Clinical trial

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)

Name

274RP101

Description

The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP).

Trial arms

Trial start

2017-03-08

Estimated PCD

2020-11-18

Trial end

2020-11-18

Status

Completed

Phase

Early phase I

Treatment

BIIB112

Administered as specified in the treatment arm.

Arms:

Part 1: BIIB112 Dose 1, Part 1: BIIB112 Dose 2, Part 1: BIIB112 Dose 3, Part 1: BIIB112 Dose 4, Part 1: BIIB112 Dose 5, Part 1: BIIB112 Dose 6, Part 2: BIIB112 High Dose, Part 2: BIIB112 Low Dose

Other names:

AAV8-RPGR

Size

Primary endpoint

Part 1: Number of Participants With Dose-Limiting Toxicities (DLTs)

Up to Month 24

Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Day 0 (surgery) in Part 1 of the study up to 24 months

Part 2: Percentage of Study Eyes With ≥7 Decibels (dB) Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry

Month 12

Part 2: Number of Participants With TEAEs

Day 0 (surgery) in Part 2 of the study up to 12 months

Eligibility 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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 50, 'type': 'ACTUAL'}}

Updated at

2024-01-18

1 organization

1 product

1 indication

Organization

Biogen

Product

BIIB112

Indication

Retinitis Pigmentosa