Inherited Epidermolysis Bullosa MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Episalvan gel
Product Name: Oleogel-S10
Pharmaceutical Form: Gel
INN or Proposed INN: Birch bark extract
CAS Number: 1640971
Sponsors
Amryt Research Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: A patient will be eligible for study participation only if all of the following criteria apply: 1. Male and female patients aged =4 years with the following subtypes of inherited EB: JEB, DEB, and Kindler syndrome [Note: Children =21 days old and =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: A patient will not be eligible to participate in this study if any of the following criteria apply: 1. Patient has EB subtype EBS 2. EB target wound that is > 9 months old or has clinical signs of local infection 3. Use of systemic antibiotics for wound-related infections within 7 days prior to enrolment 4. Administration of systemic or topical steroids (except for inhaled, ophthalmic or topical applications, such as budesonide suspension for oesophageal strictures [e.g., Pulmicort Respules® 0.25 mg/2 mL or 0.5 mg/2 mL]) within 30 days before enrolment 5. Immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrolment 6. Patient has undergone stem cell transplant or gene therapy for the treatment of inherited EB 7. Current and/or former malignancy including basal cell carcinomas and squamous cell carcinomas 8. Enrolment in any interventional study or treated with any investigational drug for any disease within 4 weeks prior to study entry 9. Factors present in the patient and/or his/her legal representative that could interfere with study compliance such as inability to attend scheduled study visits or compliance with home dressing changes 10. Pregnant or nursing women 11. Women of childbearing potential, including postmenarchal female adolescents, and men who are not willing to use an effective form of birth control with failure rates <1% per year (e.g., implant, injectable, combined oral contraceptive, intrauterine contraceptive device, sexual abstinence, vasectomy or vasectomised partner) during participation in the study (and at least 3 months thereafter) 12.Patient is a member of the investigational team or his/her immediate family 13.Patient lives in the same household as a study participant.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the double-blind phase is to compare the efficacy of Oleogel-S10 (treatment arm A) with vehicle (treatment arm B) in the promotion of healing of EB partial thickness wounds. This will be assessed as evidenced by the incidence of the first complete closure of the EB target wound (defined as EB partial thickness wound of 10 cm2 to 50 cm2 in size aged =21 days and <9 months) in patients with inherited EB (subtypes JEB, DEB, or Kindler syndrome) within 45±7 days of treatment. ; Secondary Objective: 1/ Compare the efficacy of IMP with placebo as evidenced by several criteria described in the protocol 2/ Compare the safety of IMP with placebo as evidenced by the incidence, severity, and relatedness of AEs and based on laboratory assessments 3/ Compare the tolerability of IMP with placebo 4/ Assess betulin exposure ;Timepoint(s) of evaluation of this end point: See section E.5.1; Primary end point(s): Proportion of patients with first complete closure of the EB target wound (defined as EB partial thickness wound of 10 cm2 to 50 cm2 in size aged =21 days and < 9 months) in patients with inherited EB (subtypes JEB, DEB, or Kindler syndrome) within 45±7 days of treatment with Oleogel S10 compared to vehicle based on clinical assessment by the investigator (the wound will be rated as "closed" at first appearance of complete reepithelialisation without drainage confirmed by a second observation after 7 days [+2 days]) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints • Time to first complete closure of the EB target wound as evidenced by clinical assessment until EDBP (D90±7) • Proportion of patients with first complete closure of the EB target wound at D14±5, D30±7, D60±7, and D90±7 based on clinical assessment by the investigator • Proportion of patients with first complete closure of the EB target wound at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7 based on patient assessment • Proportion of patients with first complete closure of the EB target wound at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7 based on blinded evaluation of photographs • Percentage change from baseline (DBP D0) in EB target wound size as evidenced by blinded evaluation of photographs taken at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7 • Change from baseline (DBP D0) in total body wound burden as evidenced by clinical assessment using Section I (assessment of the skin except for the anogenital region) of the 'EB Disease Activity and Scarring Index' (EBDASI) at D30±7, D60±7, and D90±7 • Change from baseline (DBP D0) in body surface area percentage (BSAP) of TBSA affected by EB partial thickness wounds as evidenced by clinical assessment based on the 'Lund and Browder' chart at D30±7, D60±7, and D90±7 • The incidence and maximum severity of wound infection between baseline (DBP D0) and D90±7 as evidenced by AEs and/or use of topical and/or systemic antibiotics (related to wound infection) • Change from baseline (DBP D0) in "background" pain using the 'Face, Legs, Activity, Cry, Consolability' (FLACC) pain rating scale in patients <4 years of age and the 'Wong Baker FACES® Pain Rating Scale' in patients =4 years of age before wound dressing changes at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7 • Chang | — |
Countries
Australia, Austria, Belgium, Croatia, Czech Republic, Denmark, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Mexico, Russian Federation, Serbia, Spain, Switzerland, Turkey, Ukraine, United Kingdom
Contacts
Public ContactDerval O'Carroll
Amryt Research Limited
Outcome results
None listed