A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Status:
Completed
Trial end date:
2020-11-30
Target enrollment:
Participant gender:
Summary
Protocol PTR-01-001 is a Phase 1/2 study of PTR-01.
The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an
8-week Follow-up Period.
Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will
consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio.
Patients in Group 1 will receive three doses of active drug followed by 3 doses of saline
control. Patients in Group 2 will receive three doses of saline control followed by 3 doses
of active drug.
Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at
a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1
patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of
active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses.