A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

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Brief Title

A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Official Title

A Phase 1/2 Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Brief Summary

      Protocol PTR-01-001 is a Phase 1/2 study of PTR-01.

      The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an
      8-week Follow-up Period.

      Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will
      consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio.
      Patients in Group 1 will receive three doses of active drug followed by 3 doses of saline
      control. Patients in Group 2 will receive three doses of saline control followed by 3 doses
      of active drug.

      Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at
      a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1
      patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of
      active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses.
    

Detailed Description

      Protocol PTR-01-001 is a saline-controlled, single and repeat dose, dose-escalation,
      crossover study designed to determine the safety, tolerability, tissue kinetics,
      pharmacodynamics and preliminary efficacy of PTR 01.

      The study is divided into three periods: an up to 4-week Screening Period, a 10-week
      Treatment Period and an 8-week Follow-up Period. During the Screening Period and Follow-up
      Period there will be no study drug treatment.

      During the Treatment Period a total of 3 doses of PTR-01 and 3 doses of saline control will
      be administered to patients for a total of 6 doses over a 10-week period in three cohorts
      dosed at 0.1, 0.3, 1.0 and 3.0 mg/kg (active drug). Twelve patients with a diagnosis of RDEB
      and a history of at least one chronic wound will be enrolled. Those patients who do not have
      documentation of genetic analysis and IF staining will have blood for genetic analysis and a
      biopsy for IF staining prior to enrollment (both required).

      Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will
      consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio.
      Patients will receive doses 2 weeks apart. Patients in Group 1 will receive three doses of
      active drug followed by 3 doses of saline control. Patients in Group 2 will receive three
      doses of saline control followed by 3 doses of active drug. This cross-over design will yield
      a total of 14 patients all of whom will receive active drug and saline control.

      Prior to randomization, patients will complete a Screening Period to assess the extent and
      impact of skin disease involvement and the chronicity of at least one wound. Only patients
      who meet all of the eligibility criteria will be randomized for treatment.

      Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at
      a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1
      patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of
      active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses. After the last
      patient in Cohort 1 has received their third dose and safety labs for all patients have been
      reviewed by the Data Safety Monitoring Board (DSMB), the next cohort may be enrolled. This
      same schedule and safety review process will be followed for all subsequent dosing cohorts,
      with Cohort 2, Cohort 3 and Cohort 4 receiving 0.3, 1.0 and 3.0 mg/kg respectively.

      Efficacy assessments will be performed prior to first dose of therapy (at the end of the
      Screening Period), after the last dose of study drug in Period 1, after the last dose of
      study drug in Period 2 of the Treatment Period and 2 weeks (Day 85) after the last dose of
      study drug (at the end of the Follow-up Period).
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Incidence of treatment-emergent adverse events

Secondary Outcome

 To measure the peak serum concentration (Cmax) of PTR-01

Condition

Recessive Dystrophic Epidermolysis Bullosa

Intervention

PTR-01

Study Arms / Comparison Groups

 PTR-01 0.1 mg/kg
Description:  Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart.

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

12

Start Date

January 9, 2019

Completion Date

November 30, 2020

Primary Completion Date

November 30, 2020

Eligibility Criteria

        Inclusion Criteria:

          1. Be at least 16 years of age.

          2. Has signed the current approved informed consent form.

          3. Has a diagnosis of RDEB based on genetic analysis and consistent with a recessive
             inheritance pattern.

          4. Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.

          5. Has at least 1 unhealed wound 10-200 cm2 for at least 6 weeks at the Screening Visit.

          6. 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 (see Section 7.4.1.2 for details on the
                  definition of non-childbearing potential).

               -  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.

          7. Be willing and able to comply with this protocol.

        Exclusion Criteria:

          1. Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.

          2. Is pregnant or nursing.

          3. Has received in the last six months any investigational gene therapy product or in the
             last three months any non-gene therapy investigational products.

          4. Is anticipated to receive new regimens of antibiotics or other anti-infectives during
             the trial.

          5. 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 successful completion of the clinical study.
      

Gender

All

Ages

16 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Theresa Podrebarac, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03752905

Organization ID

PTR-01-001


Responsible Party

Sponsor

Study Sponsor

Phoenix Tissue Repair, Inc.


Study Sponsor

Theresa Podrebarac, MD, Study Director, Phoenix Tissue Repair


Verification Date

March 2021