Pharmacokinetics and Pharmacodynamics Study of LBS-008 in Healthy Volunteers Aged 50-85
Condition: Healthy Volunteer; Dry Age-related Macular Degeneration
MeSH terms: Macular Degeneration
Study type: Interventional
Phase: Phase 1

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.
    

Intervention name(s): Tinlarebant (LBS-008)
Intervention type(s): Drug
Intervention description(s): A single dose of Tinlarebant (LBS-008) will be administered to each study participant on study Day 1.
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Basic Science