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

Stargardt Disease 1 Clinical Trial

— DRAGON  

Official title:

Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05244304
• Other study ID #: LBS-008-CT03
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: March 28, 2022
• Est. completion date: October 2025

Study information

• Verified date: June 2024
• Source: Belite Bio, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 104
• Est. completion date: October 2025
• Est. primary completion date: August 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 20 Years

Eligibility

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).

Related Conditions & MeSH terms

• Macular Degeneration
• Stargardt Disease
• Stargardt Disease 1

Intervention

Drug:
• Tinlarebant
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
• Placebo
Not active drug

Locations

Country	Name	City	State
Australia	Belite Study Site	East Melbourne	Victoria
Australia	Belite Study Site	South Brisbane
Australia	Belite Study Site	Westmead	New South Wales
Belgium	Belite Study Site	Gent
China	Belite Study Site	Beijing
China	Belite Study Site	Shanghai
France	Belite Study Site	Paris
Germany	Belite Study Site	Bonn
Germany	Belite Study Site	Tübingen
Hong Kong	Belite Study Site	Kowloon
Netherlands	Belite Study Site	Nijmegen
Switzerland	Belite Study Site	Basel
Taiwan	Belite Study Site	Taipei
Taiwan	Belite Study Site	Taoyuan City
United Kingdom	Belite Study Site	London
United Kingdom	Belite Study Site	Southampton
United States	Belite Study Site	Minneapolis	Minnesota
United States	Belite Study Site	Palo Alto	California
United States	Belite Study Site	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Belite Bio, Inc

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  China,  France,  Germany,  Hong Kong,  Netherlands,  Switzerland,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline		Baseline thru month 24
Other	To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline		Baseline thru month 24
Other	To measure change in quantitative autofluorescence (qAF) level from baseline		Baseline thru month 24
Other	To measure change in retinal sensitivity by microperimetry from baseline		Baseline thru month 24
Primary	To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline		Baseline thru month 24
Secondary	To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline		Baseline thru month 24
Secondary	To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline		Baseline thru month 24
Secondary	To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline		Baseline thru month 24
Secondary	To measure change in plasma concentration of RBP4 levels (µM) from baseline		Baseline thru month 24
Secondary	The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline		Baseline thru month 24
Secondary	To assess the systemic and ocular safety and tolerability of tinlarebant	Frequency, duration, and severity of AEs	Baseline thru month 24