Trial ID or NCT#

NCT03752905

Status

recruiting iconRECRUITING

Purpose

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.

Official Title

A Phase 1/2 Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Eligibility Criteria

Ages Eligible for Study: Older than 16 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No

Investigator(s)

Jean Y. Tang MD PhD
Jean Y. Tang MD PhD
General dermatologist, Dermatologic oncologist, Cutaneous oncology specialist
Professor of Dermatology at the Stanford University Medical Center
M. Peter Marinkovich, MD
M. Peter Marinkovich, MD
Dermatologist, Blistering disease specialist, Psoriasis specialist
Associate Professor of Dermatology

Contact us to find out if this trial is right for you.

CONTACT

Melissa Barriga
(650) 850-1674