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.
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Safety endpoint will be assessed through collection and analysis of adverse events.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of Hematology- laboratory blood test profile.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of MCH result in laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of MCV result in laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of haematocrit result in laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of hemoglobin result in laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of RBC result in laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of Sodium, potassium and chloride results in chemistry laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of: Glucose fasting , BUN/Ur, creatinine, bilirubin total and direct, calcium, uric acid, and bilirubin result, in chemistry laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of Alkaline phosphatase, AST, ALT and GGT result in chemistry- laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of Albumin and total protein result in chemistry- laboratory blood test.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of Serology -in laboratory blood test profile.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of urine laboratory profile.
Timeframe: Up to 112 days
Safety endpoint-will be assessed by the % of change from normal range in the collection of Specific gravity , pH, blood and nitrites result in urine laboratory profile.
Timeframe: Up to 112 days
Safety endpoint-Mean change in body temperature measurement from baseline.
Timeframe: Up to 112 days
Safety endpoint-Mean change in pulse measurement from baseline.
Timeframe: Up to 112 days
Safety endpoint- Mean change in blood pressure measurement from baseline.
Timeframe: Up to 112 days
Safety endpoint-ECG
Timeframe: Up to 112 days