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Completed

NCT Number: NCT03252847

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

Phase 1 of the study is a dose escalation of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 1 were assigned to a dose group based on when they enrolled (i.e., sequential assignment).

Phase 2 of the study is a cohort expansion of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 2 were randomized to immediate or deferred treatment.

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Key information

Conditions

X-Linked Retinitis Pigmentosa

Age range

5 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom

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About this study

This is an open-label phase 1/2 dose-escalation and cohort expansion trial to determine the safety and efficacy of subretinal administration of AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key inclusion Criteria:

  • Males aged 5 years or older
  • 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

Treatment and study plan

AAV5-RPGR

Genetic

Single, subretinal administration of AAV5-RPGR

Primary outcomes

  1. Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Product (ATIMP), Not Surgery Alone.

    Time frame: 9 weeks

    The primary outcome is defined as any of the below occurring during the 9 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Product (ATIMP), not surgery alone:

    • Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more
    • Severe unresponsive inflammation
    • Infective endophthalmitis
    • Ocular malignancy
    • Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)

Secondary outcomes

  1. Improvements in Visual Function as Assessed by Visual Acuity

    Time frame: Baseline and Month 6

    Change from baseline to Week 26 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.

  2. Improvements in Retinal Function as Assessed by Static Perimetry

    Time frame: Baseline and Month 6

    The number of responders in point-by-point data in Static Perimetry within the full visual field over time.

    A responder at a single time point is defined as a participant with at least 5 of the same loci with ≥7 dB improvement from baseline at the specific time point and one time point prior.

  3. Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Emotional Distress Domain Score

    Time frame: Baseline and Month 6

    Change from baseline to Week 26 in LLQ Emotional Distress Domain Score in adults. LLQ uses a scale from 0 to 100, with higher scores reflecting less impairment. A positive change from baseline reflects improvement, and a negative reflects worsening.

  4. Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Extreme Lighting Domain Score

    Time frame: Baseline and Month 6

    Change from baseline to Week 26 in LLQ Extreme Lighting Domain Score in adults. LLQ uses a scale from 0 to 100, with higher scores reflecting less impairment. A positive change from baseline reflects improvement, and a negative reflects worsening.

  5. Quality of Life Measured by the Low Luminance Questionnaire (LLQ) General Dim Lighting Domain Score

    Time frame: Baseline and Month 6

    Change from baseline to Week 26 in LLQ General Dim Lighting Domain score in adults. LLQ uses a scale from 0 to 100, with higher scores reflecting less impairment. A positive change from baseline reflects improvement, and a negative reflects worsening.

  6. Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Mobility Domain Score

    Time frame: Baseline and Month 6

    Change from baseline to Week 26 in LLQ Mobility Domain Score in adults. LLQ uses a scale from 0 to 100, with higher scores reflecting less impairment. A positive change from baseline reflects improvement, and a negative reflects worsening.

  7. Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Peripheral Vision Domain Score

    Time frame: Baseline and Month 6

    Change from baseline to Week 26 in LLQ Peripheral Vision Domain Score in adults. LLQ uses a scale from 0 to 100, with higher scores reflecting less impairment. A positive change from baseline reflects improvement, and a negative reflects worsening.

Sponsors and collaborators

Lead sponsor

MeiraGTx UK II Ltd

Industry

Collaborators

  • Bionical Emas
  • Syne Qua Non Limited

Registry information

Official study title

An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/5-hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

Important dates

Study start
2017
Primary completion
2021
Study completion
2021
First posted
Aug 17, 2017
Registry last updated
Jan 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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