This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

RBP4

Status and phase: Completed; Phase 2; Phase 1

Conditions: Stargardt Disease

Treatments: Drug: tinlarebant

Study type: Interventional

Funder types: Industry

Identifiers

NCT05266014

LBS-008-CT02

Details and patient eligibility

About

Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis.

This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.

Enrollment: 13 patients

Sex: All

Ages: 12 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Major Inclusion Criteria:

Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene.

Major Exclusion Criteria:

Any ocular disease other than Stargardt disease at baseline that, in the opinion of the PI, would complicate assessment of a treatment effect.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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13 participants in 1 patient group

tinlarebant

Experimental group

Description:

Daily, oral administration of one tinlarebant.

Treatment:

Drug: tinlarebant