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Treatment of epidermolytic ichthyosis using epidermise sheet (Jace) cultured from autologous revertant somatic mosaic site (epidermise sheet cultured from autologous revertant somatic mosaic site)

epidermise sheet cultured from autologous revertant somatic mosaic site (epidermise sheet cultured from autologous revertant somatic mosaic site)

Kodera Yasuhiro

Mar. 31, 2022

3

Subjects satisfying all the following criteria: (1)Those who have been diagnosed with epidermolytic ichthyosis by the investigators and have revertant mosaicism. (2)Causative gene mutations of epidermolytic ichthyosis and revertant mosaicism have been confirmed by genetic analysis . (3)Those who have one or more sites of hyperkeratosis that regenerative medicine products should be transplanted. (4)Those who have received sufficient informed consent. (5)Over 20 years old at the time of consent acquisition.

Consent holders (3): screening failures (0), registrants (3), discontinuation (0), Complate (3). Of the 3 registrants, full analysis set (3), per protocol set (3), the observation phase safety analysis target (3), and the treatment phase safety analysis target population (3).

Overall adverse events 10, 3 (100.0%): application site erosion 1, 1 (33.3%), wound bleeding 1, 1 (33.3%), wound complications 5, 2 (66.7%), skin ulcers 1, 1 (33.3%), skin bacterial infections 2, 2 (66.7%), and serious illnesses were 0, 0 (0.0%). Causal relationship with regenerative medicine product can be denied in either case.

The primary endpoint, "hyperkeratosis improvement rate at 4 weeks after transplantation," in the three subjects was 39.52%, 100.0%, and 100.0%, respectively. On the other hand, the secondary endpoints "hyperkeratosis improvement rate 24 weeks after transplantation" were 0%, 30.37% and 25.30%, respectively.

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%.

https://jrct.mhlw.go.jp/latest-detail/jRCTb041190097

Akiyama Masashi

Nagoya University Graduate School of Medicine

65 Tsurumai-cho, Showa-ku, Nagoya-city, Aichi

makiyama@med.nagoya-u.ac.jp

Takahashi Hironobu

Nagoya University Hospital

65 Tsurumai-cho, Showa-ku, Nagoya-city, Aichi

+81-52-744-2479

Iga-shinsa@adm.nagoya-u.ac.jp

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

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).

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.

Both

epidermolytic ichthyosis

Transplantation of cultured epidermis sheet

C564367

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

Improvement rate of site-specific hyperkeratosis 4 weeks after final transplantation (%)
Average improvement rate of hyperkeratosis up to 24 weeks after initial transplantation (%)
Average improvement rate of site-specific hyperkeratosis up to 24 weeks after initial transplantation (%)

Complete

+81-52-744-2479

Jan. 13, 2021