TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA

Phase 1

• Conditions: Epidermolysis bullosa caused by nonsense mutations or splice site mutations; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2020-002337-15-NO
• Lead Sponsor: Oslo University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-06-29
• Last Update Posted: 9 May 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Part A 1.The patient has EB caused by nonsense mutation 2.The patient has a symmetrical distribution of wounds which allows for wounds on one side of the body to serve as control wounds, while study therapy is applied to the other side. 3.The patient (if an adult) or parent(s)/guardian(s) is capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
Part B 1.The patient has EB caused by splice site mutations 2.The patient (if an adult) or parent(s)/guardian(s) is capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Part A 1.Known contact allergy against gentamicin sulfate or other ingredients in the ointment 2.Known intolerance to gentamicin sulfate of any sort 3.Moderate or severely reduced kidney function (eGFR <30) 4.Use other experimental therapy against EB 5.Receiving systemic aminoglycosides during the last 3 months
Part B 1.The patient also carries a nonsense mutation that can be the cause of EB

Study & Design

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