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Trial record 35 of 140 for:    "Retinitis pigmentosa"

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 : April 14, 2017
Sponsor:
Information provided by (Responsible Party):
Nightstar Therapeutics

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

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Biological: AAV-RPGR Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Dose Escalation, Phase 1/2 Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)
Actual Study Start Date : March 16, 2017
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : February 2019


Arm Intervention/treatment
Experimental: Low dose AAV-RPGR
Single, subretinal administration of a single low dose range AAV-RPGR
Biological: AAV-RPGR
Comparison of different dosages of AAV-RPGR

Experimental: Medium dose AAV-RPGR
Single, subretinal administration of a single medium dose range AAV-RPGR
Biological: AAV-RPGR
Comparison of different dosages of AAV-RPGR

Experimental: High dose AAV-RPGR
Single, subretinal administration of a single high dose range AAV-RPGR
Biological: AAV-RPGR
Comparison of different dosages of AAV-RPGR




Primary Outcome Measures :
  1. Incidence of dose limiting toxicities (DLTs) [ Time Frame: 12 months ]
    Proportion of patients with a DLT

  2. Incidence of treatment emergent adverse events (TEAEs) [ Time Frame: 12 months ]
    Proportion of patients with a TEAE


Secondary Outcome Measures :
  1. Change in best corrected visual acuity (BCVA) from baseline to Month 12 [ Time Frame: 12 months ]
    BCVA will be assessed at each visit using the Early Treatment Diabetic Retinopathy Study (EDTRS) protocol. Missing post-baseline values will be imputed using last observation carried forward (LOCF) method.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key inclusion Criteria:

  • male
  • >= 18 years of age
  • have a genetically confirmed diagnosis of X-linked retinitis pigmentosa

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
Contact: NightstaRx +44 207 611 2077 enquiries@nightstarx.com

Locations
United Kingdom
Manchester Royal Eye Hospital Recruiting
Manchester, United Kingdom
Oxford Eye Hospital Recruiting
Oxford, United Kingdom
Sponsors and Collaborators
Nightstar Therapeutics
Investigators
Study Director: Chief Medical Officer Nightstar Therapeutics

Responsible Party: Nightstar Therapeutics
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: April 14, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Cone-Rod Dystrophies
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn