NCT05667688

Pharmacokinetics and Pharmacodynamics Study of LBS-008 in Healthy Volunteers Aged 50-85

A Phase 1b, Open-Label, Parallel Single-Dose Study to Evaluate the Pharmacokinetics and Pharmacodynamics of Tinlarebant in Healthy Volunteers Aged 50-85

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: RBP4 Pty Ltd · Industry
• Sex: All
• Age: 50 Years – 85 Years
• Healthy volunteers: Accepted

Summary

This is a Phase 1b, parallel single-dose study to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of Tinlarebant when administered as an oral dose to elderly healthy volunteers. This study will evaluate 2 dose levels in 2 cohorts comprising up to a total of 16 participants (8 per cohort). Dose levels will be evaluated in parallel.

Detailed description

This is a Phase 1b, parallel single-dose study to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of tinlarebant when administered as an oral dose to healthy volunteers aged 50-85. Tinlarebant (previously called LBS-008) is being developed as an oral treatment to slow or halt the progression of dry age-related macular degeneration (AMD) and juvenile onset macular degeneration, known as Stargardt disease (STGD). Accumulation of lipofuscin bisretinoids in the retina is associated with several retinal degenerative diseases including dry AMD and STGD. A total of up to 16 healthy volunteers aged 50-85 are planned to be enrolled into 2 cohorts (8 participants per cohort). Participants will receive a single oral dose of tinlarebant, either 5 mg (Cohort 1) or 10 mg (Cohort 2). The total maximum study duration for participants in this study is 45 days, inclusive of visit windows. This includes a screening period of up to 27 days (Day -28 to Day -2), confinement to the clinical facility over 2 nights (Day -1 to Day 2) and 5 outpatient visits including the EOS visit or Day 15. Tinlarebant will be administered in tablet form; a single tinlarebant tablet will contain 5 mg of IP. Participants in Cohort 1 will receive 1 tinlarebant tablet (5 mg dose) and participants in Cohort 2 will receive 2 tinlarebant tablets (10 mg dose). Enrolment and dosing of participants in Cohorts 1 and 2 may occur concurrently. Healthy volunteers aged 50-85, will be screened between Day -28 and Day -2, inclusive. Participants will be admitted to the clinical facility on Day -1 with tinlarebant dosing to occur on the following day (Day 1). All participants will be dosed under fasted conditions. Participants will be confined at the clinical facility from Day -1 through to Day 2 with discharge following completion of all PK and PD blood sample collections and other scheduled assessments on Day2. If participants experience any clinically significant (CS) AEs during the confinement period, they may remain in the clinical facility for further observation at the discretion of the Principal Investigator (PI). Blood samples for PK and PD analysis will be collected pre-dose and at timepoints up to 24 hours (Day 2) post-dose during the confinement period. Participants will be required to attend the clinic as outpatients on Days 3, 4, 6 and 8 for further safety assessments, PK and PD blood sampling. Participants will then return to the clinical facility for final safety assessments at an end of study (EOS) vision Day 15. In the event of early study termination prior to the EOS visit (Day 15), participants will be requested to attend the clinic for an early termination (ET) visit.

Conditions

• Healthy Volunteer
• Dry Age-related Macular Degeneration

Interventions

Type	Name	Description
DRUG	Tinlarebant (LBS-008)	A single dose of Tinlarebant (LBS-008) will be administered to each study participant on study Day 1.

Timeline

• Start date: 2022-11-28
• Primary completion: 2023-01-04
• Completion: 2023-01-18
• First posted: 2022-12-28
• Last updated: 2023-02-23

Locations

1 site across 1 country: Australia

Regulatory

• FDA-regulated drug study