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

NCT03752905 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PTR-01-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

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.

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Keywords

RDEB

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events

  The primary endpoint of this study is safety and tolerability, as assessed by treatment-emergent adverse events, infusion-associated reactions (IAR) and immunogenicity

  Up to Day 127

Secondary Outcomes (8)

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

  Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose

• To measure the time to peak concentration (Tmax) of PTR-01

  Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose

• To measure the area under the curve (AUC) of PTR-01

  Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose

• To measure the clearance of PTR-01

  Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose

• To measure the half-life (t1/2) of PTR-01

  Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose

Other Outcomes (15)

• Change from Baseline in suction blister time

  Baseline and Day 127

• Change from Baseline in target wound size

  Baseline and Day 127

• Change from Baseline in healing of up to 5 chronic wounds

  Baseline and Day 127

Study Arms (5)

PTR-01 0.1 mg/kg

EXPERIMENTAL

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

Drug: PTR-01

PTR-01 0.3 mg/kg

EXPERIMENTAL

Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart.

Drug: PTR-01

PTR-01 1.0 mg/kg

EXPERIMENTAL

Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart.

Drug: PTR-01

Normal Saline

PLACEBO COMPARATOR

Saline control to mimic PTR-01.

Drug: Normal saline

PTR-01 3.0 mg/kg

EXPERIMENTAL

Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart.

Drug: PTR-01

Interventions

PTR-01 DRUG

Recombinant human collagen 7 (rC7)

Also known as: Recombinant human collagen 7 (rC7)

PTR-01 0.1 mg/kg; PTR-01 0.3 mg/kg; PTR-01 1.0 mg/kg; PTR-01 3.0 mg/kg

Normal saline DRUG

Saline control

Normal Saline

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Be at least 16 years of age.
• Has signed the current approved informed consent form.
• 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.
• Has at least 1 unhealed wound 10-200 cm2 for at least 6 weeks at the Screening Visit.
• 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.
• Be willing and able to comply with this protocol.

You may not qualify if:

• Has known systemic hypersensitivity to any of the inactive ingredients in 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 successful completion of the clinical study.

Sponsors & Collaborators

• Phoenix Tissue Repair, Inc.: lead

Study Sites (3)

Stanford University

Redwood City, California, 94063, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Thomas Jefferson Univeristiy

Philadelphia, Pennsylvania, 19107, United States

Location

Related Links

• Sponsor website

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Interventions

Saline Solution

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

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Theresa Podrebarac, MD

  Phoenix Tissue Repair

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Single-blind
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center

Study Record Dates

• First Submitted: November 19, 2018
• First Posted: November 26, 2018
• Study Start: January 9, 2019
• Primary Completion: November 30, 2020
• Study Completion: November 30, 2020
• Last Updated: March 9, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)