epidermise sheet cultured from autologous revertant somatic mosaic site

Phase 1

• Conditions: epidermolytic ichthyosis

• Registration Number: JPRN-jRCTb041190097
• Lead Sponsor: Akiyama Masashi

Brief Summary

In this study, cultured epidermal sheets prepared from the revertant site of patients who were diagnosed with epidermolytic ichthyosis and had revertant mosaicism were transplanted to the hyperkeratotic lesions. The primary endpoints, "hyperkeratosis improvement rates at 4 weeks after transplantation," in the three subjects were 39.52%, 100.0%, and 100.0%. The secondary endpoints, "hyperkeratosis improvement rates 24 weeks after transplantation" were 0%, 30.37% and 25.30%.

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-12-19
• Study Completion: 2022-03-31
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Patients who are diagnosed with epidermolytic ichthyosis and have a revertant somatic mutation, the responsible gene mutation * was identified by genetic testing.
* Heterozygote mutation must be demonstrated in either keratin 1 (KRT1) or keratin 10 (KRT10).

Exclusion Criteria

Patients hypersensitive to penicillin, kanamycin, streptomycin, and amphotericin B antibiotics. Patients with a history of hypersensitivity to penicillin antibiotics and aminoglycoside antibiotics.
Patients who are allergic to cattle, mice and pigs.
Patients with malignant neoplastic lesions other than the skin, or patients with a history within 3 years prior to enrollment.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Improvement rate of hyperkeratosis 4 weeks after the final transplant (%)

Secondary Outcome Measures

Name	Time	Method
Improvement rate of site-specific hyperkeratosis 4 weeks after final transplantation (%)<br>Average improvement rate of hyperkeratosis up to 24 weeks after initial transplantation (%)<br>Average improvement rate of site-specific hyperkeratosis up to 24 weeks after initial transplantation (%)