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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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22 March 2021 |
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Main ID: |
NCT03752905 |
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Date of registration:
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19/11/2018 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
PTR-01-001 |
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Scientific title:
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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) |
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Date of first enrolment:
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January 9, 2019 |
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Target sample size:
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12 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT03752905 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Theresa Podrebarac, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Phoenix Tissue Repair |
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Key inclusion & exclusion criteria
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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.
Age minimum:
16 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Recessive Dystrophic Epidermolysis Bullosa
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Intervention(s)
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Drug: PTR-01
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Drug: Normal saline
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Primary Outcome(s)
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Incidence of treatment-emergent adverse events
[Time Frame: Up to Day 127]
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Secondary Outcome(s)
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To measure the area under the curve (AUC) of PTR-01
[Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose]
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To measure the half-life (t1/2) of PTR-01
[Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose]
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To measure the peak serum concentration (Cmax) of PTR-01
[Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose]
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To measure the time to peak concentration (Tmax) of PTR-01
[Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose]
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Change from Baseline in anchoring fibrils
[Time Frame: Screening and Day 127]
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To measure the clearance of PTR-01
[Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose]
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Change from Baseline in rC7
[Time Frame: Screening and Day 127]
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Duration of rC7 residence in tissue
[Time Frame: Screening and Day 127]
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Secondary ID(s)
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PTR-01-001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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