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

Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - MGT-RPGR-021 gene therapy study in patients with XLRP

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON52296

Enrollment

Registered

2021-10-18

Start date

2022-07-01

Completion date

Unknown

Last updated

2025-10-27

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.Male or female. 2.3 years of age or older. 3.Has X-linked retinitis pigmentosa (generalized rod-cone dystrophy) confirmed by a retinal specialist AND has a predicted disease-causing sequence variant in RPGR confirmed by a sponsor-approved laboratory. Please refer to the protocol for additional inclusion criteria

Exclusion criteria

Exclusion criteria: 1.Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration. 2.Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule. 3.Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery or YAG capsulotomy). 4.History of an ocular implant, with the exception of an intraocular lens. 'Please refer to the protocol for additional exclusion criteria'

Design outcomes

Primary

Measure	Time frame
Change from baseline to week 52 in binocular VMA	—

Secondary

Measure	Time frame
Retinal Function assessed by - Changes from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 - Changes from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 - Pointwise response in full visual field at Week 52 - Pointwise response in worse-seeing eye in full visual field at Week 52 - Pointwise response in the central 30 degrees visual field at Week 52 - Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 - Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma Functional Vision assessed by - Vision-guided mobility assessment response in the "worse-seeing eye" as assessed by VMA at Week 52 - Change from baseline in the modified Low Luminance Questionnaire (mLLQ) Extreme lighting domain score at Week 52 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 at Week 52 - Change from baseline in best corrected visual acuity (BCVA) by ETDRS chart letter score in monocular assessment at Week 52 - Change from baseline in low luminance visual acuity by ETDRS chart letter score in worseseeing eye at Week 52 Adverse Events Laboratory assessments Please refer to the clinical trial protocol for the full list of secondary end points.	—

Measure

Time frame

Retinal Function assessed by - Changes from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 - Changes from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 - Pointwise response in full visual field at Week 52 - Pointwise response in worse-seeing eye in full visual field at Week 52 - Pointwise response in the central 30 degrees visual field at Week 52 - Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 - Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma Functional Vision assessed by - Vision-guided mobility assessment response in the "worse-seeing eye" as assessed by VMA at Week 52 - Change from baseline in the modified Low Luminance Questionnaire (mLLQ) Extreme lighting domain score at Week 52 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 at Week 52 - Change from baseline in best corrected visual acuity (BCVA) by ETDRS chart letter score in monocular assessment at Week 52 - Change from baseline in low luminance visual acuity by ETDRS chart letter score in worseseeing eye at Week 52 Adverse Events Laboratory assessments Please refer to the clinical trial protocol for the full list of secondary end points.

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)