A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

  • STATUS
    Recruiting
  • End date
    Dec 31, 2021
  • participants needed
    6
  • sponsor
    Phoenix Tissue Repair, Inc.
Updated on 26 January 2021

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.

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.

Details
Condition Epidermolysis Bullosa Dystrophica
Treatment PTR-01
Clinical Study IdentifierNCT04599881
SponsorPhoenix Tissue Repair, Inc.
Last Modified on26 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age greater than or equal to 12 yrs?
Gender: Male or Female
Do you have Epidermolysis Bullosa Dystrophica?
Do you have any of these conditions: Do you have Epidermolysis Bullosa Dystrophica??
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

Exclusion Criteria

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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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