X-Linked Retinitis PigmentosaNCT04312672
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
An observational study of X-Linked Retinitis Pigmentosa, run by MeiraGTx Ocular UK Ltd at 5 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- X-Linked Retinitis Pigmentosa
- What is being tested?
- AAV5-hRKp.RPGR
- How many people
- 42 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 31 Jul 2017to 17 Sep 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Males receiving AAV5-hRKp.RPGR in the MGT009 study
Who may be able to join
- Received AAV5-hRKp.RPGR in the MGT009 Study
Who cannot join
- Unwilling or unable to meet with the requirements of the study
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
United States3 sites
Univ of Michigan Medical Center
Ann Arbor, Michigan, 48105
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
United Kingdom2 sites
Leeds Teaching Hospitals NHS Trust
Leeds, LS9 7TF
Moorfields Eye Hospital
London, EC1V 9EL
About this study
Official 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)
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.
- Conditions
- X-Linked Retinitis Pigmentosa
- Being tested
- AAV5-hRKp.RPGR
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV5-hRKp.RPGRBiological
Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.
| Study group | Role | What they receive |
|---|---|---|
| Follow up cohort | no intervention follow up study Receives: Biological: AAV5-hRKp.RPGR |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants
Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported.
Measured: 60 Months
Secondary outcomes (8)
Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)
Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported.
Measured: Up to 60 Months
Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)
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.
Measured: Up to 60 Months
Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score
The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported.
Measured: Up to 60 Months
Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions
Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported.
Measured: Up to 60 Months
Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry
Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported.
Measured: Up to 60 months
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field
Change in retinal sensitivity by pointwise comparison of data in static perimetry within the full visual field will be reported.
Measured: Up to 60 months
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field
Change in retinal sensitivity by pointwise comparison of data in static perimetry within the Central 30 Degrees visual field will be reported.
Measured: Up to 60 months
Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry
Change in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be reported.
Measured: Up to 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2020
- Study start31 Jul 2017
- Primary completion17 Sep 2025
- Study completion17 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- MeiraGTx Ocular UK Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, MeiraGTx Ocular UK Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04312672
- Sponsor's protocol ID
- MGT010
- Other IDs
- MGT010, 2018-000425-31, 2018
- Status last verified
- Sep 2026
- First submitted
- 15 Jul 2019
- Last updated
- 25 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT04312672
- Acronym / sponsor ID
- MGT010
- Conditions
- X-Linked Retinitis Pigmentosa
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- MeiraGTx Ocular UK LtdIndustry (pharma, biotech, devices)
- Study start
- 31 Jul 2017 (actual)
- Primary completion
- 17 Sep 2025 (actual)
- Study completion
- 17 Sep 2025 (actual)
- First posted
- 18 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Follow up cohort | – | Biological: AAV5-hRKp.RPGRno intervention follow up study |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Males receiving AAV5-hRKp.RPGR in the MGT009 study
Key inclusion criteria (1 listed)
- Received AAV5-hRKp.RPGR in the MGT009 Study
Key exclusion criteria (1 listed)
- Unwilling or unable to meet with the requirements of the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV5-hRKp.RPGRParticipants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up. | Biological | Follow up cohort |
Primary outcome
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.
8 secondary outcomes
Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)
Time frame: Up to 60 Months
Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)
Time frame: Up to 60 Months
Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score
Time frame: Up to 60 Months
Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions
Time frame: Up to 60 Months
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 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 Central 30 Degrees Visual Field
Time frame: Up to 60 months
Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry
Time frame: Up to 60 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
MeiraGTx Ocular UK Ltd
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of X-Linked Retinitis Pigmentosa, run by MeiraGTx Ocular UK Ltd at 5 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants (time frame: 60 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2026; AzTrials last refreshed its copy on 6 Nov 2026.