Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC
Status:
COMPLETED
Trial end date:
2024-11-07
Target enrollment:
Participant gender:
Summary
This Phase 1b, open-label, single-center, prospective trial will be assessing the safety, tolerability, and efficacy of topical KM-001 1% in patients with PPPK1 or PC diseases. In this study 2 cohorts will be recruited:
1. Cohort 1: up to 11 eligible patients, will be enrolled to be treated twice daily, for 12 weeks, with 1% topical KM-001, on the plantar surfaces (2 feet).
2. Cohort 2: up to 8 eligible patients, will be enrolled to be treated twice daily, for 16 weeks, with 1% topical KM-001, on the plantar surfaces (2 feet).
Safety (AEs, blood work \[at specific visits\], vital signs), tolerability, and efficacy parameters (overall lesion improvement) will be assessed during in-clinic visits (Cohort 1: during Screening, Enrolment, and on Days 7, 28, 42, 63, 84 \[end of treatment, EoT\], 112 \[End of Study, EoS\] post first investigational medicinal product (IMP) administration; Cohort 2: during Screening, Enrolment, and on Days 7, 28, 42, 63, 84, 112 \[EoT\], 140 \[EoS\] post first investigational medicinal product (IMP) administration).
PK samples will be collected to assess plasma levels of KM-001 on
* Screening (Day -14 to -0): any time during the visit. (or on Day 1 up to 30 minutes pre-dose if missed during Screening)
* Day 7 and at EoT (Cohort 1: Day 84; Cohort 2: Day 112) up to 30 minutes pre-dose, and at 1 h, 2 h, 3 h, 6 h (+15 min) post-dose
* Days 28, 42 for both Cohorts, and Day 84 for Cohort 2: 1 sample after the first dose, before the second dose, as late as possible in the visit.
* End of Study (EoS, Day 112 (Cohort 1) or Day 140 (cohort 2)), or at Early Termination (ET): at any time during the visit.
The patient will complete a patient-reported diary, consisting of treatment compliance and self-assessments for efficacy.
Follow up- 2 weeks after EoT by phone call, and 4 weeks after EoT in clinic visit.