Choroideremia (CHM) MedDRA version: 20.1 Level: LLT Classification code 10008791 Term: Choroideremia System Organ Class: 100000004853
Conditions
Interventions
Product Code: BIIB111 (AAV2-REP1)
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: timrepigene emparvovec
CAS Number: 1905415-02-2
Current Sponsor code: AAV2-REP1
Other descriptive n
Sponsors
NightstaRx Ltd (A Biogen Company)
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: 1. Are willing and able to give informed consent for participation in the study to have both eyes treated. 2. Have documentation of a genetically-confirmed diagnosis of CHM. 3. Have active disease clinically visible within the macular region of both eyes. 4. 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* *If previously treated with BIIB111 in an antecedent study, participants may be eligible for participation following Sponsor approval. 5. For participants who received treatment with BIIB111 in an antecedent study, have biological samples available to complete an adequate immunology profile. NOTE: Other protocol defined Inclusion criteria may apply Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 57 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
Exclusion criteria: Key Exclusion Criteria: 1. Have a history of amblyopia or inflammatory disorder in either eye. 2. 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. 3. Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye. 4. 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: • with a contraindication to oral corticosteroid (e.g., prednisolone/prednisone) • with clinically significant cataract in either eye • who, in the clinical opinion of the Investigator, is not an appropriate candidate for sub-retinal surgery. 5. 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. NOTE: Other protocol defined Exclusion criteria may apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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).;Secondary Objective: Not Applicable;Primary end point(s): • Best-Corrected Visual Acuity(BCVA) • Ophthalmic Examination Assessment: Intraocular Pressure (IOP) • Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination • Ophthalmic Examination Assessment: Dilated Ophthalmoscopy • Ophthalmic Examination Assessment: Lens Opacity Grading • Spectral Domain Optical Coherence Tomography (SD-OCT) • Fundus Autofluorescence (AF) • Fundus Photography • Microperimetry • Percentage of Participants with Adverse Events (AEs) • Vector Shedding Post-treatment • Number of Participants with Cell-Based and Antibody-Based Immune Responses Against BIIB111 • Number of Participants with Significant Change in Vital Signs Post-Treatment;Timepoint(s) of evaluation of this end point: Assessments will be performed at timepoints described in the protocol / Up to 2 years. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change from Baseline in BCVA • Change from Baseline in AF • Change from Baseline in SD-OCT • Change from Baseline in Microperimetry ;Timepoint(s) of evaluation of this end point: Assessments will be performed at timepoints described in the protocol / Up to 2 years. | — |
Countries
France, Germany, United States
Contacts
Public ContactClinical Operations Department
NightstaRx Ltd (A Biogen Company)
Outcome results
None listed