CompletedPhase 2

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

United States, France66 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 range18 Years

SexMALE

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:
✕. Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously, except if treated within an antecedent study with BIIB111.

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

Timeframe: 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 range18 Years

SexMALE

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:
✕. Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously, except if treated within an antecedent study with BIIB111.

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

Timeframe: Month 12