CompletedPhase 2

A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111

United States, France, Germany66 participantsStarted 2017-11-29

Plain-language summary

The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).

Who can participate

Age range

18 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Are willing and able to give informed consent for participation in the study to have both eyes treated.
. Have documentation of a genetically-confirmed diagnosis of CHM.
. Have active disease clinically visible within the macular region of both eyes.
. Have a BCVA of ≥34 ETDRS letters (20/200 or better Snellen acuity) in both eyes, or in the untreated eye, if the other eye was previously treated with BIIB111\*
. For participants who received treatment with BIIB111 in an antecedent study, have biological samples available to complete an adequate immunology profile.

Exclusion criteria

. Have a history of amblyopia or inflammatory disorder in either eye.
. Are unwilling to use barrier contraception methods or abstain from sexual intercourse for a period of 3 months following treatment with BIIB111 in either eye.
. Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye.
. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the participants at risk because of participation in the study, or may influence the results of the study or the participant's ability to participate in the study. This includes but is not limited to a potential participants:

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12

Timeframe: Month 12

Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12

Timeframe: Month 12

Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination

Timeframe: Baseline, Month 12

Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Dilated Ophthalmoscopy

Timeframe: Baseline, Month 12

Ophthalmic Examination Assessment: Number of Participants With Lens Opacity Grading

Timeframe: Month 12

Spectral Domain Optical Coherence Tomography (SD-OCT): Foveal Subfield Thickness at Month 12

Timeframe: Month 12

SD-OCT: Total Macular Volume at Month 12

Trial details

NCT IDNCT03507686

SponsorBiogen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-06-29

Results submitted2023-06-29

View on ClinicalTrials.gov More Choroideremia trials

Who can participate

Age range

18 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Are willing and able to give informed consent for participation in the study to have both eyes treated.
. Have documentation of a genetically-confirmed diagnosis of CHM.
. Have active disease clinically visible within the macular region of both eyes.
. Have a BCVA of ≥34 ETDRS letters (20/200 or better Snellen acuity) in both eyes, or in the untreated eye, if the other eye was previously treated with BIIB111\*
. For participants who received treatment with BIIB111 in an antecedent study, have biological samples available to complete an adequate immunology profile.

Exclusion criteria

. Have a history of amblyopia or inflammatory disorder in either eye.
. Are unwilling to use barrier contraception methods or abstain from sexual intercourse for a period of 3 months following treatment with BIIB111 in either eye.
. Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye.
. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the participants at risk because of participation in the study, or may influence the results of the study or the participant's ability to participate in the study. This includes but is not limited to a potential participants:

What they're measuring

Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12

Timeframe: Month 12

Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12

Timeframe: Month 12

Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination

Timeframe: Baseline, Month 12

Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Dilated Ophthalmoscopy

Timeframe: Baseline, Month 12

Ophthalmic Examination Assessment: Number of Participants With Lens Opacity Grading

Timeframe: Month 12

Spectral Domain Optical Coherence Tomography (SD-OCT): Foveal Subfield Thickness at Month 12

Timeframe: Month 12

SD-OCT: Total Macular Volume at Month 12