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A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa | Clinical Trials | Clareo Health

COMPLETEDPHASE2

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

A Phase 2 Open-Label Study of PTR-01 in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

NCT04599881•Phoenix Tissue Repair, Inc.

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.

Eligibility

Age

12 - No limit years

Sex

ALL

Healthy Volunteers

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

Locations (2)

Stanford University

Redwood City, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Key Dates

Start Date

October 15, 2020

Primary Completion Date

August 28, 2021

Completion Date

September 1, 2021

Last Updated

September 16, 2021

Enrollment

ACTUAL participants

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Interventions

PTR-01

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 16, 2021Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

Inclusion Criteria

Exclusion Criteria

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