X-Linked Retinitis Pigmentosa (XLRP)Phase 2LandscapeNCT07174726
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
A Phase 2 study testing Adeno-associated virus vector expressing a human RPGR gene in people with X-Linked Retinitis Pigmentosa (XLRP), run by Beacon Therapeutics at 6 sites in United States.
Trial summary
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP. Read more about this study
- Who is it for?
- Ages 12 to 50 years, men only
- Conditions the study is for
- X-Linked Retinitis Pigmentosa (XLRP)
- What is being tested?
- Adeno-associated virus vector expressing a human RPGR gene
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 6 sitesUnited States
- Study dates
- 10 Sep 2025to 30 Dec 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 12 to 50 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- General inclusion criteria
- Provide written informed consent or assent (per local regulation) prior to the conduct of any study-related procedures. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent.
- Be between 12 and 50 years of age (inclusive) at the time of providing informed consent and assent (as applicable)
- Be male (XY chromosome) and have at least 1 documented pathogenic or likely pathogenic variant in the RPGR gene, within exons 1-14 and/or ORF15, from an appropriately certified or accredited laboratory
- Have a clinical diagnosis of XLRP
- Be in good general health to withstand subretinal surgery and perioperative medications based on a complete physical examination and results from hematology, chemistry, and coagulation analyses performed at screening
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- Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend all study visits
- If the participant has a parent or caregiver, the parent or caregiver must be able to follow study instructions, comply with the protocol, and attend study visits with the participant, as required
- Ocular inclusion criteria (both eyes)
- Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200) in each eye based on an ETDRS chart at each screening visit. The ETDRS letter score is the main visual acuity inclusion criterion for participants. Participants unable to read the ETDRS letters may use a tumbling "E" chart for the BCVA assessments
- Have an LLVA ≤ 64 letters (approximately Snellen, 20/50) in both eyes based on an ETDRS chart at each screening visit. Participants unable to read the ETDRS letters may use a tumbling "E" chart for the LLVA assessments
- Have an LLD of >10 letters
- Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability and fixation, per the Investigator's discretion
- Have a detectable mean macular sensitivity by MAIA microperimetry at baseline between 1 and 12 dB in both eyes, as determined by the Investigator and confirmed by the central reading center (CRC), with a fixation loss ≤ 20% at each screening visit
- Have a detectable sub-foveal EZ line as assessed by SD-OCT in both eyes as confirmed by the CRC
Who cannot join
- General Exclusion Criteria
- Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel that, in the opinion of the Investigator, would interfere with the potential therapeutic effect of the study drug or the quality of the assessments
- For participants with herpes simplex virus (HSV):
- Have a history of oral or genital herpes and be unable and/or unwilling to use a prophylactic antiviral medication.
- Have a history of ocular herpes.
- Have active oral or genital herpes or are currently receiving treatment for active HSV infection
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- Have complicating systemic diseases (e.g., medical conditions causing immunosuppression, autoimmunity, active systemic infection) that would preclude the gene transfer or ocular surgery if not adequately managed or treated
- Have a known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications
- Have used anticoagulant agents that may alter coagulation (e.g., warfarin, heparin, apixaban, or high-dose docosahexaenoic acid [fish oil]) within 7 days prior to study drug administration (ibuprofen, aspirin, or similar agents are acceptable
- Have received any vaccination/immunization within 60 days prior to Day 1 and/or during screening with the exception of the influenza vaccine, which is only exclusionary if received within 28 days prior to Day 1
- Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to study drug administration. Corticosteroids used on an as-needed basis administered by insufflation, inhalation, or local administration to the skin and mucosa such as Symbicort® (budesonide/formoterol), Flonase® (fluticasone propionate), and skin creams and ointments containing corticosteroids shall not be exclusionary
- If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following study drug administration
- Have any other condition or reason that, in the opinion of the Investigator, would prevent the participant from completing all study assessments
- Have any other condition or reason that, in the opinion of the Investigator, makes the participant unsuitable for the study
- Are currently participating or recently participated in any other research protocol involving investigational agents or therapies that, in the opinion of the Investigator, would make the participant unsuitable for the study. Recent participation is defined as participation within 90 days of initial screening for this study OR within 10 half-lives of the investigational drug, whichever is longer
- Have previously received any adeno-associated virus (AAV) gene therapy product, stem cell therapy, cell-based therapy, or similar biologics
- Ocular exclusion criteria (either eye)
- Have pre-existing eye conditions in either eye that would preclude the planned surgery, interfere with the interpretation of study endpoints, or increase the risk of surgical complications (e.g., corneal opacities, diabetic retinopathy, retinal vasculitis, glaucoma, active cystoid macular edema)
- Have significant media opacity impacting evaluation of the retina or vitreous in either eye. This includes cataracts considered to be a major contributor to reducing visual acuity and/or if the participant is likely to require cataract extraction within 3 months of study drug administration
- Had intraocular surgery in either eye within the 90 days prior to the planned administration of study drug
- Have any active ocular/intraocular infection or inflammation (e.g., severe blepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis, idiopathic or autoimmune-associated uveitis, or herpetic lesions) in either eye
- Have a history of corticosteroid-induced raised intraocular pressure (IOP) of > 25 mmHg following corticosteroid exposure in either eye, despite topical IOP-lowering pharmacologic therapy
- Have any artificial retinal implant or prosthesis in either eye
- Have an absence of clear ocular media and/or inadequate pupil dilation to facilitate good quality SD-OCT images in either eye.
- Have any history of rhegmatogenous retinal detachment in either eye
- Have myopia (spherical equivalent) exceeding -10 diopters Investigator or the presence of pathologic myopia in either eye.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Cincinnati Eye Institute
Cincinnati, Ohio, 45242
Retina Foundation of the Southwest
Dallas, Texas, 75231
Duke Eye Center
Durham, North Carolina, 27710
University of Florida Jacksonville Ophthalmology
Jacksonville, Florida, 32209
Bascom Palmer Eye Institute
Miami, Florida, 33136
OHSU Casey Eye Institute
Portland, Oregon, 97239
About this study
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
Read the detailed description
XLRP is a genetic (inherited) eye disease that affects cells in the retina (the lining of the back of the eye that detects light). It causes night blindness and gradual worsening of your vision.
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
- Conditions
- X-Linked Retinitis Pigmentosa (XLRP)
- Being tested
- Adeno-associated virus vector expressing a human RPGR gene
- Keywords
- XLRP, retinal degeneration, RPGR, adeno-associated virus, AAV, gene therapy
- Medical subject headings
- Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adeno-associated virus vector expressing a human RPGR geneBiological
Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501
| Study group | Role | What they receive |
|---|---|---|
| Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes | Experimental | On Day 1, participants will undergo a pars plana vitrectomy and receive a central subretinal administration of laruparetigene zovaparvovec in their first treated study eye.
For the first six participants, participants will undergo a pars plana vitrectomy followed by a central subretinal administration of laruparetigene zovaparvovec in their second eye between 30 and 60 days after the first surgery.
Once the first six study participants have been dosed in both eyes, the interval between dosing of the first and second eye may be reduced to a minimum of 7 days (and up to 14 days) for up to four of the remaining participants enrolled in the study, provided the following conditions are met:
* No unexpected safety concerns were observed following dosing of the first eye.
* An independent safety review by the Sponsor, Investigator, and DSMC Chair confirms that bilateral dosing may proceed. Receives: Biological: Adeno-associated virus vector expressing a human RPGR gene |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number and percentage of participants experiencing Grade 3 or higher ocular or non-ocular treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (SAEs), at Month 12
Measured: Day 0 - Month 12
Secondary outcomes (11)
The number and percentage of participants experiencing ocular or non-ocular TEAEs, including treatment-emergent SAEs during the Study period
Measured: Day 0-5 years
Change from baseline in low-luminance visual acuity (LLVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity at Month 12
Measured: Day 0 - Month 12
Change from baseline in mean sensitivity across the whole grid, as measured by macular integrity assessment (MAIA) microperimetry at Month 12
Measured: Day 0 - Month 12
Response, as measured by MAIA microperimetry, where response is defined as a ≥ 7 dB visual sensitivity improvement from baseline in at least 5 loci at Month 12
Measured: Day 0 - Month 12
Change from baseline in full-field stimulus threshold (FST) at Month 12
Measured: Day 0 - Month 12
Change from baseline in best-corrected visual acuity (BCVA) using ETDRS visual acuity at Month 12
Measured: Day 0 - Month 12
Change from baseline in low-luminance deficit using ETDRS visual acuity at Month 12
Measured: Day 0 - Month 12
Proportion of responding eyes at Month 12 where responder is defined as an improvement of at least 15 letters on LLVA
Measured: Day 0 - Month 12
Change from baseline in ellipsoid zone (EZ) area measured by spectral domain-optical coherence tomography (SD-OCT) at Month 12
Measured: Day 0 - Month 12
Change from baseline in 7 domain scores from a Michigan Retinal Degeneration Questionnaire (MRDQ) at Month 12
Measured: Day 0 - Month 12
Change from baseline in the Mobility Standardized Test-Virtual Reality (MOST-VR) course test score at Month 12
Measured: Day 0- Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2025
- Study start10 Sep 2025
- Primary completion31 Dec 2029 (estimated)
- Study completion30 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked Retinitis Pigmentosa (XLRP)Studies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- To evaluate the long-term safety and tolerability of laruparetigene zovaparvovec administered bilaterally via subretinal injection
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beacon Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- None None, Principal investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07174726
- Sponsor's protocol ID
- RPGR-005 Landscape
- Acronym
- Landscape
- Status last verified
- Jun 2026
- First submitted
- 5 Sep 2025
- Last updated
- 25 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT07174726
- Acronym / sponsor ID
- Landscape / RPGR-005 Landscape
- Conditions
- X-Linked Retinitis Pigmentosa (XLRP)
- Keywords
- XLRP, retinal degeneration, RPGR, adeno-associated virus, AAV, gene therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Beacon TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 10 Sep 2025 (actual)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 30 Dec 2030 (estimated)
- First posted
- 16 Sep 2025 (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 purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
Detailed description
XLRP is a genetic (inherited) eye disease that affects cells in the retina (the lining of the back of the eye that detects light). It causes night blindness and gradual worsening of your vision.
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- To evaluate the long-term safety and tolerability of laruparetigene zovaparvovec administered bilaterally via subretinal injection
| Arm | Type | Receives |
|---|---|---|
| Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes | Experimental | Biological: Adeno-associated virus vector expressing a human RPGR geneOn Day 1, participants will undergo a pars plana vitrectomy and receive a central subretinal administration of laruparetigene zovaparvovec in their first treated study eye. For the first six participants, participants will undergo a pars plana vitrectomy followed by a central subretinal administration of laruparetigene zovaparvovec in their second eye between 30 and 60 days after the first surgery. Once the first six study participants have been dosed in both eyes, the interval between dosing of the first and second eye may be reduced to a minimum of 7 days (and up to 14 days) for up to four of the remaining participants enrolled in the study, provided the following conditions are met: * No unexpected safety concerns were observed following dosing of the first eye. * An independent safety review by the Sponsor, Investigator, and DSMC Chair confirms that bilateral dosing may proceed. |
Who may qualify
- Ages
- 12 to 50 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- General inclusion criteria
- Provide written informed consent or assent (per local regulation) prior to the conduct of any study-related procedures. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent.
- Be between 12 and 50 years of age (inclusive) at the time of providing informed consent and assent (as applicable)
- Be male (XY chromosome) and have at least 1 documented pathogenic or likely pathogenic variant in the RPGR gene, within exons 1-14 and/or ORF15, from an appropriately certified or accredited laboratory
- Have a clinical diagnosis of XLRP
Key exclusion criteria (26 listed)
- General Exclusion Criteria
- Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel that, in the opinion of the Investigator, would interfere with the potential therapeutic effect of the study drug or the quality of the assessments
- For participants with herpes simplex virus (HSV):
- Have complicating systemic diseases (e.g., medical conditions causing immunosuppression, autoimmunity, active systemic infection) that would preclude the gene transfer or ocular surgery if not adequately managed or treated
- Have a known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications
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 |
|---|---|---|
| Adeno-associated virus vector expressing a human RPGR geneMale participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501 | Biological | Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes |
Primary outcome
The number and percentage of participants experiencing Grade 3 or higher ocular or non-ocular treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (SAEs), at Month 12
Time frame: Day 0 - Month 12
11 secondary outcomes
The number and percentage of participants experiencing ocular or non-ocular TEAEs, including treatment-emergent SAEs during the Study period
Time frame: Day 0-5 years
Change from baseline in low-luminance visual acuity (LLVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity at Month 12
Time frame: Day 0 - Month 12
Change from baseline in mean sensitivity across the whole grid, as measured by macular integrity assessment (MAIA) microperimetry at Month 12
Time frame: Day 0 - Month 12
Response, as measured by MAIA microperimetry, where response is defined as a ≥ 7 dB visual sensitivity improvement from baseline in at least 5 loci at Month 12
Time frame: Day 0 - Month 12
Change from baseline in full-field stimulus threshold (FST) at Month 12
Time frame: Day 0 - Month 12
Change from baseline in best-corrected visual acuity (BCVA) using ETDRS visual acuity at Month 12
Time frame: Day 0 - Month 12
Change from baseline in low-luminance deficit using ETDRS visual acuity at Month 12
Time frame: Day 0 - Month 12
Proportion of responding eyes at Month 12 where responder is defined as an improvement of at least 15 letters on LLVA
Time frame: Day 0 - Month 12
Change from baseline in ellipsoid zone (EZ) area measured by spectral domain-optical coherence tomography (SD-OCT) at Month 12
Time frame: Day 0 - Month 12
Change from baseline in 7 domain scores from a Michigan Retinal Degeneration Questionnaire (MRDQ) at Month 12
Time frame: Day 0 - Month 12
Change from baseline in the Mobility Standardized Test-Virtual Reality (MOST-VR) course test score at Month 12
Time frame: Day 0- Month 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2026.
- Individual participant dataSharing planned: No
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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.
None None principal investigator
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Adeno-associated virus vector expressing a human RPGR gene in people with X-Linked Retinitis Pigmentosa (XLRP), run by Beacon Therapeutics at 6 sites in United States.
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 15 inclusion and 26 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: under way, not looking for participants, as of the sponsor's last update on 25 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
The number and percentage of participants experiencing Grade 3 or higher ocular or non-ocular treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (SAEs), at Month 12 (time frame: Day 0 - Month 12). 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 Jun 2026.