A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

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Brief Title

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

Official Title

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

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.
    

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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Wound healing

Secondary Outcome

 Delivery of PTR-01 to skin

Condition

Recessive Dystrophic Epidermolysis Bullosa

Intervention

PTR-01

Study Arms / Comparison Groups

 PTR-01 3 mg/kg
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

6

Start Date

October 15, 2020

Completion Date

September 1, 2021

Primary Completion Date

August 28, 2021

Eligibility Criteria

        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
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04599881

Organization ID

PTR-01-002


Responsible Party

Sponsor

Study Sponsor

Phoenix Tissue Repair, Inc.


Study Sponsor

, , 


Verification Date

September 2021