LBS-008-CT02
11 evidence topics · 7 sources
Objective
Location and study date
Study design
Phase 1b/2 dose-finding and two-year extension: design
Eligibility criteria
Phase 1b/2: adolescent age range
Treatment
Phase 1b/2: oral regimen
Study outcomes
Rationale for using systemic and ocular safety and tolerability as the primary endpoint
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Phase 1b/2 dose-finding and two-year extension: enrollment
Description of analysis sets
External natural-history comparison population
Results
Participant disposition
Phase 1b/2 dose-finding and two-year extension: adverse-event withdrawals
Baseline characteristics
Efficacy results
Phase 1b/2 dose-finding and two-year extension: 24-month results
Phase 1b/2 dose-finding and two-year extension: additional analysis
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Study limitations
Summary
The supporting study was open-label and enrolled 13 participants. Its 24-month lesion result was reported for 12 participants, and the visual-acuity analysis concerned a subgroup of 6 with prior bilateral visual loss. The natural-history comparison used selected ProgStar participants rather than a concurrently randomized placebo group. These design features and small denominators limit causal interpretation of lesion and visual-acuity findings. The reported genotype distribution, with severe pathogenic or likely pathogenic ABCA4 variants in 11 participants (85%), further describes the selected study population.