Clinical trial NCT05244304: Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Stargardt Disease 1
Lead sponsor: Belite Bio, Inc
Sponsor class: INDUSTRY
First posted: 2022-02-17
Enrollment: 104
Sex: ALL
Minimum age (years): 12
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This is a Phase 3 randomized, double-masked, parallel group, multicenter study to evaluate the efficacy and safety of Tinlarebant 5 mg in the treatment of adolescent subjects with STGD1.
Primary purpose: TREATMENT

Brief 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

Detailed 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.

Eligibility criteria: 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).

Primary outcomes:
measure: To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
timeFrame: Baseline thru month 24
Secondary outcomes:
measure: To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
timeFrame: Baseline thru month 24
measure: To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
timeFrame: Baseline thru month 24
measure: To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
timeFrame: Baseline thru month 24
measure: To measure change in plasma concentration of RBP4 levels (μM) from baseline
timeFrame: Baseline thru month 24
measure: 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
timeFrame: Baseline thru month 24
measure: To assess the systemic and ocular safety and tolerability of tinlarebant
timeFrame: Baseline thru month 24
Other outcomes:
measure: To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline
timeFrame: Baseline thru month 24
measure: To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline
timeFrame: Baseline thru month 24
measure: To measure change in quantitative autofluorescence (qAF) level from baseline
timeFrame: Baseline thru month 24
measure: To measure change in retinal sensitivity by microperimetry from baseline
timeFrame: Baseline thru month 24
Locations:
city: Palo Alto
country: United States
facility: Belite Study Site
state: California
city: Minneapolis
country: United States
facility: Belite Study Site
state: Minnesota
city: Salt Lake City
country: United States
facility: Belite Study Site
state: Utah
city: Westmead
country: Australia
facility: Belite Study Site
state: New South Wales
city: East Melbourne
country: Australia
facility: Belite Study Site
state: Victoria
city: South Brisbane
country: Australia
facility: Belite Study Site
city: Gent
country: Belgium
facility: Belite Study Site
city: Beijing
country: China
facility: Belite Study Site
city: Shanghai
country: China
facility: Belite Study Site
city: Paris
country: France
facility: Belite Study Site
city: Bonn
country: Germany
facility: Belite Study Site
city: Tübingen
country: Germany
facility: Belite Study Site
city: Kowloon
country: Hong Kong
facility: Belite Study Site
city: Nijmegen
country: Netherlands
facility: Belite Study Site
city: Basel
country: Switzerland
facility: Belite Study Site
city: Taipei
country: Taiwan
facility: Belite Study Site
city: Taoyuan City
country: Taiwan
facility: Belite Study Site
city: London
country: United Kingdom
facility: Belite Study Site
city: Southampton
country: United Kingdom
facility: Belite Study Site
Study officials:
References: