A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease

A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease

Status

Active, not recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06388083

Acronym

DRAGON II

Enrollment

Registered

2024-04-29

Start date

2024-07-31

Completion date

2028-02-01

Last updated

2026-03-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Stargardt Disease 1, STGD1

Brief summary

The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease

Detailed description

This study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.

Interventions

DRUGTinlarebant

5 mg tablet

DRUGPlacebo

Placebo tablets

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender

ALL

Age

12 Years to 20 Years

Healthy volunteers

Inclusion criteria

* Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene. * Subjects must have a defined aggregate atrophic lesion in 1 or both eyes. * Minimum BCVA is required in the study eye

Exclusion criteria

* Any ocular disease other than STGD1 that, in the opinion of the investigator, would complicate assessment of a treatment effect. * History of ocular surgery in the study eye in the last 3 months. * Any prior gene therapy.

Design outcomes

Primary

Measure	Time frame
To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy	From baseline to Month 24

Secondary

Measure	Time frame
To measure the annualized rate of change in total area of atrophy	From baseline to Month 24
Change in BCVA measured by the ETDRS method	From baseline to Month 24

Countries

Japan, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 13, 2026