epidermolysis bullosa
Conditions
Interventions
KOI2 1mg/kg drip infusion administration for 30 minutes. First week
once per day for four days, second- to forth-week
twice per week (at least once in three to four days).
Sponsors
Osaka University Hospital
Toho University Omori Medical Center
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients understands the protocol and agrees to provide written informed consent to participate in the study on a voluntary basis. For patient under 20-year old, informed consent of voluntary participation should be obtained by a legal guardian. Informed assent is collected, if possible, with a patient more than 7-year old. (2) Patients who are confirmed at the present day or before to satisfy following item number 1, and at least one item out of the following number 2 to 5. 1. Blister, erosion, and/or ulcer formations induced by daily minor trauma have been continued on the skin and oral mucosa from beginning of the birth or from 0~6 year old. 2. Existence of patient(s) suffering from dystrophic epidermolysis bullosa in the family member. 3. Intradermal blister formation right beneath the basement membrane has been confirmed by histological study. 4. Decrease or absence of type VII collagen at the cutaneous basement membrane zone has been confirmed by immunofluorescent study. 5. Mutation of type VII collagene gene (COL7A1) has been identified. (3) Total area of vesicles, erosions and ulcers is more than 20 cm2 at the timing of obtaining informed consent. The size of each vesicle, erosion and/or ulcer is measured by length x width using a vernier caliper (Large size can be measured by a ruler). The sum of the areas for the whole body is calculated, excluding those under 2 mm by length. (4) Patient agrees to take appropriate birth control measures from day 1 through 1 month after the last drug administration (including condom, birth control by female partner, and/or others). (5) A woman of childbearing potential, agrees to use a highly effective method of contraception from screening visit through 1 month after the last drug administration.
Exclusion criteria
Exclusion criteria: Epidermolysis bullosa other than malnutrition type (2) Urine pregnancy test is positive (female only) at screening visit (3) Breast feeding female patient (4) HIV, HBs, HCV positive at screening (5) A subject who participated in other clinical trials within 90 days and received a clinical trial drug (including a placebo) (6) A subject who is participating in any other clinical trials. (7) A subject whose liver dysfunction data match the predefined exclusion standard (8) A subject who has acute worsening of epidermolysis bullosa (such as tracheal/esophageal stenosis, acute nephritis) (9) Ineligible by other reasons judged by the investigators
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change rate of total area of blisters, erosions and ulcers compared to those of base line | — |
Countries
Japan
Contacts
Public ContactYoko Kawamura
Osaka University Hospital Department of Medical Innovation
Outcome results
None listed