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Pachyonychia CongenitaPhase 2NCT07893483

Safety, Tolerability, and Efficacy of Oral KM-023 in Adult Patients With Pachyonychia Congenita (pEDD-PC)

A Phase 2 study testing KM-023 in people with Pachyonychia Congenita, run by Orvida Pharma (formerly Kamari Pharma) at 2 sites in Israel.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

The goal of this clinical trial is to learn if KM-023 is safe and well tolerated in adults with Pachyonychia Congenita (PC). The study will also explore whether KM-023 may help reduce pain, improve mobility, and lessen the impact of PC on participants' daily lives. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pachyonychia Congenita
What is being tested?
KM-023
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
2 sitesIsrael
Study dates
Dec 2026to Nov 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • To be eligible to participate in this trial, an individual must meet all the following criteria:
  • Read, understood, and signed an informed consent form (ICF) before any investigational procedures are performed.
  • Male or female, aged ≥ 18 years at the time of Screening.
  • Clinical diagnosis of KRT6A-pEDD-PC, KRT6B-pEDD-PC, KRT6C-pEDD-PC, KRT16-pEDD-PC and KRT17-pEDD-PC, with confirmed heterozygous variant in either KRT6A, KRT6B, KRT6C, KRT16, or KRT17 genes.
  • Apparent and visible calluses on the plantar area.
  • The Screening TUG test duration with socks was longer than the aged-matched healthy normal range (Kear, Guck \& McGaha (2016) and Bohannon (2006)).
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  • Age range Normative duration for TUG completion (Sec) 18-29 8.57 30-49 8.86 50 years or older 9.90
  • The patient has trimmed the plantar calluses no later than 4 weeks prior to D1.
  • The patient must have adequate renal function, defined as eGFR ≥60 mL/min/1.73 m² calculated using the Chronic Kidney Disease Epidemiology Collaboration CKD-EPI (or equivalent standardized method as reported by the local laboratory), with no clinically significant renal disease, as judged by the investigator.
  • Female participants of childbearing potential, and male participants capable of fathering a child, must meet the contraception requirements and agree to use one highly effective method and one physical barrier method of contraception consistently and correctly with their partners:
  • Female participants of childbearing potential must use contraception, hormonal contraceptives starting at least 1 month prior to screening and all other contraceptive methods from 14 days prior to first study drug administration and continuing for a duration of 6 months after the last dose of KM-023.
  • Male participants and their female partners of childbearing potential must use contraception from the first day of study drug administration and continuing for a duration of 90 days after the last dose of KM-023. The female partners, if using hormonal contraceptives, should also have begun hormonal contraceptives at least 1 month prior to screening of the male participant.
  • Negative pregnancy test at the baseline visit, and negative urinary pregnancy test at every clinic visit.
  • Female participants of non-childbearing potential must meet one of the following criteria:
  • Absence of menstrual bleeding for 1 year prior to screening without any other medical reason.
  • Documented hysterectomy or bilateral oophorectomy at least 3 months before the trial.
  • Not using prescription medication 14 days prior to admission; and 7 days prior to admission for over the counter (OTC) medication/vitamins/supplements (with the exception of contraception, occasional paracetamol and standard dose of multivitamins). As an exception, acetaminophen/paracetamol may be used at doses of ≤ 1g/day.
  • Willing and able to comply with the treatment and follow-up visits.
  • Willing to refrain from current treatments, in compliance with the list of prohibited medications.
  • Covered by a health insurance system where applicable, and/or in compliance with the recommendations of the national laws in force relating to biomedical research.

Who cannot join

  • An individual who meets any of the following criteria will be excluded from participation in this trial:
  • Participants who are staff members at the investigational site directly involved in conducting the study, their family members, site staff members under the supervision of the Investigator, or Sponsor employees directly involved in the study.
  • The presence of other significant dermatological conditions that could interfere with the evaluation of the study treatment's safety and efficacy.
  • Active or uncontrolled infections (e.g., sepsis, tuberculosis, hepatitis B or hepatitis C at the screening visit, human immunodeficiency virus [HIV]) that could complicate participation in the study.
  • Any medical or active psychological condition or any clinically relevant laboratory abnormality, such as, but not limited, to elevated alanine aminotransferase (ALT) or aspartate aminotransferase (AST) (> 2 × upper normal limit [ULN]) in combination with elevated bilirubin (> 1.5 × ULN), at the baseline visit.
  • History or presence of any condition that may interfere with the absorption of the IMP, including but not limited to inflammatory bowel disease, chronic or recurrent gastrointestinal disorders, history of gastric or intestinal surgery that may affect drug absorption.
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  • Inability to swallow oral medication or the presence of any condition that may impair the safe administration of oral treatments (e.g., dysphagia).
  • Evidence or history of clinically relevant cardiovascular disease, or family history of long QT syndrome.
  • Supine (after 5 min of rest) 12-lead ECG demonstrating QTc >450 msec for men and >470 msec for women, or a QRS interval >120 msec. If QTc or QRS exceed these limits, the ECG should be repeated once and the average of the two QTc or QRS values should be used to determine the participant's eligibility (The Fridericia formula (QTcF) will be calculated for the purpose of this study).
  • Has a diabetic foot ulcer.
  • Having received any of the prohibited treatments in Table 2 within the specified timeframe before inclusion.
  • Currently participating or has participated in any other clinical trial with an IMP or device within the 4 months prior to the screening visit. Prior participation in trials with KM-023 is acceptable.
  • Cutaneous infection or another underlying condition of the skin which may impact the assessments or trial participants.
  • Cutaneous infection of the assessed areas (plantar) within 2 weeks before enrollment or any infection of treatment area requiring treatment with oral, parenteral antibiotics, antivirals, antiparasitic or antifungals or any topical within 2 weeks before enrollment.
  • Pregnant or breastfeeding.
  • Known relevant allergies to any drug or excipients in the formulation.
  • Major surgery within the last 3 months, or minor surgery (requiring local anaesthetic) in the month prior to enrollment in the study, or any planned surgery during the trial.
  • Unwilling or unable to comply with all scheduled visits, treatment plan, laboratory tests and other study procedures and the restrictions described in the protocol.
  • Any other condition or previous therapy that, in the opinion of the Investigator or their designee, would render the participant unsuitable for this study, including an inability to fully comply with the study protocol requirements or a likelihood of noncompliance with study procedures.

Trial locations and contacts

2 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Israel2 sites

  • Ichilov Medical Center

    Tel Aviv

    Dr Liat Samuelov, liatsa@tlvmc.gov.il

  • Shamir Medical Center

    Tsrifin

    Dr Marina Eskin Schwartz

Other trials in Israel

About this study

Official title: Phase IIa Open Label Study to Evaluate the Safety, Tolerability, and Efficacy of Oral KM-023 in Adult Patients With Palmoplantar Epidermal Differentiation Disorders - Pachyonychia Congenita (pEDD-PC): KRT6A-pEDD-PC, KRT6B-pEDD-PC, KRT6C-pEDD-PC, KRT16-pEDD-PC and KRT17-pEDD-PC

The goal of this clinical trial is to learn if KM-023 is safe and well tolerated in adults with Pachyonychia Congenita (PC). The study will also explore whether KM-023 may help reduce pain, improve mobility, and lessen the impact of PC on participants' daily lives.

The main questions it aims to answer are:

Read more

What medical problems or side effects do participants experience when taking KM-023? Does KM-023 improve participants' ability to walk and perform physical activities? Does KM-023 reduce pain, especially pain in the soles of the feet? How does KM-023 affect the severity and impact of PC symptoms?

Researchers will evaluate changes during treatment compared with participants' condition at the start of the study.

The study will include 10 adults with genetically confirmed PC who have visible painful calluses on the soles of their feet. All participants will receive KM-023; there is no placebo group.

Each participant will be involved in the study for up to 170 days, including screening, treatment, and follow-up.

Being tested
KM-023
Medical subject headings
Pachyonychia Congenita

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • KM-023Drug

    Oral solution

Study groups in NCT07893483
Study groupRoleWhat they receive
Treatment armExperimentalNot described
Receives: Drug: KM-023

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To assess the safety of repeated doses of oral KM-023 in individuals with PC.

    The incidence, seriousness, relationship, and outcome of TEAEs.

    Measured: Day 112

  2. To assess the safety of repeated doses of oral KM-023 in individuals with PC.

    Change from baseline in clinical laboratory parameters.

    Measured: Day 112

  3. To assess the safety of repeated doses of oral KM-023 in individuals with PC.

    Change from baseline in vital signs.

    Measured: Day 112

  4. To assess the safety of repeated doses of oral KM-023 in individuals with PC.

    Change from baseline in 12-lead ECG

    Measured: Day 112

  5. To assess the safety of repeated doses of oral KM-023 in individuals with PC.

    Change from baseline in physical examination.

    Measured: Day 112

  6. To assess the tolerability of repeated doses of oral KM-023 in individuals with PC.

    Proportions of participants who completed 112 days of treatment and who discontinued treatment due to a TEAE (tolerability).

    Measured: Day 112

Secondary outcomes (10)

  1. Change from baseline to Day 112 in duration of the Timed up and Go (TUG) test completion.

    Measured: Day 112

  2. Change from baseline to Day 112 in post-TUG test pain NRS score.

    Measured: Day 112

  3. Change from baseline to Day 112 in pre-TUG test pain numeric rating scale (NRS) score.

    Measured: Day 112

  4. Change from baseline to Day 112 in the weekly average of three times a week plantar pain NRS assessment.

    Measured: Day 112

  5. Plasma and skin (when feasible) concentration of KM-023 and potentially M10 at every in-clinic visit.

    Measured: Day 112

  6. Change from baseline to Day 112 in patient reported outcomes measurement information system (PROMIS) physical function (PF) custom questionnaire score.

    Measured: Day 112

  7. Assessment of the disease characteristics of PC based on patient-reported responses to a questionnaire evaluating key disease manifestations, symptom progression, and impact on daily life.

    Measured: Day 112

  8. Change from Baseline to Day 112 in total Patient-Reported Impact of Dermatological Diseases (PRIDD) score.

    Measured: Day 112

  9. Change from Baseline to Day 112 in Clinical Global Impression Severity (CGI-S) score.

    Measured: Day 112

  10. Change from Baseline to Day 112 in Clinical Global Impression Change (CGI-C) score.

    Measured: Day 112

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Oct 2026
  2. Study startDec 2026 (estimated)
  3. Primary completionApr 2027 (estimated)
  4. Study completionNov 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07893483
Sponsor's protocol ID
KM-023-PC-202
Status last verified
Sep 2026
First submitted
6 Sep 2026
Last updated
26 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing KM-023 in people with Pachyonychia Congenita, run by Orvida Pharma (formerly Kamari Pharma) at 2 sites in Israel.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 20 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To assess the safety of repeated doses of oral KM-023 in individuals with PC. (time frame: Day 112). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07893483.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.

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