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Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)

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ClinicalTrials.gov Identifier: NCT03252847
Recruitment Status : Recruiting
First Posted : August 17, 2017
Last Update Posted : July 17, 2019
Sponsor:
Collaborators:
Syne Qua Non Limited
Bionical-Emas Pharma Ltd
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Tracking Information
First Submitted Date  ICMJE August 1, 2017
First Posted Date  ICMJE August 17, 2017
Last Update Posted Date July 17, 2019
Actual Study Start Date  ICMJE July 14, 2017
Estimated Primary Completion Date November 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 16, 2017)
Incidence of Adverse Events related to the sub retinal administration of AAV2-RPGR [ Time Frame: 18 months ]
Safety is defined as the absence of ATIMP-related safety events
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03252847 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 16, 2017)
  • Improvement in visual function [ Time Frame: 18 months ]
    Improvements in visual function as assessed by ocular examination
  • Improvement in retinal function [ Time Frame: 18 months ]
    Improvements in retinal function as assessed by retinal assessement
  • Improvement in Quality of Life [ Time Frame: 18 months ]
    •Quality of life will be measured by QoL questionnaire
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)
Official Title  ICMJE An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2-.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Brief Summary A clinical trial of AAV2 vector for patients with X-linked retinitis pigmentosa (XLRP)
Detailed Description This is an open-label phase I/II dose-escalation trial to determine the safety and efficacy of subretinal administration of AAV2 vector in participants with XLRP caused by mutations in RPGR.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description:
Biological
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE X-Linked Retinitis Pigmentosa
Intervention  ICMJE Genetic: AAV-RPGR
Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm
Study Arms  ICMJE
  • Experimental: Low dose AAV-RPGR
    Single, subretinal administration of low dose AAV-RPGR
    Intervention: Genetic: AAV-RPGR
  • Experimental: Intermediate dose AAV-RPGR
    Single, subretinal administration of intermediate dose AAV-RPGR
    Intervention: Genetic: AAV-RPGR
  • Experimental: High dose AAV-RPGR
    Single, subretinal administration of high dose AAV-RPGR
    Intervention: Genetic: AAV-RPGR
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 16, 2017)
36
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2020
Estimated Primary Completion Date November 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key inclusion Criteria:

  • Are aged 5 years or older male
  • Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)

Key exclusion Criteria:

• Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: MeiraGTx UK II Ltd +44 (0)20 3866 4320 ocularinfo@meiragtx.com
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03252847
Other Study ID Numbers  ICMJE MGT009
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party MeiraGTx UK II Ltd
Study Sponsor  ICMJE MeiraGTx UK II Ltd
Collaborators  ICMJE
  • Syne Qua Non Limited
  • Bionical-Emas Pharma Ltd
Investigators  ICMJE
Principal Investigator: James Bainbridge, Prof University College, London
PRS Account MeiraGTx UK II Ltd
Verification Date February 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP