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A phase 2, multicenter, open-label, uncontrolled study of S-005151 in patients with dystrophic epidermolysis bullosa to evaluate the efficacy for intractable ulcers

A phase 2 study of S-005151 in patients with dystrophic epidermolysis bullosa to evaluate the efficacy for intractable ulcers

Juan Carlos Gomez

Shionogi & Co., Ltd.

1-8, Doshomachi 3-chome, Chuo-ku, Osaka, Osaka

shionogiclintrials-admin@shionogi.co.jp

Corporate Communications Department

Shionogi & Co., Ltd.

1-8, Doshomachi 3-chome, Chuo-ku, Osaka, Osaka

+81-6-6209-7885

shionogiclintrials-admin@shionogi.co.jp

Recruiting

Oct. 28, 2022

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

Patients with dystrophic epidermolysis bullosa who currently meet or previously met 1 and at least one of 2 to 5 below.
1. Patients who have easily developed and continue to have blisters, erosions, and ulcers in the skin and oral mucosa due to minor mechanical irritation from immediately after birth or around the age of 0 to 6 years.
2. Patients who have patients with dystrophic epidermolysis bullosa in the family.
3. Patients with blisters formed in the dermis just below the basement membrane by histological examination of the initial site of blister.
4. Patients with decreased /disappeared type VII collagen in the basement membrane of the skin observed by immunofluorescent staining in the non-bullous area.
5. Patients with type VII collagen gene (COL7A1) mutation by genetic diagnosis.
Patients with dystrophic epidermolysis bullosa who have intractable ulcers that have not closed for 12 weeks or more at the time of enrollment, have an area of 4 cm2 or more, and have not seen an area reduction of 50% or more during the pre-observation period compared to Visit 1.

Patients with epidermolysis bullosa other than dystrophic.
Patients who newly received long-term systemic administration of steroids or immunosuppressants due to complications of epidermolysis bullosa or other reasons from informed consent to starting administration.

Both

dystrophic epidermolysis bullosa

1.0 mg/kg of S-005151 is administered intravenously

closure of intractable ulcer

+81-3-3762-4151

No

none