First in human, Phase 1a/1b Study of KM-023 in healthy volunteers and Olmsted syndrome patients

• Research type

  Research Study

• Full 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.

• IRAS ID

  1011932

• Contact name

  Liora Braiman

• Contact email

  Liora@kamaripharma.com

• Sponsor organisation

  Kamari Pharma

• Clinicaltrials.gov Identifier

  NCT07090889

• Research summary

  This clinical trial is testing a new experimental drug, KM-023, first in healthy participants, then in Olmsted Syndrome (OS) patients.
  The first part (France) will consist of administration to groups of healthy participants of KM-023 single dose (or placebo, i.e. a dummy product), followed by repeated administrations of KM-023 (or placebo), for 5 days, to other groups. The selected participants will be hospitalized about 2 days for single dose, and about 8 days for repeated doses. Neither the participants nor the study doctor will know who will receive the study drug or the placebo.
  The trial will start with the lowest dose of KM-023 in the first group and will gradually be increased in following groups for the single dose, after safety and tolerability review by a group of experts. Similar doses will be evaluated with repeated administration. Participants will undergo a series of examinations, including taking blood samples, for safety and 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:after fasting and with a meal, to check any effect of food.
  The second part (France/UK) will be conducted in OS patients, who will take KM-023 twice daily, for a duration of 12 weeks, and will be monitored for 16 weeks. The patients will first undergo a selection visit to check if they can take part in the trial. If they are included, there will be a first full day at the hospital. The patients will receive the study drug for the first time. 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. Then, there will be five visits (two of them will be 1-day hospitalization periods) to perform safety and efficacy assessments and measure the concentrations of KM-023 in the blood. Between the visits, the clinical teams will contact the patients at home on a regular basis

• REC name

  Wales REC 1

• REC reference

  25/WA/0144

• Date of REC Opinion

  9 Jul 2025

• REC opinion

  Further Information Favourable Opinion