Title

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

    Phase 1/Phase 2
  • Study Type

    Interventional
  • Status

    Completed No Results Posted
  • Intervention/Treatment

    aav-rpgr ...
  • Study Participants

    50
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).
This study was previously posted by NightstaRx Ltd. In October, 2020, sponsorship of the trial was transferred to Biogen.
Study Started
Mar 16
2017
Primary Completion
Nov 18
2020
Study Completion
Nov 18
2020
Last Update
Nov 22
2021

Biological BIIB112

Administered as specified in the treatment arm

  • Other names: AAV8-RPGR

Part 1: BIIB112 Dose 1 Experimental

Participants will receive a single Dose 1 of BIIB112 by sub-retinal injection on Day 0.

Part 1: BIIB112 Dose 2 Experimental

Participants will receive a single Dose 2 of BIIB112 by sub-retinal injection on Day 0.

Part 1: BIIB112 Dose 3 Experimental

Participants will receive a single Dose 3 of BIIB112 by sub-retinal injection on Day 0.

Part 1: BIIB112 Dose 4 Experimental

Participants will receive a single Dose 4 of BIIB112 by sub-retinal injection on Day 0.

Part 1: BIIB112 Dose 5 Experimental

Participants will receive a single Dose 5 of BIIB112 by sub-retinal injection on Day 0.

Part 1: BIIB112 Dose 6 Experimental

Participants will receive a single Dose 6 of BIIB112 by sub-retinal injection on Day 0.

Part 2: BIIB112 High Dose Experimental

Participants will receive a single high dose of BIIB112 by sub-retinal injection.

Part 2: BIIB112 Low Dose Experimental

Participants will receive a single low dose of BIIB112 by sub-retinal injection.

Part 2: Untreated Group No Intervention

Participants will receive no intervention to allow for a controlled comparison.

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.
No Results Posted