A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Not Recruiting

Trial ID: NCT03752905

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)

Stanford Investigator(s)

Jean Y. Tang MD PhD
Jean Y. Tang MD PhD

Professor of Dermatology

M. Peter Marinkovich, MD
M. Peter Marinkovich, MD

Associate Professor of Dermatology

Eligibility

Inclusion Criteria:

1. Be at least 16 years of age.
2. Has signed the current approved informed consent form.
3. Has a diagnosis of RDEB based on genetic analysis and consistent with a recessive inheritance pattern.
4. Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.
5. Has at least 1 unhealed wound 10-200 cm2 for at least 6 weeks at the Screening Visit.
6. Agrees to use contraception as follows:

* For women of childbearing potential (WOCBP) agrees to use highly effective contraceptive (including abstinence) methods from Screening, through the study, and for at least 10 weeks after the last dose of study drug. Non-childbearing potential is defined as a female who meets either of the following criteria: age ≥50 years and no menses for at least 1 year or documented hysterectomy, bilateral tubal ligation, or bilateral oophorectomy (see Section 7.4.1.2 for details on the definition of non-childbearing potential).
* For males, agrees to use a condom with any WOCBP sexual partner from Day 1 of study treatment, through the study, and at least 10 weeks after the last dose of study drug.
7. Be willing and able to comply with this protocol.

Exclusion Criteria:

1. Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.
2. Is pregnant or nursing.
3. Has received in the last six months any investigational gene therapy product or in the last three months any non-gene therapy investigational products.
4. Is anticipated to receive new regimens of antibiotics or other anti-infectives during the trial.
5. Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the patient, or may preclude the patient's successful completion of the clinical study.

Intervention(s):

drug: PTR-01

drug: Normal saline

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Melissa Barriga
650-850-1674