Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)

NCT ID: NCT05143190

Last Updated: 2022-11-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-02
• Study Completion Date: 2022-08-09

Brief Summary

A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.

Detailed Description

Protocol PTR-01-003 is a 4-part Phase 2, open-label study of PTR-01 in patients who satisfactorily completed study PTR-01-002 and meet current enrollment criteria.

In Part 1, patients will be monitored monthly for up to 2 months with patient-reported and Investigator assessments. In Part 2, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 3 in which patients will receive a dose of 3.0 mg/kg monthly for a total of 5 additional doses. At the end of each dosing period, efficacy assessments will be performed. During Part 4, patients will be evaluated at Months 1 and 3 after completion of dosing to assess the durability of wound healing and other efficacy parameters. Safety will be assessed continuously throughout the study.

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

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 once monthly for a total of 5 doses.

Group Type: EXPERIMENTAL

PTR-01

Intervention Type: DRUG

Intravenous recombinant collagen 7

Interventions

PTR-01

Intravenous recombinant collagen 7

Intervention Type: DRUG

Other Intervention Names

Recombinant collagen 7 (rC7)

Eligibility Criteria

Inclusion Criteria

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 satisfactorily completed participation in PTR-01-002.

3. 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.

4. 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. 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 (other than PTR-01).

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

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

Sponsors

Phoenix Tissue Repair, a BridgeBio company

UNKNOWN

Sponsor Role: collaborator

Phoenix Tissue Repair, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Dan Rudin, MD

Role: STUDY_DIRECTOR

Phoenix Tissue Repair

Locations

Stanford University

Redwood City, California, United States

Columbia University Irving Medical Center

New York, New York, United States

Countries

United States

Other Identifiers

PTR-01-003

Identifier Type: -

Identifier Source: org_study_id