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

Biogen

Status and phase: Completed; Phase 2; Phase 1

Conditions: X-Linked Retinitis Pigmentosa

Treatments: Biological: BIIB112

Study type: Interventional

Funder types: Industry

Identifiers

NCT03116113

274RP101

2016-003852-60 (EudraCT Number)

Details and patient eligibility

About

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).

Full description

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Enrollment: 50 patients

Sex: Male

Ages: 10+ years old

Volunteers: No Healthy Volunteers

Inclusion and 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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50 participants in 9 patient groups

Part 1: BIIB112 Dose 1

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 1: BIIB112 Dose 2

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 1: BIIB112 Dose 3

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 1: BIIB112 Dose 4

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 1: BIIB112 Dose 5

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 1: BIIB112 Dose 6

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 2: BIIB112 High Dose

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 2: BIIB112 Low Dose

Experimental group

Description:

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

Treatment:

Biological: BIIB112

Part 2: Untreated Group

No Intervention group

Description:

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