Tinlarebant for Stargardt Disease
(DRAGON II Trial)
Recruiting in Palo Alto (17 mi)
+12 other locations
Age: < 65
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2 & 3
Recruiting
Sponsor: Belite Bio, Inc
Disqualifiers: Ocular disease, Ocular surgery, Gene therapy
Prior Safety Data
Trial Summary
What is the purpose of this trial?The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
How is the drug Tinlarebant unique for treating Stargardt disease?
Tinlarebant is unique because it aims to reduce the accumulation of vitamin A dimers and lipofuscin, which are substances that build up in the eyes of people with Stargardt disease, potentially slowing the progression of vision loss. Currently, there are no standard treatments that have fully passed clinical trials for this condition.
12345Eligibility Criteria
This trial is for individuals with Stargardt Disease, a type of inherited eye disorder that causes vision loss. Participants should have a diagnosis of this condition to be eligible.Inclusion Criteria
Minimum BCVA is required in the study eye
I have Stargardt disease with a confirmed ABCA4 gene mutation.
I have a specific type of eye damage in one or both eyes.
Exclusion Criteria
I have received gene therapy before.
I have no eye conditions affecting treatment assessment besides STGD1.
I have not had eye surgery in the last 3 months.
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Phase 1b Treatment
Open-label evaluation of pharmacokinetics, pharmacodynamics, safety, and tolerability of daily doses of 5 mg tinlarebant for 7 days in Japanese subjects
1 week
Phase 2/3 Treatment
Randomized, double-masked, placebo-controlled evaluation of safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant for 24 months
24 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The study is testing the effectiveness and safety of a drug called Tinlarebant compared to a placebo in treating Stargardt Disease. It's designed to see if Tinlarebant can help with symptoms or slow down progression.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: LBS-008, TinlarebantExperimental Treatment1 Intervention
5 mg tablet taken orally once a day
Group II: PlaceboPlacebo Group1 Intervention
Placebo tablets for tinlarebant 5 mg prepared similarly
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Belite Study SitePhoenix, AZ
Belite Study SiteLa Jolla, CA
Belite Study SiteMiami, FL
Belite Study SiteBoston, MA
More Trial Locations
Loading ...
Who Is Running the Clinical Trial?
Belite Bio, IncLead Sponsor
References
Stargardt Disease. [2019]Stargardt disease (STGD) is one of the most common macular dystrophies in young adults. It progresses slowly. Its prevalence is about 1:8000-10,000. Age of onset is a surrogate marker: The earlier the onset, the more severe the disease course. Onset usually occurs in childhood or early adolescence, at about 10-15 years of age. Vision is between about 20/70 and 20/200. The fundus shows a bull's eye pattern or beaten-bronze appearance, with or without yellowish flecks (fundus flavimaculatus). Fluorescein angiography may show dark choroid in about 80% of cases. On fundus autofluorescence (FAF), newer flecks appear hyperautofluorescent (hyperAF); older ones become progressively more hypoAF with time. Some flecks are surrounded by a ring of decreased AF. Peripapillary sparing is one the characteristics of Stargardt disease, but this area can be involved in about 2-7% of cases. The reason for this sparing is unclear; this area may be more resilient to the deleterious effect of ABCA4 gene mutation, and there might be a more favorable RPE photoreceptor ratio, resulting in less lipofuscin build-up, in the presence of a thicker overlying peripapillary retinal nerve fiber layer. Patients with Stargardt disease should avoid bright light and excessive vitamin A.
Stargardt disease: monitoring incidence and diagnostic trends in the Netherlands using a nationwide disease registry. [2022]To assess the incidence of Stargardt disease (STGD1) and to evaluate demographics of incident cases.
Clinical and molecular characteristics of childhood-onset Stargardt disease. [2022]To describe the clinical and molecular characteristics of patients with childhood-onset Stargardt disease (STGD).
[New possibilities in the treatment of Stargardt disease]. [2020]Stargardt disease is a hereditary retinal dystrophy associated with mutations in the ABCA4 gene. Currently, no etiopathogenetic drugs nor treatment methods for Stargardt disease have completely passed clinical trials. The review summarizes experimental and clinical studies of drugs aimed at reducing the accumulation of vitamin A dimers, lipofuscin, complement inhibition and RPE regeneration by stem cell transplantation, as well as gene therapy studies with intravitreal vector injection of the ABCA4 functional gene.
A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy. [2022]Stargardt disease (STGD, also known as fundus flavimaculatus; FFM) is an autosomal recessive retinal disorder characterized by a juvenile-onset macular dystrophy, alterations of the peripheral retina, and subretinal deposition of lipofuscin-like material. A gene encoding an ATP-binding cassette (ABC) transporter was mapped to the 2-cM (centiMorgan) interval at 1p13-p21 previously shown by linkage analysis to harbour the STGD gene. This gene, ABCR, is expressed exclusively and at high levels in the retina, in rod but not cone photoreceptors, as detected by in situ hybridization. Mutational analysis of ABCR in STGD families revealed a total of 19 different mutations including homozygous mutations in two families with consanguineous parentage. These data indicate that ABCR is the causal gene of STGD/FFM.