Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene

Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - MGT-RPGR-022 Gene therapy follow-up study in patients with XLRP

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON51971

Enrollment

Registered

2021-10-18

Start date

2022-02-28

Completion date

Unknown

Last updated

2024-09-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

genetic eye disease XLRP

Interventions

AAV5-hRKp.RPGR gene therapy is injected in the subretinal space of the treatment eye in the operating room by a retinal surgeon who is trained and qualified to deliver the investigational product. D

Eligibility

Age

2 Years to 64 Years

Inclusion criteria

Inclusion criteria: 1.Previously completed participation in Study MGT-RPGR-021. 2.Must reconfirm that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study.

Exclusion criteria

Exclusion criteria: There are no specific exclusion criteria.

Design outcomes

Primary

Measure	Time frame
Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse Events Laboratory Assessment	—

Secondary

Measure	Time frame
Major secondary efficacy endpoints will include the following: •Retinal Function assessed by - Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry - Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma (MRS10) in static perimetry - Pointwise response in full visual field - Pointwise response in worse-seeing eye in full visual field - Pointwise response in the central 30 degrees visual field - Pointwise response in worse-seeing eye in the central 30 degrees visual field - Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry •Functional Vision assessed by - Vision-guided mobility assessment response in the "worse-seeing eye" as assessed by VMA - Change from baseline in the modified Low Luminance Questionnaire (mLLQ) - Extreme lighting domain score •Visual Function assessed by - Change from baseline in low luminance visual acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in monocular assessment - Change from baseline in best corrected visual acuity (BCVA) by ETDRS chart letter score in monocular assessment - Change from baseline in low luminance visual acuity by ETDRS chart letter score in worse-seeing eye	—

Measure

Time frame

Major secondary efficacy endpoints will include the following: •Retinal Function assessed by - Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry - Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma (MRS10) in static perimetry - Pointwise response in full visual field - Pointwise response in worse-seeing eye in full visual field - Pointwise response in the central 30 degrees visual field - Pointwise response in worse-seeing eye in the central 30 degrees visual field - Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry •Functional Vision assessed by - Vision-guided mobility assessment response in the "worse-seeing eye" as assessed by VMA - Change from baseline in the modified Low Luminance Questionnaire (mLLQ) - Extreme lighting domain score •Visual Function assessed by - Change from baseline in low luminance visual acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in monocular assessment - Change from baseline in best corrected visual acuity (BCVA) by ETDRS chart letter score in monocular assessment - Change from baseline in low luminance visual acuity by ETDRS chart letter score in worse-seeing eye

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)