A Clinical Trial which compare the safety and efficacy of a wound gel, the study treatment, or a sunflower oil-based vehicle gel in patients with Inherited Epidermolysis Bullosa (EB)

Phase 1

Active, not recruiting

• Conditions: Inherited Epidermolysis Bullosa; MedDRA version: 20.0Level: PTClassification code 10014989Term: Epidermolysis bullosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2016-002066-32-BE
• Lead Sponsor: Amryt Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-01-17
• Last Update Posted: 18 March 2020

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: All
• Target Recruitment: 192

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 <4 years may be included only after confirmation by the Independent Data Monitoring Committee (IDMC) upon review of the safety and bioanalytical data at the interim safety review stage]
2. Patients with an EB target wound (i.e., EB partial thickness wound of 10 cm2 to 50 cm2 in size aged =21 days and <9 months)
3. 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
4. Patient and/or his/her legal representative must be able and willing to follow study procedures and instructions
Are the trial subjects under 18? yes
Number of subjects for this age range: 96
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 96
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified