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

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Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Stargardt Disease 1; STGD1

Treatments: Drug: Tinlarebant; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT06388083

LBS-008-CT07

Details and patient eligibility

About

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

Full 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.

Enrollment: 60 estimated patients

Sex: All

Ages: 12 to 20 years old

Volunteers: No 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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60 participants in 2 patient groups, including a placebo group

LBS-008, Tinlarebant

Experimental group

Description:

5 mg tablet taken orally once a day

Treatment:

Drug: Tinlarebant

Placebo

Placebo Comparator group

Description:

Placebo tablets for tinlarebant 5 mg prepared similarly

Treatment:

Drug: Placebo