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

NCT05143190 | Completed Phase 2 FDA Drug

• 1 other identifier: PTR-01-003
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 2

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.

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Keywords

Dystrophic Epidermolysis Bullosa; Epidermolysis Bullosa; RDEB; EB

Outcome Measures

Primary Outcomes (2)

• Sustained wound healing

  Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)

  Up to 246 days

• Incidence of treatment-emergent adverse events

  Safety and tolerability, as assessed by treatment-emergent adverse events

  Up to 246 days

Secondary Outcomes (34)

• Delivery of recombinant collagen 7 (PTR-01) to skin

  Up to 246 days

• Formation of anchoring fibrils

  Up to 246 days

• Change in wound surface area

  Up to 246 days

• Change in skin integrity, as assessed by suction blister time

  Up to 246 days

• Change in skin integrity, as assessed by time to re-blistering

  Up to 246 days

Study Arms (1)

PTR-01

EXPERIMENTAL

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.

Drug: PTR-01

Interventions

PTR-01 DRUG

Intravenous recombinant collagen 7

Also known as: Recombinant collagen 7 (rC7)

PTR-01

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Has satisfactorily completed participation in PTR-01-002.
• 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.
• Be willing and able to comply with this protocol.

You may not qualify if:

• Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.
• Has previously had an anaphylactic reaction to PTR-01.
• Is pregnant or nursing.
• 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).
• Is anticipated to receive new regimens of antibiotics or other anti-infectives during the trial.
• Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the

Sponsors & Collaborators

• Phoenix Tissue Repair, Inc.: lead
• Phoenix Tissue Repair, a BridgeBio company: collaborator

Study Sites (2)

Stanford University

Redwood City, California, 94063, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Officials

• Dan Rudin, MD

  Phoenix Tissue Repair

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Masking Details: Open-label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase 2 open-label

Study Record Dates

• First Submitted: November 9, 2021
• First Posted: December 3, 2021
• Study Start: December 2, 2021
• Primary Completion: June 20, 2022
• Study Completion: August 9, 2022
• Last Updated: November 21, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)