Efficacy and Safety Study with 24-month Follow-up of Oleogel-S10 in Patients a group of inherited disorders that involve the formation of blisters following trivial trauma

• Register: REBEC
• Last refreshed on: 29 May 2023
• Main ID: RBR-4vz9c4
• Date of registration: 16/02/2018
• Prospective Registration: Yes
• Primary sponsor: Hospital da Criança Santo Antônio - Irmandade Santa Casa de Misericórdia de Porto Alegre
• Public title: Efficacy and Safety Study with 24-month Follow-up of Oleogel-S10 in Patients a group of inherited disorders that involve the formation of blisters following trivial trauma
• Scientific title: Double-blind, Randomised, Vehicle-controlled, Phase III, Efficacy and Safety Study with 24-month Open-label Follow-up of Oleogel-S10 in Patients with Inherited Epidermolysis Bullosa
• Date of first enrolment: 01/03/2018
• Recruitment status: Not yet recruiting
• URL: http://ensaiosclinicos.gov.br/rg/RBR-4vz9c4
• Study type: Intervention
• Study design: Clinical trial interventional, of treatment, parallel, two arms, double blind, controlled randomized, phase 3, control case, prospective
• Phase: 3

Countries of recruitment

Argentina, Australia, Austria, Brazil, Chile, Croatia, Czech Republic, France, Germany, Greece, Hong Kong, Ireland, Israel, Italy, Mexico, Singapore, Spain, Switzerland, United Kingdom

Contacts

• Name: Ana Elisa Bau
• Affiliation: Núcleo de Pesquisa Clínica do Hospital Pequeno Príncipe

• Name: Livia Gomes
• Affiliation: Inc Research Br Serviços de Pesquisa Clínica LTDA

Key inclusion & exclusion criteria

Inclusion criteria: Male and female patients with any subtype of inherited Epidermolyse bullosa age more than 4 years; Patients with an Epidermolyse bullosa target wound; Patient and/or his/her legal representative has/have been informed, has/have read and
understood the patient information/informed consent form, and has/have given written
informed consent; Patient and/or his/her legal representative must be able and willing to follow study
procedures and instructions.
Exclusion criteria: Epidermolyse bullosa target wound with clinical signs of local infection; Use of systemic antibiotics for wound-related infections within seven days prior to enrolment; Administration of systemic or topical steroids within 30 days before enrolment; Immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrolment; Patient has undergone stem cell transplant or gene therapy for the treatment of inherited EB; Current and/or former malignancy; Enrolment in any interventional study or treated with any investigational drug for any
disease within 4 weeks prior to study entry; 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; Pregnant or nursing women and women of childbearing potential including postmenarchal female adolescents not willing to use an effective form of birth control
(e.g., implant, injectable, combined oral contraceptive, intrauterine contraceptive device, sexual abstinence, vasectomised partner) during participation in the study (and at least 3 months thereafter); Patient is a member of the investigational team or his/her immediate family; Patient lives in the same household as a study participant

Age minimum: 4Y
Age maximum:
Gender: -

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa

Intervention(s)

Arm 1: Birch bark extract (Oleogel-S10) and a non-adhesive wound dressings, for 90 days, 82 patients
Arm 2: Vehicle (placebo) and an non-adhesive wound dressings, for 90 days, 82 patients
After this period, the patients will enter the single-arm, open-label, 24-month FU period.
For Brazil, there are 6 patients designated for site. The information with the number of participants per intervention was only reported for global data.

E02.319.267.120

Drug

N04.452.706.477

Primary Outcome(s)

Evaluation by clinical exam of the complete closure of the EB target wound within 45±7 days of treatment, comparing the usage of Oleogel-S10 and Placebo. Statistical analysis of the primary endpoint will be performed by Cui, Hung, Wang (CHW) method.

Source(s) of Monetary Support

Amryt Research Limited

Inc Research Br Serviços de Pesquisa Clínica LTDA

Secondary Sponsor(s)

Inc Research Br Serviços de Pesquisa Clínica LTDA

Amryt Research Limited