Status:
COMPLETED
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Lead Sponsor:
Phoenix Tissue Repair, Inc.
Conditions:
Recessive Dystrophic Epidermolysis Bullosa
Eligibility:
All Genders
16+ years
Phase:
PHASE1
PHASE2
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 ...
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 e...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
January 9 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 30 2020
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT03752905
Start Date
January 9 2019
End Date
November 30 2020
Last Update
March 9 2021
Active Locations (3)
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1
Stanford University
Redwood City, California, United States, 94063
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
Thomas Jefferson Univeristiy
Philadelphia, Pennsylvania, United States, 19107