NCT04926129

Brief Summary

The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
201

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Sep 2017

Longer than P75 for all trials

Geographic Reach
7 countries

22 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 13, 2017

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

July 30, 2019

Completed
1.9 years until next milestone

First Posted

Study publicly available on registry

June 14, 2021

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 16, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 16, 2022

Completed
Last Updated

February 10, 2023

Status Verified

February 1, 2023

Enrollment Period

5.3 years

First QC Date

July 30, 2019

Last Update Submit

February 9, 2023

Conditions

X-Linked Retinitis Pigmentosa

Keywords

RP GTPase regulator (RPGR)Degenerative retinal diseaseXLRPNatural historyRare disease

Outcome Measures

Primary Outcomes (2)

  • Change from Baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) Best-corrected Visual Acuity (BCVA)

    BCVA will be assessed for both eyes using the ETDRS visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If \<20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant.

    Up to Month 24

  • Change from Baseline in Retinal Sensitivity Assessed with Microperimetry

    Macular analyser integrity assessment (MAIA) microperimetry will be conducted for both eyes to assess changes in retinal sensitivity within the macula.

    Up to Month 24

Secondary Outcomes (17)

  • Change from Baseline in Contrast Sensitivity

    Up to Month 24

  • Change from Baseline in Low Luminance Visual Acuity (LLVA)

    Up to Month 24

  • Change from Baseline in 25-item Visual Function Questionnaire (VFQ-25) Score

    Up to Month 24

  • Change from Baseline in Retinitis Pigmentosa (RP)-Specific Patient-Reported Outcome (PRO) Questionnaire

    Up to Month 24

  • Change from Baseline in EuroQol-5 Dimension 5-level (EQ-5D-5L)

    Up to Month 24

  • +12 more secondary outcomes

Study Arms (2)

Subgroup 1

Participant's eye with Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA) ≥74 letters (Equivalent to: Snellen 6/9 or 20/32; decimal 0.63; Logarithm of the minimum angle of resolution \[LogMAR\] 0.2) will be enrolled.

Other: Other: Assessments

Subgroup 2

Participant's eye with ETDRS BCVA 34-73 letters, inclusive (Equivalent to: Snellen 6/12 - 6/60 or 20/40 - 20/200; decimal 0.5 - 0.1; LogMAR 0.3-1.0) will be enrolled.

Other: Other: Assessments

Interventions

Other: AssessmentsOTHER

Administered as specified in the treatment arm.

Also known as: BIIB112
Subgroup 1Subgroup 2

Eligibility Criteria

Age7 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

201 participants with X-linked Retinitis Pigmentosa (XLRP).

You may qualify if:

  • Have documentation of a pathogenic mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene.
  • Are willing and able to undergo ophthalmic examinations, as required by protocol, for up to 24 months
  • Have an ETDRS BCVA in at least 1 eye of ≥34 letters (Equivalent to Snellen ≥ 6/60 or 20/200; decimal 0.1; LogMAR 1.0).
  • Mean retinal sensitivity in the eligible eye as assessed by microperimetry:
  • Males with a mean retinal sensitivity of 68 loci ranging from ≥0.1 decibels (dB) and ≤20 dB.
  • Females with a mean retinal sensitivity of 68 loci ranging from ≥0.1 dB and ≤25 dB.
  • If female, have symptomatic disease with impairment of visual function.

You may not qualify if:

  • Have a history of amblyopia in the eligible eye.
  • Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may put the participant at risk because of participation in the study, may influence the results of the study, may influence the participant's ability to perform study diagnostic tests, or impact the participant's ability to participate in the study. This includes clinically significant cataracts.
  • Have participated in another research study involving an investigational medicinal product in the past 12 weeks or received a gene/cell-based therapy at any time previously (including but not limited to Intelligent Implant System implantation, ciliary neurotrophic factor therapy, nerve growth factor therapy).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (22)

Research Site

Phoenix, Arizona, 85020, United States

Location

Research Site

Los Angeles, California, 90095, United States

Location

Research Site

San Francisco, California, 94143, United States

Location

Research Site

Miami, Florida, 33136, United States

Location

Research Site

New York, New York, 10032, United States

Location

Research Site

Portland, Oregon, 97239, United States

Location

Research Site

Dallas, Texas, 75231, United States

Location

Research Site

Houston, Texas, 77030, United States

Location

Research Site

Salt Lake City, Utah, 84132, United States

Location

Research Site

Madison, Wisconsin, 53705, United States

Location

Research Site

Montreal, Quebec, H4A 3J1, Canada

Location

Research Site

Vancouver, V5Z3N9, Canada

Location

Research Site

Helsinki, Finland

Location

Research Site

Montpellier, France

Location

Research Site

Bonn, Germany

Location

Research Site

Tübingen, Germany

Location

Research Site

Leiden, Netherlands

Location

Research Site

Nijmegen, Netherlands

Location

Research Site

Leeds, United Kingdom

Location

Research Site

Manchester, United Kingdom

Location

Research Site

Oxford, United Kingdom

Location

Research Site

Southampton, United Kingdom

Location

Related Publications (1)

  • von Krusenstiern L, Liu J, Liao E, Gow JA, Chen G, Ong T, Lotery AJ, Jalil A, Lam BL, MacLaren RE; XIRIUS Part 1 Study GroupXOLARIS Study Group. Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations. JAMA Ophthalmol. 2023 Mar 1;141(3):275-283. doi: 10.1001/jamaophthalmol.2022.6254.

    PMID: 36757689DERIVED

MeSH Terms

Conditions

Rare Diseases

Interventions

Cotoretigene Toliparvovec

Condition Hierarchy (Ancestors)

Disease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Medical Director

    Biogen

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 30, 2019

First Posted

June 14, 2021

Study Start

September 13, 2017

Primary Completion

December 16, 2022

Study Completion

December 16, 2022

Last Updated

February 10, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share

Locations

GB(4)US(10)FI(1)FR(1)DE(2)NL(2)CA(2)