Retinitis PigmentosaNCT03349242
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
An observational study of Retinitis Pigmentosa, run by MeiraGTx UK II Ltd at 8 sites in 3 countries.
Trial summary
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading to severe visual impairment. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- How many people
- 140 participants
- Where
- 8 sitesin 3 countries
- Study dates
- 19 Dec 2017to 19 Apr 2024
- 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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with RPGR-associated retinal dystrophy
Who may be able to join
- Males \& Females aged 5 years or older
- Have RPGR-associated retinal dystrophy
- Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate
- Are able to undertake age-appropriate clinical assessments as specified in the protocol
- Have genetic mutation within the RPGR gene confirmed by an accredited lab or research lab.
Who cannot join
- Are unable or unwilling to undertake consent or clinical testing
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
United States6 sites
Kellogg Eye Center
Ann Arbor, Michigan, 48105
Emory Eye Centre
Atlanta, Georgia, 30322
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114
Shiley Eye Institute - UCSD
La Jolla, California, 92093
UPMC Eye Centre
Pittsburgh, Pennsylvania, 15213
Stanford University, Spencer Center for Vision Research
Stanford, California, 94303
About this study
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading to severe visual impairment. RP usually occurs as an isolated retinal disorder, but it may also be seen in association with systemic abnormalities.
Read the detailed description
X-linked Retinitis Pigmentosa (XLRP) is a severe form of RP with early onset of nyctalopia and progression to legal blindness by the 3rd to 4th decade. Most affected males show symptomatic night blindness before the age of 10 years, are often myopic and show fundus abnormalities and ERG changes in early childhood. Examination of close female relatives is helpful in the absence of a family history, as the recognition of the XL carrier state will confirm the diagnosis.
- Conditions
- Retinitis Pigmentosa
- Medical subject headings
- Retinitis Pigmentosa
What is being measured
The primary outcome is the main question the study is designed to answer.
Analysis of retinal structure and function to assess disease progression
Retinal structure will be measured using Adaptive optics and SD-OCT and Fundal autofluorescence.
Measured: 6 years
Secondary outcomes (4)
Retinal Sensitivity
To be assessed by Microperimetry
Measured: 6 years
Retinal Structural detailed phenotyping
Retinal Structure measured by Adaptive Optics (
Measured: 6 years
Fundus Autofluorescence
Presence or Absence
Measured: 6 years
Visual Fields testing
Assessment of Visual Fields with analysis of hill vision by perimetry
Measured: 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2017
- Study start19 Dec 2017
- Primary completion19 Apr 2024
- Study completion19 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Other
- Time perspective
- Prospective
- Enrolment
- 140 participants (actual)
Sponsor and investigators
- Lead sponsor
- MeiraGTx UK II Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Janssen, LP
- Responsible party
- Sponsor
- Investigators
- Michel Michaelides, Prof, Principal investigator, UCL/Moorfields
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03349242
- Sponsor's protocol ID
- MGT011
- Status last verified
- Jun 2024
- First submitted
- 13 Nov 2017
- Last updated
- 20 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 20 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
- ClinicalTrials.gov ID
- NCT03349242
- Acronym / sponsor ID
- MGT011
- Conditions
- Retinitis Pigmentosa
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 140 participants (actual)
- Lead sponsor
- MeiraGTx UK II LtdIndustry (pharma, biotech, devices)
- Collaborators
- Janssen, LP
- Study start
- 19 Dec 2017 (actual)
- Primary completion
- 19 Apr 2024 (actual)
- Study completion
- 19 Apr 2024 (actual)
- First posted
- 21 Nov 2017 (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.
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading to severe visual impairment. RP usually occurs as an isolated retinal disorder, but it may also be seen in association with systemic abnormalities.
Detailed description
X-linked Retinitis Pigmentosa (XLRP) is a severe form of RP with early onset of nyctalopia and progression to legal blindness by the 3rd to 4th decade. Most affected males show symptomatic night blindness before the age of 10 years, are often myopic and show fundus abnormalities and ERG changes in early childhood. Examination of close female relatives is helpful in the absence of a family history, as the recognition of the XL carrier state will confirm the diagnosis.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with RPGR-associated retinal dystrophy
Key inclusion criteria (5 listed)
- Males \& Females aged 5 years or older
- Have RPGR-associated retinal dystrophy
- Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate
- Are able to undertake age-appropriate clinical assessments as specified in the protocol
- Have genetic mutation within the RPGR gene confirmed by an accredited lab or research lab.
Key exclusion criteria (1 listed)
- Are unable or unwilling to undertake consent or clinical testing
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Analysis of retinal structure and function to assess disease progression
Time frame: 6 years
Retinal structure will be measured using Adaptive optics and SD-OCT and Fundal autofluorescence.
4 secondary outcomes
Retinal Sensitivity
Time frame: 6 years
Retinal Structural detailed phenotyping
Time frame: 6 years
Fundus Autofluorescence
Time frame: 6 years
Visual Fields testing
Time frame: 6 years
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 20 Jun 2024.
- Individual participant dataSharing planned: Undecided
Related trials for Retinitis Pigmentosa
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Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment
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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.
Michel Michaelides, Prof principal investigator
UCL/Moorfields
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Retinitis Pigmentosa, run by MeiraGTx UK II Ltd at 8 sites in 3 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 5 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 20 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Canada, 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?
Analysis of retinal structure and function to assess disease progression (time frame: 6 years). 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 20 Jun 2024; AzTrials last refreshed its copy on 7 Nov 2026.