A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 … (NCT04850118) | Clinical Trial Compass
Active — Not RecruitingPhase 2/3
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
United States85 participantsStarted 2024-03-14
Plain-language summary
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.
Who can participate
Age range12 Years – 50 Years
SexMALE
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Inclusion criteria
✓. 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 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.
✓. Have a clinical diagnosis of XLRP.
✓. 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.
✓. Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200)
✓. Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye
✓. 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.
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 agent or the quality of the assessments.
✕
What they're measuring
1
The proportion of participants with a ≥15 letter increase from baseline in LLVA
. For participants with herpes simplex virus (HSV):
✕. Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication.
✕. Have a history of ocular herpes.
✕. Have active oral or genital herpes or are currently receiving treatment for HSV infection.
✕. Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications.
✕. Have used anti-coagulant agents that may alter coagulation
✕. 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