Not Authorised Therapeutic confirmatory (Phase III) X-linked retinitis pigmentosa (XLRP)

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

EU CTIS ID: 2024-511181-36-00

What this study is testing

Primary objective in Europe is to evaluate the efficacy of two doses of AGTC-501 after a single subretinal administration compared to an untreated control as assessed by macular integrity assessment (MAIA) microperimetry at Month 12

Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent.
Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant’s reliability, and fixation, in the study eye per the Investigator’s discretion.
Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, between 1-12 dB in the study eye, as determined by the Investigator and confirmed by the Central Reading Center (CRC), with fixation loss ≤20% at each screening visit.
Have a detectable sub-foveal ellipsoid zone (EZ) line as assessed by SD-OCT in the study eye and confirmed by the CRC.
If both eyes meet all entry criteria, the study eye is the worse-seeing eye as measured by ETDRS BCVA. If both eyes are eligible and have the same BCVA, the choice of study eye will be at the discretion of the Investigator and/or Surgeon.
Be between 12 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable).

You likely can't join if

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 agent or the quality of the assessments
Are currently participating or recently participated in any other research protocol involving investigational agents or therapies that 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 AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics.
Have pre-existing eye conditions 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 [CME]).
Have significant media opacity impacting evaluation of the retina or vitreous. 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 treatment administration.
Had intraocular surgery within 90 days of study treatment administration (Day 1).

See the full eligibility criteria

Who can join

Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent.
Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant’s reliability, and fixation, in the study eye per the Investigator’s discretion.
Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, between 1-12 dB in the study eye, as determined by the Investigator and confirmed by the Central Reading Center (CRC), with fixation loss ≤20% at each screening visit.
Have a detectable sub-foveal ellipsoid zone (EZ) line as assessed by SD-OCT in the study eye and confirmed by the CRC.
If both eyes meet all entry criteria, the study eye is the worse-seeing eye as measured by ETDRS BCVA. If both eyes are eligible and have the same BCVA, the choice of study eye will be at the discretion of the Investigator and/or Surgeon.
Be between 12 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable).
Be male (XY chromosome) and have at least one documented pathogenic or likely pathogenic variant in the RPGR gene within exons 1-14 and/or ORF15 from a Clinical Laboratory Improvement Amendments of 1988 (CLIA)-certified 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 hematology and clinical chemistry evaluations performed at screening.
Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend study visits for the duration of the study.
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.
Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200) based on an ETDRS chart at each screening visit. ETDRS letter score is the main VA inclusion criterion for participants. Participants unable to read the ETDRS letters may utilize a tumbling “E” chart for BCVA assessments.
Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye based on an ETDRS chart at each screening visit. Participants unable to read the ETDRS letters may utilize a tumbling “E” chart for LLVA assessments.

What rules you out

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 agent or the quality of the assessments
Are currently participating or recently participated in any other research protocol involving investigational agents or therapies that 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 AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics.
Have pre-existing eye conditions 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 [CME]).
Have significant media opacity impacting evaluation of the retina or vitreous. 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 treatment administration.
Had intraocular surgery within 90 days of study treatment administration (Day 1).
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).
Have a history of corticosteroid-induced raised intraocular pressure (IOP) of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
Have any artificial retinal implant or prosthesis.
Have absence of clear ocular media and/or inadequate pupil dilation to facilitate good quality SD-OCT images.
Have any history of rhegmatogenous retinal detachment.
For participants with herpes simplex virus (HSV): a. Have history of oral or genital herpes and are unable and/or unwilling to utilize prophylactic antiviral medication. b. Have a history of ocular herpes. c. Have active oral or genital herpes or are currently receiving treatment for HSV infection.
Have myopia (spherical equivalent) exceeding −10 diopters (or axial length of >30 mm if PI deems it appropriate to measure) or presence of pathologic myopia in the study eye.
Have passed the Low Contrast Ora-VNC mobility course ≤0.35 lux light level in either eye or binocularly at any screening visit.
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 known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications.
Have used anti-coagulant agents that may alter coagulation (e.g., warfarin, heparin, apixaban, or high dose docosahexaenoic acid [DHA; fish oil]) within 7 days prior to study treatment administration (ibuprofen, aspirin, or similar are acceptable).
Have received any vaccination/immunization within 28 days prior to screening and/or during screening, except for the influenza vaccine, which is only exclusionary if they have received the influenza vaccine within 28 days prior to randomization.
Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to screening and/or intend to use during screening. 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 treatment administration.
Have any other condition or reason that, in the opinion of the Investigator, would prevent a participant from completing study assessments during the study.

The study team makes the final eligibility decision.

Where it's taking place

Australia
United States
United Kingdom
Canada

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling male, 18-64 years, 0-17 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Australia; United States; United Kingdom; Canada. Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.