Epidermolysis bullosa MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
InMed Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Individuals must meet all of the following criteria to be included in the study: 1. Male or female patients aged =18 years with documented diagnosis of any of the following types of inherited EB: • EB simplex • Junctional EB • Dystrophic EB • Kindler EB Enrolment will be extended to patients =12 years old and 3 months c. not located at anatomical sites with high likelihood of accidental trauma (e.g., knees, elbows) 3. Female patients of childbearing potential or men whose sexual partners are women of childbearing potential (WOCBP) must be able and willing to use at least 1 method of contraception until the patient’s last study visit. The following are permitted contraceptive methods: intrauterine device, hormonal contraceptives (e.g., oral, patch, or injectable), male vasectomy (if vasectomy was medically confirmed). Abstinence from heterosexual intercourse is acceptable when this is the usual lifestyle of the patient. A female patient is considered not of childbearing potential when postmenopausal (at least 12 consecutive months without menses without an alternative medical cause) or otherwise permanently sterile (for which acceptable methods include hysterectomy, bilateral salpingectomy, or bilateral oophorectomy with or without hysterecto
Exclusion criteria
Exclusion criteria: Individuals meeting any of the following criteria at Screening or Baseline are ineligible to participate in this study: 1. EB index areas have evidence of infection. 2. Patient has evidence of a systemic infection or has used systemic antibiotics for EB-related infections within 7 days before the Baseline Visit. 3. Administration of systemic corticosteroids within 30 days or of topical (except ophthalmic) corticosteroids on chosen index areas within 14 days before the Baseline Visit. Corticosteroids for inhalation or intranasal use are permitted provided they are taken at a stable dosing. 4. Immunosuppressive therapy or cytotoxic chemotherapy within 60 days before the Baseline Visit. 5. Use of any high potency opioid within 30 days prior to Baseline Visit. 6. Use of cannabis, cannabis extracts, or any cannabinoid products for medical or recreational use by any route of administration within 2 weeks prior to the Baseline Visit. The patient can be using the cannabinoid-containing product at Screening but must commit to avoiding cannabinoid use in the prescribed timeframe. 7. Patient has undergone stem cell transplant or gene therapy for the treatment of inherited EB. 8. History of malignancy including basal cell carcinomas and squamous cell carcinomas. 9. Arterial or venous disorder resulting in ulcerated wounds. 10. Uncontrolled diabetes mellitus. 11. Presence of chronic pruritus believed to be primarily attributable to concomitant pathologies or conditions other than EB (e.g., renal or cholestatic pathologies). 12. Patient has received blood transfusion to treat anaemia within the past 3 months. 13. Patient has received any investigational drug within 30 days or 5 half-lives (whichever is longer) before the Baseline Visit. 14. Patient has an underlying condition which in the opinion of the investigator places the patient at unacceptable risk. 15. Women who are pregnant, breastfeeding (lactating), or planning to become pregnant during the study. 16. Patient is considered by the investigator, for any reason, to be an unsuitable candidate for the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To evaluate the safety of INM-755 cream in patients with EB. - To obtain preliminary evidence of efficacy of INM-755 cream on wound and non-wound affected skin areas in patients with EB.;Secondary Objective: - To assess systemic exposure to cannabinol in a subset of patients.;Primary end point(s): The primary safety endpoints are: • Incidence of treated index areas with erythema, oedema, scaling, and stinging/burning identified from local tolerability assessment (LTA) by type of treatment and severity • Treatment-emergent local adverse event (AE) incidence, severity, and relationship with treatment;Timepoint(s) of evaluation of this end point: Through out the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Other safety endpoints are: • Treatment-emergent systemic AE incidence, severity, and relationship with treatment • Change from Baseline (CFB) in vital signs • Incidence of treatment-emergent abnormal physical examination findings Efficacy Endpoints Preliminary evidence of efficacy will be evaluated based on the following endpoints as applicable: Non-wound Itch: • CFB in weekly average non-wound itch severity summarised from daily assessments by the patient using a 100 mm visual analogue scale (VAS) • Proportion of non-wound itch index areas achieving a 20 mm, 30 mm, or >30 mm reduction of itch VAS from Baseline to End of Treatment (EOT) • Patient's Impression of Change of Non-wound Itch (PIC-I) as assessed by the Dynamic Pruritus Scale (DPS) at each index area after 2 weeks and 4 weeks of treatment Wound Surface Area: • Absolute and percent CFB in surface area of each index wound at the scheduled visits • Incidence of at least 25%, 50%, 75%, and 100% reduction in surface area from Baseline and incidence of first complete closure (defined as complete re-epithelialisation without drainage) of each index wound at the scheduled visits • Time to achieve 50% reduction from Baseline in surface area of each index wound • Time to achieve complete closure of each index wound Procedural Wound Pain: • CFB in weekly average index wound pain score associated with dressing change (procedural pain) summarised from assessments by the patient using a 100 mm VAS • Proportion of index wounds with associated procedural pain achieving a 20 mm, 30 mm, or >30 mm reduction of pain VAS from Baseline to EOT • Patient’s Impression of Change-Procedural Pain (PIC-PP) for each wound index area after 2 weeks and 4 weeks of treatment Background Wound Pain: • CFB in weekly average index wound pain score not associated with dressing change (background pain) summarised from daily assessments by the patient using a 100 mm VAS • Proportion of index wou | — |
Countries
Austria, France, Germany, Greece, Israel, Italy, Serbia
Contacts
Public ContactClinical Research Executive
InMed Pharmaceuticals Inc.
Outcome results
None listed