A study using a currently licensed treatment for psoriasis which is expected to reduce the blistering symptoms of severe EBS in adults and children.

 

Dr Christine Chiaverini works at Centre Hospitalier Universitaire de Nice, France on this project to begin the repurposing of tablets that are currently used to treat psoriasis. Twenty people, six years of age or older, whose severe EBS causes at least four new blisters per day, will be offered apremilast tablets. The study will last twenty weeks for each person: after an initial screening they will take the tablets for eight weeks, stop for four weeks then take the tablets again for a further eight weeks. Outcomes such as blistering, pain, itch and quality of life will be measured during periods with and without treatment and compared. Positive results will support the next step of a placebo controlled clinical trial.

Contents:

• Project summary (top of page)
• About our funding
• Latest progress summary
• About our researchers
• Why this research is important
• Researcher's abstract
• Researcher's progress update

 

About our funding:

Research leader	Dr Christine Chiaverini
Institution	Archet 2 Hôpital, Centre Hospitalier Universitaire de Nice, France
Type of EB	EBS
Patient involvement	Twenty people, six years and older with EBS
Funding amount	€157,670
Project length	2 years
Start date	TBC 2023
DEBRA internal ID	GR000008

 

Latest progress summary:

(due 2024)

About our researchers:

Dr Christine Chiaverini is a dermatologist at the Archet 2 Hôpital, Nice and published preliminary results on treating EBS with apremilast in 2019.

Co-researchers: Dr Emmanuelle Bourrat; Prof Christine Bodemer, Dr Christine Labreze-Hautier, Prof Juliette Mazereeuw.

 

Why this research is important:

The purpose of this study is to evaluate the efficacy and tolerance of apremilast, an immunomodulatory therapy already used for psoriasis and Becet’s disease, to treat patients with severe EBS over the age of 6 years.

Christine Chiaverini

Researcher's abstract:

Grant title: GEBULO. A 20-Week Multicentre, Open Study Assessing the Efficacy and Safety of Apremilast (Otezla®) in Patients > 6 years of age with EB simplex generalized.

Epidermolysis bullosa simplex is characterized by skin and mucosa fragility. The severe
form (EBS-sev) is associated with significant morbidity and alteration of quality of life, both
in children and adults. There is currently no specific treatment for EBS. Recently, we have
shown that patients with EBS-sev have clinical hallmarks of inflammatory skin disease
and that a specific inflammatory pathway, the Th17 immune response, seems to be
involved in the onset of skin lesions. Based on these data, we treated with apremilast (a
treatment for psoriasis with anti TH17 effect) 4 adults with EBS-sev. A dramatic and long
lasting decrease in the number of blisters was observed, with acceptable tolerance for 3
patients.
We therefore propose to perform a 20-week multicenter, open study assessing the
efficacy and safety of apremilast in patients ≥ 6 years of age with EBS-sev.
We planned to include 20 patients with an dominant KRT5/14 EBS-sev and at least 4
new blisters a day.
The primary objective is to describe efficacy of apremilast over the 3 periods of a
challenge-dechallenge-rechallenge design like study in these patients.
Secondary objectives are to describe
- the safety of apremilast treatment over the study period
- the evolution of efficacy and health outcomes measures within each study period
The study consists of 4 periods:
- period 1: Screening period
- period 2: First treatment period (Week 0 to 8).
- period 3 : No treatment period (Week 8 to 12).
- period 4 : Second treatment period f(Week 12 to 20).

 

Return to contents

 

Researcher's progress update:

Due 2024