Double-blind, Vehicle-controlled, Phase I Study to Evaluate Safety and Efficacy of a 0.3% and 1% Topical Formulation of KM-001 for Management of Moderate to Severe Pruritus in Adult Patients With Lichen Simplex Chronicus (LSC)

Kamari Pharma Ltd2 sites in 1 country55 target enrollmentMay 24, 2022

InterventionsIMP Application KM-001 Chemistry Hematology Urinalysis Serelogy 12-Lead ECG Pregnancy test Blood PK sampling Physical Examination Vital signa Investigator's Global Assessment Itch Assessment via PP-NRSj E-diary data

DrugsIMP Application KM-001

Overview

Phase: Phase 1
Intervention: IMP Application KM-001
Conditions: Lichen Simplex Chronicus
Sponsor: Kamari Pharma Ltd
Enrollment: 55
Locations: 2
Primary Endpoint: Safety endpoint-Hematology blood test
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a phase 1, multi-center, randomized, vehicle-controlled, double-blinded, parallel-group study.

Approximately 6 sites will conduct the study at Germany. Approximately 61 patients (male and female) planned to be screened. 51 patient planned to be randomized.

Patients will be randomized to 1 of 3 treatment arms (KM-001 0.3%, KM-001 1%, or vehicle cream) iina a ration of 1:1:1

Patient's duration of participation will be up to 7 weeks,

a screening period with 1 visit (Visit 1) within up to 14 days (Days -14 to -1),
a 4-week treatment period with 3 visits (Visit 2 on Day 0, Visit 3 on Day 7, Visit 4 at Day 28 and 2 phone calls on Days 14 and 21, and
a 1-week follow-up period with 1 visit (Visit 5 on Day 35), as well as unscheduled visits as needed

Since KM-001 is tested in humans for the first time, the safety of KM-001 will be evaluated in a subgroup of 6 patients (sentinel group) at selected sites prior to screening of the remaining sites.

Efficacy assessments will include subjective assessments of itch and investigator assessment of the treatment effect on LSC target lesion using scoring systems.

Safety parameter (including physical examination, vital signs, ECG, standard laboratory test, and PK analysis) will be monitored from the signing of the informed consent form (ICF) until the last follow-up visit. Recording of AEs and serious AEs (SAEs) will be done throughout the study with special attention to local AEs in the treatment area (LSC target lesion, dermal safety).

Detailed Description

This phase I study will be performed in patients with LSC instead of healthy subjects as in these patients, the significant histological changes (acanthosis and parakeratosis) result in heavily altered physiology and anatomy of the skin and any data including tolerability generated by administering the IMP on healthy skin can hardly be extrapolated to patients with LSC. The setting can be compared with early studies in Psoriasis patients where it is established to include patients from the beginning. Based on preclinical experiments, no pharmacological relevant systemic absorption is expected. PK sampling will be done for confirmation. Parallel group comparison is a common method and provides optimal conditions for examining efficacy. Comparisons to a vehicle is a common procedure. Randomization provides the most reliable and impartial method of determining differences between treatments as in this case active versus vehicle. Double-blind conditions minimize any possible observer bias regarding treatment effects. A vehicle control is included to evaluate the efficacy, safety, and tolerability of the cream without the active ingredient and to differentiate whether the drug substance or the drug product might cause any effects. A duration of a 4-week treatment period is assumed to be appropriate to assess efficacy, safety, and tolerability based on preclinical data

Registry

clinicaltrials.gov

Start Date

May 24, 2022

End Date

April 4, 2023

Last Updated

2 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Kamari Pharma Ltd

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Read, understood, and signed an ICF before any investigational procedure(s) are performed.
•Male or female and aged ≥18 to 75 years at the time of screening.
•Chronic moderate to severe pruritus defined as:
•Itching that has been present for 6 weeks or more prior to the screening AND
•At the screening visit (Visit 1) and enrollment visit (Visit 2): peak pruritus-numerical rating scale (PP-NRS)24h is ≥
•Clinical diagnosis of LSC for at least 8 weeks prior to screening:
•LSC lesions defined as dry, scaly, thickened plaques of skin (lichenification) caused by repeated rubbing or scratching, on upper limbs, trunk, and/or lower limbs.
•To have no more than 3 lesions, total size not exceeding 5% of BSA from which one (target lesion) will be selected for treatment (minimum area of lesion selected for treatment: 0.5% BSA).
•Female patients of childbearing potential1 must use a highly effective birth control method(failure rate ˂1% per year when used consistently and correctly) (17) throughout the study and for at least 4 weeks after last application of IMP.
•In addition to the hormonal contraception, female patients must agree to use a supplemental barrier method during intercourse with a male partner (i.e., male condom) throughout the study and for at least 4 weeks after last application of IMP.