A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

• Status: Not yet recruiting
• Trial end date: 2021-12-31
• Target enrollment: 6
• Participant gender: All

Summary

Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Phoenix Tissue Repair, Inc.

Criteria

Inclusion Criteria:

Patients must meet all of the following criteria to be eligible for study participation in the three month run-in period of the study:

1. Willing to provide informed consent form, or if 12 to <18 years of age, legal guardian has provided informed consent form and the minor has signed an assent form acknowledging that they understand and agree to study procedures.

2. Has a diagnosis of RDEB based on genetic analysis and consistent with a recessive inheritance pattern.

3. Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.

4. 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 7.4.1.1 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.

5. Be willing and able to comply with this protocol.

Exclusion Criteria:

Patients with any of the following will be excluded from participation in the study:

1. Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.

2. Has previously had an anaphylactic reaction to PTR-01.

3. Is pregnant or nursing.

4. Has received in the last six months any investigational gene therapy product or in the last three months any non-gene therapy investigational products.

5. Is anticipated to receive new regimens of antibiotics or other anti-infectives during the trial.

6. 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.