NCT04644627: GENTELBULL

Topical Gentamicin Nonsense Suppression Therapy of EB

Completed Phase 1, PHASE2 Last updated 3 May 2022

What this trial tests

Phase 1, PHASE2 trial testing Gentamicin Sulfate in Epidermolysis Bullosa in 4 participants. Completed in 1 May 2022.

Timeline

1 December 2020

Primary endpoint
11 January 2022

1 May 2022

Quick facts

Lead sponsor	Oslo University Hospital
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	1 December 2020
Primary completion	11 January 2022
Estimated completion	1 May 2022
Sites	1 location across Norway

Drugs / interventions tested

• Gentamicin Sulfate — full drug profile →

Conditions studied

• Epidermolysis Bullosa — all drugs for Epidermolysis Bullosa →

Sponsor

Oslo University Hospital

Who can join

Eligibility, any sex, with Epidermolysis Bullosa. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The overall purpose of this study is to address whether topical gentamicin therapy is an effective and feasible treatment. Specifically, we will investigate the effect of non-intensive treatment (once daily or every other day) on skin protein expression, as well as quantify the effect on wound healing in patients with EB caused by PSC (part A). Furthermore, we will address in vitro whether gentamicin restores protein expression of genes affected by SSM in fibroblasts derived from skin biopsies obtained from patients with EB caused by SSM (part B). If these in vitro experiments yield positive results, the patients donating the cells will be offered to enter part A of this study. The overall duration of part A in this study is planned to be 18 weeks per patients and consists of a 6 weeks treatment period followed by a 12 week follow up period. Each patient will attend 3 study visits: at week 0, week 6 and week 18. All patients will be included within a time period of 12 months. The overall duration of part B will be up to 8 weeks per patients of which 4-7 weeks are spent to prepare fibroblasts obtained from skin biopsies. Then 5 days of in vitro intervention and subsequent analysis follows. Altogether, the duration of the GENTELBULL study will be 78 weeks or less.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa.
   Welponer T, Prodinger C, Pinon-Hofbauer J, Hintersteininger A, et al · · 2021 · cited 14× · PMID 34110606 · DOI 10.1007/s13555-021-00561-5
2. Stairways to Advanced Therapies for Epidermolysis Bullosa.
   De Rosa L, Enzo E, Palamenghi M, Sercia L, et al · · 2023 · cited 12× · PMID 36167646 · DOI 10.1101/cshperspect.a041229
3. Challenges in Treating Genodermatoses: New Therapies at the Horizon.
   Morren MA, Legius E, Giuliano F, Hadj-Rabia S, et al · · 2021 · cited 10× · PMID 35069188 · DOI 10.3389/fphar.2021.746664