Olmsted SyndromePhase 1NCT07090889
Study of KM-023 in Healthy Volunteers and Patients With Olmsted Syndrome.
A Phase 1 study testing KM-023 in people with Olmsted Syndrome, run by Orvida Pharma (formerly Kamari Pharma) at 4 sites in 3 countries.
Trial summary
The trial is conducted in order to assess the safety of KM-023 and to observe what effects, both beneficial and adverse, it has on the body, and the way the body absorbs and eliminates KM-023, first in healthy participants, then in Olmsted Syndrome patients. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Olmsted Syndrome
- What is being tested?
- KM-023
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 8 Jul 2025to 21 Nov 2026
- Phase
- Phase 1
- 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 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Part A
- Healthy female and male participants.
- Female participants of non-childbearing potential, childbearing potential and males capable of fathering a child must meet the contraception requirements.
- Non-smoking and no use of any tobacco or nicotine products (by declaration) for a period of at least 1 month prior to screening visit.
- Not using prescription medication 14 days prior to admission; and 7 days prior to admission for over the counter (OTC) medication/vitamins/supplements.
- Must not be taking any medication that could potentially impair hepatic or renal function at the time of screening.
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- No history of alcohol or other drugs of abuse.
- Body Mass Index (BMI) of 18.0 to 32.0 kg/m2 (inclusive); and a total body weight >50.0 kg (110 lbs) and <100.0 kg (220 lbs) at screening and Day -1.
- No vaccines given within 14 days of dosing.
- 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.
- Part B
- Read, understood, and signed an ICF before any investigational procedure(s) are performed.
- Clinical diagnosis of OS, with confirmed TRPV3 or PERP mutation, and visible palmoplantar keratoderma.
- Stable medical condition over the previous 1 month.
- Willingness and ability to comply with the treatment and follow-up visits.
- Willingness to refrain from current treatments, in compliance with the list of prohibited medications.
- No contraindications to study medication as determined by the Investigator.
- Female participants of non-childbearing potential, childbearing potential and males capable of fathering a child must meet the contraception requirements.
- Negative serum pregnancy test at screening, and negative urinary pregnancy test at Day -1.
Who cannot join
- Part A
- Participants who are staff members at the investigational site directly involved in conducting the study.
- Evidence or history of clinically relevant hepatic, hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease.
- History of regular alcohol consumption exceeding 14 drinks/week for females or 21 drinks/week for males within 6 months of screening.
- Screening supine BP ≥145 mm Hg (systolic) or ≥90 mm Hg (diastolic)
- Participants with any abnormalities in clinical laboratory tests, and serology results, at screening, considered by the study physician as clinically significant.
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- Pregnant or breastfeeding female participants.
- Participants who donated blood or received blood or plasma derivatives in the 30 days preceding study drug administration.
- Dosed in another clinical trial within at least 10 tissue half-lives prior to dosing or 4 months.
- Known relevant allergy to any drug or excipients in the formulation
- Participants with any acute medical situation (e.g., acute infection) within 48h of screening or start of dosing, which is considered of significance by the Investigator.
- Major surgery within the last 3 months 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.
- Part B
- 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 abnormalities, such as, but not limited, to elevated ALT or AST (> 3 × ULN) in combination with elevated bilirubin (> 2 × ULN), at the screening/baseline visit.
- Diabetic foot ulcers.
- Patient is unwilling to refrain from using prohibited medications during the clinical trial.
- Currently participating or participated in any other clinical trial of an IMP or device, within the past 4 months before the screening visit.
- Cutaneous infection or another underlying condition of the skin which may impact the assessments or trial participants.
- Cutaneous infection of the area to be treated with IMP within 2 weeks before the screening visit or any infection of treatment area requiring treatment with oral, parenteral antibiotics, antivirals, antiparasitics or antifungals or any topical within 2 weeks before the screening visit.
- Pregnant or breastfeeding patient.
- Having received any of the prohibited treatments in Table 2 within the specified timeframe before inclusion.
- Known relevant allergy to any drug or excipients in the formulation.
- Major surgery within the last 3 months, or minor surgery (requiring local anesthetic) during last 1 month, 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 patient 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
4 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 3 countries
France2 sites, 2 recruiting
- Recruiting
Paris Descartes Necker Cochin, Hôpital Necker-Enfants Malades Bat Imagine RDC
Paris
FALL Magatte, (+33) 01 44 49 45 33, magatte.fall@aphp.fr
- Recruiting
Biotrial
Rennes
Dr Sophie Hays, +33 2 99 59 91 91, caroline.lohier@biotrial.com
United States1 site, 1 recruiting
- Recruiting
Northwestern University, Department of Dermatology
Chicago, Illinois, 60611
Stephanie Rangel Rangel, 312-503-5942, stephanie.rangel@northwestern.edu
Amy Paller, MS, MD
United Kingdom1 site, 1 recruiting
- Recruiting
Royal London Hospital
London
Mary Natividad, +447821666833, m.natividad@nhs.net
Edel O'Toole
About this study
Official title: A Combined Single and Multiple Ascending Dose Phase 1a/1b, Double-blind, Placebo-Controlled, and Food-effect Study to Evaluate the Safety and Pharmacokinetics of Oral KM-023 in Healthy Participants. Followed by a 3-month Open Label Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Oral KM-023 in Olmsted Syndrome Patients
The trial is conducted in order to assess the safety of KM-023 and to observe what effects, both beneficial and adverse, it has on the body, and the way the body absorbs and eliminates KM-023, first in healthy participants, then in Olmsted Syndrome patients. In addition, the study will examine the presence of KM-023 in the different layers of the skin. Finally, the efficacy of KM-023 will be investigated in Olmsted Syndrome patients.
The first part of this trial will be conducted with healthy participants in France, then after its completion, the second part of the trial will be conducted with Olmsted Syndrome patients in France and the United Kingdom.
Read more
The first part of the trial is itself divided in two parts: first, a series of single doses will be given to groups of healthy participants, then other groups of healthy participants will be given repeated doses of KM-023 twice daily for 5 days.
The participants will be hospitalized approximately 2 days for the investigation of single doses, and approximately 8 days for the investigation of the multiple doses.
Some participants will receive the KM-023, and other will receive a placebo, which is a non-active product.
Four (4) different dose levels will be given in the trial part studying single administration, and 3 different dose levels in the trial part studying repeated administrations. The trial will start with the lowest doses, and the dose levels will be gradually increased after the safety and tolerability of the previous dose level has been assessed by a group of experts (including physicians). In order to assess the safety, a series of examinations will be conducted, including taking blood samples. Blood samples will also be collected a regular timepoints in order to evaluate the changes in concentration of KM-023 in blood over time. One group of participants will receive a single dose of KM-023 twice: once after fasting, and once with a meal, to check any effect of food on the concentration of KM-023 in blood.
In the second part of the trial, Olmsted Syndrome patients will take KM-023 twice daily, for a duration of 12 weeks.
There will be a first hospitalization day, during which the patients will receive the study drug, and will be taught how to self-administer KM-023 at home during the ambulatory periods. Safety and efficacy tests will be performed, and the concentration of KM-023 in blood will be monitored. Patients will then go back home with their treatment, to be taken every day. After the first hospitalization day, there will be five visits (two of them will be 1-day hospitalization periods), to monitor the safety and tolerability, the efficacy, and the concentration of KM-023 in blood. Between the hospitalization visits, the clinical teams will call the patients at home on a regular basis to check the safety and tolerability.
The duration of participation, including the selection period and the follow-up period, is a maximum 36 days when only one dose is given, 51 days when the dose is given on two occasions to assess the effect of food, 40 days when treatment is given twice daily for 5 days, and 18 weeks in the case of Olmsted Syndrome patients.
- Conditions
- Olmsted Syndrome
- Being tested
- KM-023, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KM-023Drug
solution for oral administration
PlaceboDrug
solution for oral administration
| Study group | Role | What they receive |
|---|---|---|
| Part A SAD (Single Ascending Dose) | Active comparator | Up to 4 cohorts of 8 healthy participants Receives: Drug: KM-023; Drug: Placebo |
| Part A MAD (Multiple Ascending Dose) | Active comparator | 3 cohorts of 8 healthy participants. Receives: Drug: KM-023; Drug: Placebo |
| Part B- OS patients | Active comparator | up to 8 participants with OS, receiving KM-023 for 12 weeks. Receives: Drug: KM-023 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and other safety-related events.
Measured: Part A- SAD(Single Ascending Dose) day 8, MAD (Multiple Ascending Dose) day 12 Part B- Day 112.
Secondary outcomes (7)
Reduction in target plantar callus thickness from baseline to visit at Week 12. (Part B only)
Measured: 12 weeks
Reduction in target plantar callus size from baseline to visit at Week 12. (Part B only)
Measured: 12 weeks
Clearance of the lesions, assessed by change from baseline to W4, 8 and 12 in Investigator Global Assessment (IGA) score. (Part B only)
Measured: 12 weeks
Peak Plasma Concentration (Cmax)
Peak plasma concentration (Cmax) of the study drug.
Measured: Part A - SAD (Single Ascending Dose) Day 8; MAD (Multiple Ascending Dose) Day 12; Part B - Day 112.
Time to Reach Peak Plasma Concentration (Tmax)
Time to reach maximum observed plasma concentration (Tmax) of the study drug.
Measured: Part A - SAD Day 8; MAD Day 12; Part B - Day 112.
Area Under the Plasma Concentration-Time Curve (AUC)
Area under the plasma concentration-time curve (AUC) of the study drug.
Measured: Part A - SAD Day 8; MAD Day 12; Part B - Day 112.
Dose Proportionality of Pharmacokinetic Parameters
Dose proportionality assessment of pharmacokinetic parameters (Cmax and AUC) within the administered dose range.
Measured: Part A - SAD Day 8; MAD Day 12; Part B - Day 112.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2025
- Study start8 Jul 2025
- Primary completion21 Nov 2026 (estimated)
- Study completion21 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Olmsted SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- The study includes two parts: Part A- a Single Ascending Dose (SAD) part followed by a Multiple Ascending Dose (MAD) part, Placebo- controlled Part B- Open Label
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Orvida Pharma (formerly Kamari Pharma)
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07090889
- Sponsor's protocol ID
- KM-023-101
- Status last verified
- Jul 2025
- First submitted
- 8 May 2025
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Combined Single and Multiple Ascending Dose Phase 1a/1b, Double-blind, Placebo-Controlled, and Food-effect Study to Evaluate the Safety and Pharmacokinetics of Oral KM-023 in Healthy Participants. Followed by a 3-month Open Label Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Oral KM-023 in Olmsted Syndrome Patients
- ClinicalTrials.gov ID
- NCT07090889
- Acronym / sponsor ID
- KM-023-101
- Conditions
- Olmsted Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- Orvida Pharma (formerly Kamari Pharma)Research network
- Study start
- 8 Jul 2025 (actual)
- Primary completion
- 21 Nov 2026 (estimated)
- Study completion
- 21 Nov 2026 (estimated)
- First posted
- 29 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The trial is conducted in order to assess the safety of KM-023 and to observe what effects, both beneficial and adverse, it has on the body, and the way the body absorbs and eliminates KM-023, first in healthy participants, then in Olmsted Syndrome patients. In addition, the study will examine the presence of KM-023 in the different layers of the skin. Finally, the efficacy of KM-023 will be investigated in Olmsted Syndrome patients.
The first part of this trial will be conducted with healthy participants in France, then after its completion, the second part of the trial will be conducted with Olmsted Syndrome patients in France and the United Kingdom.
The first part of the trial is itself divided in two parts: first, a series of single doses will be given to groups of healthy participants, then other groups of healthy participants will be given repeated doses of KM-023 twice daily for 5 days.
The participants will be hospitalized approximately 2 days for the investigation of single doses, and approximately 8 days for the investigation of the multiple doses.
Some participants will receive the KM-023, and other will receive a placebo, which is a non-active product.
Four (4) different dose levels will be given in the trial part studying single administration, and 3 different dose levels in the trial part studying repeated administrations. The trial will start with the lowest doses, and the dose levels will be gradually increased after the safety and tolerability of the previous dose level has been assessed by a group of experts (including physicians). In order to assess the safety, a series of examinations will be conducted, including taking blood samples. Blood samples will also be collected a regular timepoints in order to evaluate the changes in concentration of KM-023 in blood over time. One group of participants will receive a single dose of KM-023 twice: once after fasting, and once with a meal, to check any effect of food on the concentration of KM-023 in blood.
In the second part of the trial, Olmsted Syndrome patients will take KM-023 twice daily, for a duration of 12 weeks.
There will be a first hospitalization day, during which the patients will receive the study drug, and will be taught how to self-administer KM-023 at home during the ambulatory periods. Safety and efficacy tests will be performed, and the concentration of KM-023 in blood will be monitored. Patients will then go back home with their treatment, to be taken every day. After the first hospitalization day, there will be five visits (two of them will be 1-day hospitalization periods), to monitor the safety and tolerability, the efficacy, and the concentration of KM-023 in blood. Between the hospitalization visits, the clinical teams will call the patients at home on a regular basis to check the safety and tolerability.
The duration of participation, including the selection period and the follow-up period, is a maximum 36 days when only one dose is given, 51 days when the dose is given on two occasions to assess the effect of food, 40 days when treatment is given twice daily for 5 days, and 18 weeks in the case of Olmsted Syndrome patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- The study includes two parts: Part A- a Single Ascending Dose (SAD) part followed by a Multiple Ascending Dose (MAD) part, Placebo- controlled Part B- Open Label
| Arm | Type | Receives |
|---|---|---|
| Part A SAD (Single Ascending Dose) | Active comparator | Drug: KM-023, Drug: PlaceboUp to 4 cohorts of 8 healthy participants |
| Part A MAD (Multiple Ascending Dose) | Active comparator | Drug: KM-023, Drug: Placebo3 cohorts of 8 healthy participants. |
| Part B- OS patients | Active comparator | Drug: KM-023up to 8 participants with OS, receiving KM-023 for 12 weeks. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (19 listed)
- Part A
- Healthy female and male participants.
- Female participants of non-childbearing potential, childbearing potential and males capable of fathering a child must meet the contraception requirements.
- Non-smoking and no use of any tobacco or nicotine products (by declaration) for a period of at least 1 month prior to screening visit.
- Not using prescription medication 14 days prior to admission; and 7 days prior to admission for over the counter (OTC) medication/vitamins/supplements.
Key exclusion criteria (29 listed)
- Part A
- Participants who are staff members at the investigational site directly involved in conducting the study.
- Evidence or history of clinically relevant hepatic, hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease.
- History of regular alcohol consumption exceeding 14 drinks/week for females or 21 drinks/week for males within 6 months of screening.
- Screening supine BP ≥145 mm Hg (systolic) or ≥90 mm Hg (diastolic)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KM-023solution for oral administration | Drug | Part A MAD (Multiple Ascending Dose), Part A SAD (Single Ascending Dose), Part B- OS patients |
| Placebosolution for oral administration | Drug | Part A MAD (Multiple Ascending Dose), Part A SAD (Single Ascending Dose) |
Primary outcome
Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and other safety-related events.
Time frame: Part A- SAD(Single Ascending Dose) day 8, MAD (Multiple Ascending Dose) day 12 Part B- Day 112.
7 secondary outcomes
Reduction in target plantar callus thickness from baseline to visit at Week 12. (Part B only)
Time frame: 12 weeks
Reduction in target plantar callus size from baseline to visit at Week 12. (Part B only)
Time frame: 12 weeks
Clearance of the lesions, assessed by change from baseline to W4, 8 and 12 in Investigator Global Assessment (IGA) score. (Part B only)
Time frame: 12 weeks
Peak Plasma Concentration (Cmax)
Time frame: Part A - SAD (Single Ascending Dose) Day 8; MAD (Multiple Ascending Dose) Day 12; Part B - Day 112.
Time to Reach Peak Plasma Concentration (Tmax)
Time frame: Part A - SAD Day 8; MAD Day 12; Part B - Day 112.
Area Under the Plasma Concentration-Time Curve (AUC)
Time frame: Part A - SAD Day 8; MAD Day 12; Part B - Day 112.
Dose Proportionality of Pharmacokinetic Parameters
Time frame: Part A - SAD Day 8; MAD Day 12; Part B - Day 112.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Northwestern University, Department of Dermatology
Chicago, United States
Paris Descartes Necker Cochin, Hôpital Necker-Enfants Malades Bat Imagine RDC
Paris, France
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing KM-023 in people with Olmsted Syndrome, run by Orvida Pharma (formerly Kamari Pharma) at 4 sites in 3 countries.
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 19 inclusion and 29 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: looking for participants now, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France, United States, United Kingdom. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and other safety-related events. (time frame: Part A- SAD(Single Ascending Dose) day 8, MAD (Multiple Ascending Dose) day 12 Part B- Day 112.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 May 2026.