A Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (Cannabinol) Cream in Patients With Epidermolysis Bullosa

755-201-EB

The purpose of this study is to evaluate the safety of INM-755 (cannabinol) cream and obtain preliminary evidence of efficacy in treating symptoms and healing wounds over a 28-day period in patients with epidermolysis bullosa (EB).

2021-12-28

2023-04-03

2023-04-19

Completed

Early phase I

19

Key Inclusion Criteria: * Male or female patients aged ≥18 years with documented diagnosis of any of the following types of inherited EB: Simplex, Junctional, Dystrophic, Kindler. Enrolment will be extended to patients ≥12 years old and \<18 years old after positive opinion of a data monitoring committee after at least 4 adult patients have completed study. * Presence of at least 1 pair of well-matched index areas. Up to 2 pairs of index areas (1 pair of non-wound and 1 pair of wound index areas) can be selected in each patient * For non-wound itch index areas, both areas should: 1. Have chronic itch with a score ≥40 mm on a 100 mm VAS 2. Not exceed 20 percent of body surface area (BSA) or be less than 1 percent of BSA 3. Be similar size, up to twice the area of the smaller index area * For wound index areas, both wounds should: 1. Have a surface area ≥5 cm\^2 and ≤50 cm\^2 inclusive and be aged ≥3 weeks 2. Be well matched for size (up to twice the area of the smaller index area) and age (both either ≥3 weeks to 3 months or \>3 months) * Female patients of childbearing potential or men whose sexual partners are women of childbearing potential (WOCBP) must use highly effective birth control * WOCBP must have a negative urine pregnancy test result at baseline * Must provide written consent (or assent for patients aged \<18 years with parental/guardian consent) Key Exclusion Criteria: * EB index areas have evidence of infection * Patient has a systemic infection or used systemic antibiotics for EB-related infections within 7 days * Use of systemic corticosteroids within 30 days or of topical corticosteroids on chosen index areas within 14 days * Immunosuppressive therapy or cytotoxic chemotherapy within 60 days * Use of any high potency opioid within 30 days * Use of cannabis, cannabis extracts, or any cannabinoid products for medical or recreational use by any route of administration within 2 weeks * Prior stem cell transplant or gene therapy for EB * History of malignancy including basal cell and squamous cell carcinomas * Arterial or venous disorder resulting in ulcerated wounds * Uncontrolled diabetes mellitus * Chronic pruritus primarily attributable to pathologies or conditions other than EB * Blood transfusion to treat anemia within the past 3 months * Use of any investigational drug within 30 days or 5 half-lives (whichever is longer) * An underlying condition which places the patient at unacceptable risk * Women who are pregnant, breastfeeding (lactating), or planning to become pregnant during the study

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Within-patient randomized, double-blind comparisons of INM-755 (cannabinol cream) versus vehicle cream (control) in matched, paired index areas', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'maskingDescription': 'INM-755 (cannabinol cream) and vehicle cream (control) are identical in appearance and texture and packaged in kits labelled with unique blinded kit numbers.', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 19, 'type': 'ACTUAL'}}

2023-07-03