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).
This is an international, multicenter study to evaluate the safety and obtain preliminary evidence of efficacy of topically applied INM-755 (cannabinol) cream in up to 20 patients with inherited EB (Simplex, Dystrophic, Junctional, or Kindler). The study uses a within-patient, double-blind design in which matched index areas are randomized to INM-755 (cannabinol) cream or vehicle cream as a control. Selected index areas may be wounds or non-wound areas. Randomized treatments are applied daily to non-wound areas and every 1, 2, or 3 days on wounds, according to the patient's schedule for dressing changes. Treatment is over a 28-day period. EB symptoms are evaluated based on patient-reported outcomes. Wound healing is measured by digital photography. In this study designed to obtain preliminary evidence of efficacy, there is no single primary efficacy endpoint. Efficacy endpoints vary according to the presenting symptoms in each patient, which may include the presence of open wounds, wound pain associated with dressing changes, background wound pain (not procedurally linked), wound itch, and itch in non-wound areas. Net benefit from INM-755 cream is evaluated within each patient and based on their clinical needs at baseline. Enrollment is beginning with adults and may be expanded to include adolescents.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
19
topical cream containing cannabinol for dermal application
topical cream base for dermal application containing no active agent
Hopital Saint Louis APHP Paris
Paris, France
CHU Toulouse - Hopital Larrey
Toulouse, France
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Germany
Andreas Syggros Hospital of Cutaneous Venereal Diseases
Athens, Greece
Change from Baseline in Non-Wound Itch by Visual Analogue Scale (VAS)
Patients will report peak (maximal) pruritus intensity over the previous 24 hours using a 100 mm VAS (0 being "no itch" to 100 mm being "worst imaginable itch") recorded daily in the study diary. This outcome measure is applicable only for patients with non-wound index areas with eligible (moderate to severe) itch.
Time frame: Baseline to Day 29
Change from Baseline in Wound Surface Area
Wound surface area will be measured by digital photography at weekly intervals. This outcome measure is applicable only for patients with wound index areas.
Time frame: Baseline to Day 29
Change from Baseline in Procedural Wound Pain by VAS
Patients will report wound pain intensity using a 100 mm VAS (0 being "no pain" to 100 mm being "worst imaginable pain") and this will be recorded in the study diary. Procedural pain will be assessed immediately after each wound dressing, which may be every 1, 2, or 3 days according to an individual patient's schedule for dressing change. This outcome measure is applicable only for patients with wound index areas associated with eligible (moderate to severe) procedural pain.
Time frame: Baseline to Day 29
Change from Baseline in Background Wound Pain by VAS
Patients will report wound pain intensity using a 100 mm VAS (0 being "no pain" to 100 mm being "worst imaginable pain") and this will be recorded in the study diary. Peak (maximal) background pain will be assessed daily in the morning (done before the dressing change). This outcome measure is applicable only for patients with wound index areas associated with eligible (moderate to severe) background pain.
Time frame: Baseline to Day 29
Change from Baseline in Wound Itch by VAS
Patients will report peak (maximal) pruritus intensity over the previous 24 hours using a 100 mm VAS (0 being "no itch" to 100 mm being "worst imaginable itch") recorded daily in the study diary. This outcome measure is applicable only for patients with wound index areas associated with eligible (moderate to severe) itch.
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Tel Aviv Sourasky Medical Center
Tel Aviv, Israel
Fondazione IRCCS Ca Granda. Ospedale Maggiore Policlinico
Milan, Milano, Italy
Instituto Dermopatico dell'Immacolata, IDI-IRCCS
Rome, Roma, Italy
Time frame: Baseline to Day 29