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

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ClinicalTrials.gov Identifier: NCT03116113
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : August 22, 2019
Sponsor:
Information provided by (Responsible Party):
NightstaRx Ltd

Brief Summary:
A clinical trial of AAV8-RPGR retinal gene therapy for patients with X-linked retinitis pigmentosa

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Biological: AAV8-RPGR Phase 2 Phase 3

Detailed Description:
This is a Phase 1/2/3, first-in-human, multi-centre, dose-escalation interventional study of AAV8-RPGR in male subjects with genetically confirmed XLRP. Part I is a dose-selection study; Part II is a dose-expansion study, comparing 2 doses, and a third untreated group to allow for a controlled comparison of efficacy and safety.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Part I unmasked,Part II masked. Outcomes assessor masked to treatment; subject, investigator, sponsor masked to dose
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : March 16, 2017
Estimated Primary Completion Date : August 2020
Estimated Study Completion Date : August 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Part I: Dose 1 AAV8-RPGR
Single, subretinal administration of dose 1 as a single dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

Experimental: Part I: Dose 2 AAV8-RPGR
Single, subretinal administration of dose 2 as a single dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

Experimental: Part I: Dose 3 AAV8-RPGR
Single, subretinal administration of dose 3 as a single dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

Experimental: Part I: Dose 4 AAV8-RPGR
Single, subretinal administration of dose 4 as a single dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

Experimental: Part I: Dose 5 AAV8-RPGR
Single, subretinal administration of dose 5 as a single dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

Experimental: Part I: Dose 6 AAV8-RPGR
Single, subretinal administration of dose 6 as a single dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

Experimental: Part II: High dose AAV8-RPGR
Single, subretinal administration of high dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

Experimental: Part II: Low dose AAV8-RPGR
Single, subretinal administration of Low dose AAV8-RPGR
Biological: AAV8-RPGR
Comparison of different dosages of AAV8-RPGR

No Intervention: Part II: Untreated Group
Untreated group to allow for a controlled comparison of efficacy and safety



Primary Outcome Measures :
  1. Part I: Incidence of dose limiting toxicities (DLTs) [ Time Frame: 24 months ]
    Proportion of patients with a DLT

  2. Part I: Incidence of treatment emergent adverse events (TEAEs) [ Time Frame: 24 months ]
    Proportion of patients with a TEAE

  3. Part II: Improvement from Baseline in microperimetry [ Time Frame: 12 Months ]
    Proportion of patients with improved microperimetry

  4. Part II: The safety endpoint is incidence of TEAEs over a 12-month period [ Time Frame: 12 Months ]
    Proportion of patients with a TEAE


Secondary Outcome Measures :
  1. Best corrected visual acuity (BCVA) [ Time Frame: 24 Months (Part I),12 Months (Part II) ]
    ETDRS visual acuity chart

  2. Microperimetry [ Time Frame: 24 Months (Part I),12 Months (Part II) ]
    Change in sensitivity (dB)

  3. Spectral domain optical coherence tomography (SD-OCT) [ Time Frame: 24 Months (Part I),12 Months (Part II) ]
    Ellipsoid Zone

  4. Fundus autofluorescence (AF) [ Time Frame: 24 Months (Part I),12 Months (Part II) ]
    To assess changes in the retina from baseline in autofluorescence imaging

  5. Visual Fields [ Time Frame: 12 Months (Part II only) ]
    Octopus 900 pro will be used to assess changes in central peripheral vison from baseline



Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key inclusion Criteria:

  • Male
  • Part I: >= 18 years
  • Part II: >= 10 years
  • Documentation of a mutation in the RPGR gene

Key exclusion Criteria:

  • participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03116113


Contacts
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Contact: Nightstarx Limited +1 866-633-4636 researchenquiries@nightstartx.com

Locations
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United States, Florida
Study Site Not yet recruiting
Gainesville, Florida, United States, 32607
Study Site Recruiting
Miami, Florida, United States, 33136
United States, Oregon
Study Site Not yet recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Study Site Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
United Kingdom
Study Site Recruiting
Manchester, United Kingdom
Study Site Recruiting
Oxford, United Kingdom
Study Site Recruiting
Southampton, United Kingdom
Sponsors and Collaborators
NightstaRx Ltd

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Responsible Party: NightstaRx Ltd
ClinicalTrials.gov Identifier: NCT03116113     History of Changes
Other Study ID Numbers: NSR-RPGR-01
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: August 22, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by NightstaRx Ltd:
XLRP
Ophthalmology
Gene Therapy
Retinitis Pigmentosa GTPase Regulator (RPGR)
AAV8
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn