DRAGON II

JRCT ID: jRCT2031240209

Registered date:07/07/2024

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Stargardt Disease
• Date of first enrollment: 24/07/2024
• Target sample size: 66
• Countries of recruitment: United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Investigational medical product: 5 mg tinlarebant tablets.
Administration route: oral.
The proposed dose level for both the Phase 1b and Phase 2/3 parts of the study is 5 mg tinlarebant once daily.
Reference product: placebo tablets
Administration route: oral.
Placebo will be administered in the Phase 2/3 part of the study only.

Outcome(s)

• Primary Outcome: To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy

Secondary Outcome

To measure the annualized rate of change in total area of atrophy
Change in BCVA measured by the ETDRS method

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: <= 20age old
• Gender: Both

Include 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

Exclude 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

Related Information

• Primary Sponsor: Mata Nathan
• Secondary ID(s): NCT06388083