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

JRCT ID: jRCT2031220378

Registered date:14/10/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: dystrophic epidermolysis bullosa
• Date of first enrollment: 28/10/2022
• Target sample size: 3
• Study type: Interventional
• Intervention(s): 1.0 mg/kg of S-005151 is administered intravenously

Outcome(s)

• Primary Outcome: closure of intractable ulcer

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both

Include criteria

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.

Exclude criteria

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.

Related Information

• Primary Sponsor: Juan Carlos Gomez