ARB study: OPGx-BEST1
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?
an investigational genetic therapy being studied as a potential treatment for BVMD and ARB caused by BEST1 mutations is designed to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells to restore normal protein function
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
- This study is testing whether a new medicine called OPGx-BEST1 is safe. It also looks at how well it works for two rare eye conditions.
- Who it's for
- You may be able to join if you have Best Vitelliform Macular Dystrophy or Autosomal-Recessive Bestrophinopathy.
- What's involved
- You would get one injection of the medicine in one eye. The team will check your eyes often to see how you are doing.
- Time commitment
- The study lasts for five years after your injection.
- 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
- Provide informed consent to study assessments.
- Able and willing to comply with all study assessments for the duration of the study.
- ≥18 years old.
- 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]).
You likely can't join if
- 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.
- 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.
- Presence of active choroidal neovascularization (CNV) that, in the opinion of the Investigator, affects vision or may require treatment.
See the full eligibility criteria
- Provide informed consent to study assessments.
- Able and willing to comply with all study assessments for the duration of the study.
- ≥18 years old.
- 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.
- Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year after IMP administration.
- 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 is enrolling
- Los Angeles, California, United States
- New York, New York, 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, but 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?
This study is enrolling now at Los Angeles, California, United States; New York, New York, United States; Cincinnati, Ohio, United States; Dallas, Texas, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.