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OpenTrials
Completed

NCT Number: NCT04312672

Long-term Follow-up Gene Therapy Study for RPGR- XLRP

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

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Key information

Conditions

X-Linked Retinitis Pigmentosa

Age range

5 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Received AAV5-hRKp.RPGR in the MGT009 Study

Exclusion criteria

  • Unwilling or unable to meet with the requirements of the study

Treatment and study plan

AAV5-hRKp.RPGR

Biological

Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.

Primary outcomes

  1. Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants

    Time frame: 60 Months

    Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported.

Secondary outcomes

  1. Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)

    Time frame: Up to 60 Months

    Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported.

  2. Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)

    Time frame: Up to 60 Months

    Change in functional vision of LLQ domain scores in PRO will be reported. The instrument is scored by domain, computed by scaling individual items from 0 to 100 and then averaging the individual items for each domain. A higher score reflects higher functional level.

  3. Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score

    Time frame: Up to 60 Months

    The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported.

  4. Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions

    Time frame: Up to 60 Months

    Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported.

  5. Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry

    Time frame: Up to 60 months

    Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported.

  6. Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field

    Time frame: Up to 60 months

    Change in retinal sensitivity by pointwise comparison of data in static perimetry within the full visual field will be reported.

  7. Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field

    Time frame: Up to 60 months

    Change in retinal sensitivity by pointwise comparison of data in static perimetry within the Central 30 Degrees visual field will be reported.

  8. Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry

    Time frame: Up to 60 months

    Change in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be reported.

Sponsors and collaborators

Lead sponsor

Janssen Research & Development, LLC

Industry

Registry information

Official study title

Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- 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
2025
Study completion
2025
First posted
Mar 18, 2020
Registry last updated
Jan 21, 2026

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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