A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112

• Register: ClinicalTrials.gov
• Last refreshed on: 20 February 2023
• Main ID: NCT03116113
• Date of registration: 29/03/2017
• Prospective Registration: No
• Primary sponsor: NightstaRx Ltd, a Biogen Company
• Public title: A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 XIRIUS
• Scientific title: A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)
• Date of first enrolment: March 16, 2017
• Target sample size: 50
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03116113
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Biogen

Key inclusion & exclusion criteria

Key Inclusion Criteria:

Part 1:

- Participants with genetically confirmed diagnosis of XLRP (with RPGR mutation).

- Participant with active disease clinically visible within the macular region in both
eyes.

Part 2:

- Participant with mean total retinal sensitivity in the study eye as assessed by
microperimetry = 0.1 dB and =8 dB.

Key exclusion Criteria:

Parts 1 and 2:

- Participant with history of amblyopia in either eye.

- Participated in a gene therapy trial previously or a clinical trial with an
investigational drug in the past 12 weeks or received a gene/cell-based therapy at any
time previously.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.



Age minimum: 10 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

X-Linked Retinitis Pigmentosa

Intervention(s)

Biological: BIIB112

Primary Outcome(s)

Part 2: Number of Participants with TEAEs [Time Frame: Up to Month 12]

Part 1: Number of Participants with Dose-Limiting Toxicities (DLTs) [Time Frame: Up to Month 24]

Part 2: Percentage of Study Eyes with =7 Decibels (dB) Improvement from Baseline at =5 of the 16 Central Loci of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry [Time Frame: Months 12]

Part 1: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [Time Frame: Up to Month 24]

Secondary Outcome(s)

Part 1 and 2: Percentage of Eyes with Change from Baseline > -5 Letters for LLVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =15 Letters Loss from Baseline for LLVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Parts 1 and 2: Change from Baseline in Mean Sensitivity of the 16 Central Loci [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =10 Letters Increase from Baseline for LLVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =5 Letters Increase from Baseline for LLVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =5 Letters Loss from Baseline for LLVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Study Eyes with =7 dB Improvement from Baseline at =5 Out of the 68 Loci in Microperimetry [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Part 1: Change from Baseline in Central Horizontal Ellipsoid Width [Time Frame: Month 1, 3, 6, 9, 12, 18 and 24]

Part 2: Change from Baseline in Volume of 30-Degree Hill of Vision Assessed by Octopus 900 [Time Frame: Baseline, Month 3, 6 and 12]

Part 2: Change from Baseline in Volume of Full Field Hill of Vision Assessed by Octopus 900 [Time Frame: Baseline, Month 3, 6 and 12]

Parts 1 and 2: Change from Baseline in Low Luminance Visual Acuity (LLVA) Score [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Part 1: Change from Baseline in Fundus Autofluorescence- Distance from Foveal Center (FC) to Nearest Border of Preserved Autofluorescence [Time Frame: Month 1, 3, 6, 9, 12, 18 and 24]

Part 1: Change from Baseline in Contrast Sensitivity [Time Frame: Month 3, 6, 12 and 24]

Parts 1 and 2: Percentage of Eyes with a =10 Letters Loss from Baseline for BCVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Part 1: Change from Baseline in Central Ellipsoid Area [Time Frame: Month 1, 3, 6, 9, 12, 18 and 24]

Parts 1 and 2: Percentage of Eyes with a =15 Letters Loss from Baseline for BCVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Part 1: Change from Baseline in Fundus Autofluorescence- Total Area of Preserved Autofluoroscence [Time Frame: Month 1, 3, 6, 12, 18 and 24]

Parts 1 and 2: Percentage of Eyes with a =10 Letters Loss from Baseline for LLVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Part 1: Change from Baseline in Volume of Full Field Hill of Vision [Time Frame: Month 6, 12, and 24]

Parts 1 and 2: Change from Baseline in Best Corrected Visual Acuity (BCVA) Score [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2: Change from Baseline in Mean Sensitivity of the 68 Central Loci [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =10 Letters Increase from Baseline for BCVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2 : Percentage of Study Eyes with =7 dB Improvement from Baseline at =5 out of the 16 Central Loci in Microperimetry [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =5 Letters Loss from Baseline for BCVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =15 Letters Increase from Baseline for LLVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12]

Part 1: Change from Baseline in Volume of 30-Degree Hill of Vision [Time Frame: Month 6, 12 and 24]

Parts 1 and 2: Percentage of Eyes with a =15 Letters Increase from Baseline for BCVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with a =5 Letters Increase from Baseline for BCVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Parts 1 and 2: Percentage of Eyes with Change from Baseline > -5 Letters for BCVA [Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12]

Secondary ID(s)

274RP101

NSR-RPGR-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors