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Tests treatment safety and results for ARB
Official title Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
ClinicalTrials.gov ID: NCT07185256
What this study is testing
What is OPGx-BEST1?
OPGx-BEST1 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for arb.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The goal of this clinical trial is to learn if drug OPGx-BEST1 works to treat BVMD and ARB Bestrophinopathy. It will also learn about the safety of drug OPGx-BEST1.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Individuals who meet all the following criteria will be eligible to participate in the study:
- Provide informed consent to study assessments.
- Able and willing to comply with all study assessments for the duration of the study.
- ≥18 years old.
- ETDRS BCVA measured with standard testing distances:
You likely can't join if
- Individuals who meet any of the following criteria will not be eligible to participate in the study.
- Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year...
- Men who are unwilling to use adequate contraception from Screening through 180 days after IMP administration. See Section 13.3 (Appendix 3) for...
- Have a pre-existing eye condition or complicating systemic disease that could preclude the planned surgery (e.g., individuals who are...
- Have a history of disease that may preclude the individual from study participation (e.g., other bestrophinopathy, such as AOFVD or ADVIRC) or that...
- Have previously received gene therapy of any kind.
See the full eligibility criteria
- Individuals who meet all the following criteria will be eligible to participate in the study:
- Provide informed consent to study assessments.
- Able and willing to comply with all study assessments for the duration of the study.
- ≥18 years old.
- ETDRS BCVA measured with standard testing distances:
- For the sentinel participant in each cohort, ≤20 letters (Snellen equivalent of 20/200 [1.30 logMAR] or worse)
- For subsequent participants in the same cohort, 65 to 20 letters inclusive (Snellen equivalent of 20/50 [0.40 logMAR] to 20/200 [1.30 logMAR]).
- Genetic confirmation on chromosome 11q12-q13.1 of BVMD or ARB with a BEST1 genetic test or IRD panel test including a BEST1 variant test, by a Clinical Laboratory Improvement Amendments (CLIA) or European certified...
- Confirmation of one disease-causing (pathogenic or likely pathogenic, autosomal-dominant) variant in the BEST1 gene for BVMD, as listed in the IB, or two variants for ARB per American College of Medical Genetics and...
- BVMD: Clinical phenotype and diagnosis consistent with advanced BVMD with active subretinal fluid or vitelliform material.
- ARB: Clinical phenotype and diagnosis consistent with ARB.
- Individuals who meet any of the following criteria will not be eligible to participate in the study.
- Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year after IMP administration. See Section 13.3 (Appendix 3) for...
- Men who are unwilling to use adequate contraception from Screening through 180 days after IMP administration. See Section 13.3 (Appendix 3) for contraception guidelines.
- Have a pre-existing eye condition or complicating systemic disease that could preclude the planned surgery (e.g., individuals who are immunocompromised, on continuous systemic immunosuppressive treatment or anticipate a...
- Have a history of disease that may preclude the individual from study participation (e.g., other bestrophinopathy, such as AOFVD or ADVIRC) or that may interfere with or preclude outcome measure testing as described in...
- Have previously received gene therapy of any kind.
- Presence of active choroidal neovascularization (CNV) that, in the opinion of the Investigator, affects vision or may require treatment.
- Presence of subretinal fibrosis that may significantly limit improvement in visual acuity.
- Have an epiretinal membrane that may require surgical intervention.
- Have undergone tube surgery for glaucoma at any time or have glaucoma that has been unstable within the past 4 years. Note: Prior incisional surgery (e.g., trabeculectomy and iridotomy) is not exclusionary.
- Had intraocular surgery within 90 days prior to planned IMP administration or have active inflammation at Screening resulting from prior ocular surgery.
- Have used any investigational drug or device within 90 days prior to planned IMP administration or plan to participate in another drug or device study during the same period as the current study.
- Have received a vaccination within 6 weeks prior to planned IMP administration or plan to be vaccinated within 6 months after IMP administration.
- Have received anticoagulant therapy within 2 weeks prior to planned IMP administration.
- Have active macular neovascularization as determined by OCT-A.
- Are incapable of performing visual function testing for reasons other than poor vision.
- Have any contraindication to the concomitant steroid regiment proscribed in the protocol.
- Have any other condition (ocular, medical, or psychological) that would not allow the individual to complete follow-up examinations during the study and/or, in the opinion of the Investigator, makes it hazardous or...
- Have a known history of hypersensitivity to constituents or excipients in the pharmaceutical formulation of the IMP.
- Have a known infection of human immunodeficiency virus (HIV), hepatitis B or C virus, or herpes simplex virus.
- Are an employee of the Sponsor or a relative of the Investigator or investigative site staff.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Gainesville, Florida, United States
- Cincinnati, Ohio, United States
- Dallas, Texas, United States
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 all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Gainesville, Florida, United States; Cincinnati, Ohio, United States; Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.