Study Designed to Evaluate Safety and Efficacy of 1% Topical Formulation of KM-001 on Type 1 Punc… (NCT05435638) | Clinical Trial Compass
CompletedPhase 1
Study Designed to Evaluate Safety and Efficacy of 1% Topical Formulation of KM-001 on Type 1 Punctate Palmoplantar Keratoderma or Pachyonychia Congenita Diseases
Israel14 participantsStarted 2022-07-17
Plain-language summary
In this phase 1 open label study for patients with type I punctate palmoplantar keratoderma or pachyonychia congenital, 2 arms will be recruited to be treated twice daily, with 1% topical KM-001.
Arm 1: up to 10 eligible patients will be treated for 12 weeks. Arm 2: up to 8 eligible patients will be treated for 16 weeks.
Treatment safety and efficacy will be assessed in the clinic visits (for arm 1 up to day 91, for arm 2 up to day 126). In between safety will also be assessed by phone visits.
At the in-clinic visits, treatment efficacy (lesion clearance - IGA, CGI-S, PGI-C, PGI-S and VAS pain) will also be assessed.
PK blood samples will be collected for arm 1: on Days 0, 7, 84 (EoT visit). One week after the end of treatment (EoT) visit, patients will return to the clinic for final safety, efficacy and PK evaluations. For arm 2, PK blood samples will be collected on days 0, 7, 84, 112 (EoT visit). Two weeks after the end of treatment (EoT) visit, patients will return to the clinic for final safety, efficacy and PK evaluations.
Who can participate
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.
Inclusion criteria
. Read, understood and signed an informed consent form (ICF) before any investigational procedure(s) are performed.
. Male or female and aged 18 - 75 years at the time of screening
. Clinical diagnosis of:
. The target treatment region is 0.5%-4% body surface area (BSA) including target lesions
. CGI-S score of ≥2 (as assessed by the PI at screening).
. Female patients of childbearing potential must agree to use a highly effective and approved method of contraception throughout the study and for 4 weeks after the last study drug administration. Male patients: female partners of male patients must use a reliable method of contraception during this study, and for 12 weeks after the last dose of study medications.
Questions worth asking your doctor
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
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.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety endpoint will be assessed through collection and analysis of adverse events
Timeframe: up to 91 days for arm 1 and 126 days for arm 2
2
Safety endpoint-will be assessed through collection and analysis of blood laboratory tests.
Timeframe: up to 91 days for arm 1 and 126 days for arm 2
3
Safety endpoint-will be assessed through collection and analysis of urine laboratory tes
Timeframe: up to 91 days for arm 1 and 126 days for arm 2
4
Safety endpoint-Vital signs- Heart rate
Timeframe: up to 91 days for arm 1 and 126 days for arm 2
5
Safety endpoint-Vital signs- Blood Pressure
Timeframe: up to 91 days for arm 1 and 126 days for arm 2
6
Safety endpoint-ECG
Timeframe: up to 91 days for arm 1 and 126 days for arm 2
7
Safety endpoint- Lesion Assessment
Timeframe: up to 91 days for arm 1 and 126 days for arm 2
. Female patients must refrain from donating eggs throughout the study and for 4 weeks after the last study drug administration. Male patients must refrain from sperm donation throughout the study and for 12 weeks after the last study drug administration.
. Female patients of non-childbearing potential must meet one of the following criteria:
Exclusion criteria
. Known hypersensitivity or any suspected cross-allergy to the active pharmaceutical ingredient and/or excipients.
. Regular alcohol consumption for males \>21 units per week and for females \>14 units per week (1 unit = 8gr of alcohol; e.g., 200 mL of 5% beer, 25 mL of 40% spirits or 125 mL of 8% wine).
. Any medical or active psychological condition or any clinically relevant laboratory abnormalities, such as, but not limited, to elevated ALT or AST (\>3 × upper limit of normal \[ULN\]) in combination with elevated bilirubin (\>2 × ULN), at screening/ baseline that may put the patient at significant risk according to the investigator's judgment, if he/she participates in the clinical study, or may interfere with study assessments (e.g., poor venous access or needle-phobia).
. Planned or expected major surgical procedure during the clinical study.
. Patient is unwilling to refrain from using prohibited medications during the clinical study.
. Currently participating or participated in any other clinical study of a drug or device, within the past 4 months before screening, or is in an exclusion period (if verifiable) from a previous study.
. Cutaneous infection or another active underlying skin condition, regardless of location.
. Cutaneous infection of the area to be applied with KM-001, requiring treatment with oral or parenteral antibiotics, antivirals, antiparasitics or antifungals, or any topical treatments during and/or up-to 2 weeks before screening.