(Day 1 up to Month 12) | STARTED | Treatment Period 2: BIIB111 | 0 | |
| Treatment Period 1(Day 1 up to Month 12) | COMPLETED | Treatment Period 1: BIIB111 | 50 | |
| Treatment Period 1(Day 1 up to Month 12) | COMPLETED | Treatment Period 2: BIIB111 | 0 | |
| Treatment Period 1(Day 1 up to Month 12) | NOT COMPLETED | Treatment Period 1: BIIB111 | 10 | |
| Treatment Period 1(Day 1 up to Month 12) | NOT COMPLETED | Treatment Period 2: BIIB111 | 0 | |
| Treatment Period 2 (Month13 to Month24) | STARTED | Treatment Period 1: BIIB111 | 0 | |
| Treatment Period 2 (Month13 to Month24) | STARTED | Treatment Period 2: BIIB111 | 56 | |
| Treatment Period 2 (Month13 to Month24) | COMPLETED | Treatment Period 1: BIIB111 | 0 | |
| Treatment Period 2 (Month13 to Month24) | COMPLETED | Treatment Period 2: BIIB111 | 53 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events88
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Conjunctival haemorrhage | Eye disorders | Treatment Period 1: BIIB111 | 38 | 60 | |
| other | Conjunctival haemorrhage | Eye disorders | Treatment Period 2: BIIB111 | 36 | 56 | |
| other | Anterior chamber cell | Eye disorders | Treatment Period 1: BIIB111 | 29 | 60 | |
| other | Anterior chamber cell | Eye disorders | Treatment Period 2: BIIB111 | 32 | 56 | |
| other | Eye pain | Eye disorders | Treatment Period 1: BIIB111 | 9 | 60 | |
| other | Eye pain | Eye disorders | Treatment Period 2: BIIB111 | 10 | 56 | |
| other | Foreign body sensation in eyes | Eye disorders | Treatment Period 1: BIIB111 | 11 | 60 | |
| other | Foreign body sensation in eyes | Eye disorders | Treatment Period 2: BIIB111 | 10 | 56 | |
| other | Conjunctival hyperaemia | Eye disorders | Treatment Period 1: BIIB111 | 11 | 60 | |
| other | Conjunctival hyperaemia | Eye disorders | Treatment Period 2: BIIB111 | 6 | 56 |
1 to 10 of 88Source ClinicalTrials.gov
See all 88 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Period 1: BIIB111 | 0 | 60 |
| Total, all-cause mortality | Treatment Period 2: BIIB111 | 1 | 56 |
| Total, other adverse events | Treatment Period 1: BIIB111 | 59 | 60 |
| Total, other adverse events | Treatment Period 2: BIIB111 | 53 | 56 |
| Total, serious adverse events | Treatment Period 1: BIIB111 | 12 | 60 |
| Total, serious adverse events | Treatment Period 2: BIIB111 | 8 | 56 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Treatment Period 1: BIIB111 vs Treatment Period 2: BIIB111: Number of Participants With Anti-drug Antibodies Post-treatment at Month 12 | Trial comparisons |
| Treatment Period 1: BIIB111 vs Treatment Period 2: BIIB111: deaths from any cause | Trial comparisons |
| Treatment Period 1: BIIB111 vs Treatment Period 2: BIIB111: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03507686
GET https://conductscience.org/api/trials/v1/NCT03507686/registered?page=2