DRAGON II

12 evidence topics · 4 sources

Objective

Study objective

“The purpose of this study is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease.” (opens the source at this quote in a new tab)

Location and study date

Trial locations

“Japan, the U.S., and the United Kingdom.” (opens the source at this quote in a new tab)

UK protocol: ethics opinion

Field	Quoted record
Ethics opinion date	“14 Jun 2024”
Ethics committee	“Wales REC 3”

Study design

DRAGON II phase 1b and phase 2/3 trials: randomization

“randomized (1:1, active: placebo)” (opens the source at this quote in a new tab)

DRAGON II phase 1b and phase 2/3 trials: study duration and visits

“The total study duration from screening through to follow-up is 26 months, with participants returning to the study site every 3 months for check-ups.” (opens the source at this quote in a new tab)

Design of the phase 1b and phase 2/3 components

Component and feature	Quoted record
Phase 1b masking	“open-label”
Phase 2/3 masking	“double-masked”
Phase 2/3 treatment duration	“24-month”

Eligibility criteria

DRAGON II phase 1b and phase 2/3 trials: inclusion criterion

“Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene.” (opens the source at this quote in a new tab)

DRAGON II phase 1b and phase 2/3 trials: age range

“12 to 20 years old” (opens the source at this quote in a new tab)

DRAGON II phase 1b and phase 2/3 trials: exclusion criterion

“Any prior gene therapy.” (opens the source at this quote in a new tab)

Treatment

Study outcomes

Rationale for using atrophic lesion growth as the primary endpoint

Primary efficacy outcome and safety assessments

“growth rate of atrophic lesions; safety and tolerability will also be assessed.” (opens the source at this quote in a new tab)

Trial-specific endpoint-validation analysis

No evidence found.

Statistical analysis description

Number of participants (Planned and analyzed)

DRAGON II phase 1b and phase 2/3 trials: enrollment

“Completed enrollment with 73 subjects, including 15 Japanese subjects for the Phase 2/3 trial in STGD1.” (opens the source at this quote in a new tab)

Description of analysis sets

No evidence found.

Results

Participant disposition

No evidence found.

Baseline characteristics

No evidence found.

Efficacy results

No evidence found.

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

No evidence found.

Study limitations

Summary

The available update establishes enrollment of 73 participants, including 15 Japanese participants, in the phase 2/3 component. The evidence collected here describes its design and eligibility rather than treatment-effect estimates or completed safety results. Its age range of 12–20 years and requirement for clinically diagnosed STGD1 with an identified ABCA4 mutation constrain the population to which future findings can directly apply.