Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease (DRAGON)

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Status and phase: Active, not recruiting; Phase 3

Conditions: Stargardt Disease 1

Treatments: Drug: Placebo; Drug: Tinlarebant

Study type: Interventional

Funder types: Industry

Identifiers

NCT05244304

LBS-008-CT03

Details and patient eligibility

About

The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

Full description

Approximately 90 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.

Enrollment: 104 patients

Sex: All

Ages: 12 to 20 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female subjects 12 to 20 years old, inclusive.
• Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene.
• Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score
• Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures.
• Subject agrees to comply with all protocol requirements.

Exclusion criteria

• Any ocular disease other than Stargardt (STGD1) at baseline 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.
• Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
• Any prior gene therapy.
• Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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104 participants in 2 patient groups, including a placebo group

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 are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance.

Treatment:

Drug: Placebo