A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

NCT04599881 | Completed Phase 2 FDA Drug

• 1 other identifier: PTR-01-002
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

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.

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Keywords

RDEB

Outcome Measures

Primary Outcomes (4)

• 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 162 days

• Incidence of treatment-emergent adverse events

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

  Up to 162 days

• Incidence of infusion-associated reactions

  Safety and tolerability, as assessed by infusion-associated reactions (IAR)

  Up to 162 days

• Incidence of anti-drug antibodies (ADA)

  Safety and tolerability, as assessed by immunogenicity through anti-drug antibody (ADA) testing

  Up to 162 days

Secondary Outcomes (39)

• Delivery of PTR-01 to skin

  Up to 162 days

• Formation of anchoring fibrils

  Up to 162 days

• Change in wound surface area, as assessed by wound imaging

  Up to 162 days

• Change in wound surface area, as assessed by Investigator Global Impression of Change (IGIC)

  Up to 162 days

• Change in total body wound surface area

  Up to 162 days

Other Outcomes (4)

• Genotype/phenotype relationships

  Up to 162 days

• Impact of pharmacokinetics on safety outcomes

  Up to 162 days

• Impact of pharmacokinetics on efficacy outcomes

  Up to 162 days

Study Arms (1)

PTR-01 3 mg/kg

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 every other week for a total of 7 doses.

Drug: PTR-01

Interventions

PTR-01 DRUG

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

Also known as: Recombinant collagen 7, rC7

PTR-01 3 mg/kg

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:

• Patients must meet all of the following criteria to be eligible for study participation in the three month run in period of the study:
• 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 a diagnosis of RDEB based on genetic analysis and consistent with a recessive inheritance pattern.
• Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.
• 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:

• Patients with any of the following will be excluded from participation in the study:
• 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.
• 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 patient, or may preclude the patient's

Sponsors & Collaborators

• Phoenix Tissue Repair, Inc.: lead

Study Sites (2)

Stanford University

Redwood City, California, 94063, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; 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 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: Open-label study

Study Record Dates

• First Submitted: October 8, 2020
• First Posted: October 23, 2020
• Study Start: October 15, 2020
• Primary Completion: August 28, 2021
• Study Completion: September 1, 2021
• Last Updated: September 16, 2021

Record last verified: 2021-09

Locations

US (2)