A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

NCT ID: NCT04599881

Last Updated: 2021-09-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-10-15
• Study Completion Date: 2021-09-01

Brief 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.

Detailed Description

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.

In Part 1, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 2 in which patients will receive a dose of 3.0 mg/kg every other week for a total of 7 doses. In Part 3, patients will be followed for 12 weeks. No investigational therapy will be administered during this time. At the end of each dosing period, an efficacy assessment will be performed. Safety will be assessed continuously throughout the study.

Following the end of Part 3, patients may be eligible for a potential long-term extension to further refine the dosing regimen, depending upon study drug availability.

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PTR-01 3 mg/kg

All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg every other week for a total of 7 doses.

Group Type: EXPERIMENTAL

PTR-01

Intervention Type: DRUG

IV recombinant collagen 7 at 3 mg/kg given weekly for 4 doses, followed by bi-weekly for 7 doses

Interventions

PTR-01

IV recombinant collagen 7 at 3 mg/kg given weekly for 4 doses, followed by bi-weekly for 7 doses

Intervention Type: DRUG

Other Intervention Names

Recombinant collagen 7; rC7

Eligibility 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.

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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Phoenix Tissue Repair, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Stanford University

Redwood City, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Countries

United States

Other Identifiers

PTR-01-002

Identifier Type: -

Identifier Source: org_study_id