Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||16 Years and Over|
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.
- - 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.
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.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
|Phase 1/Phase 2|
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|Phoenix Tissue Repair, Inc.|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Theresa Podrebarac, MD|
|Principal Investigator Affiliation||Phoenix Tissue Repair|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
|Overall Status||Active, not recruiting|
The disease, disorder, syndrome, illness, or injury that is being studied.
|Recessive Dystrophic Epidermolysis Bullosa|
|Study Website:||View Trial Website|
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).
Experimental: PTR-01 0.1 mg/kg
Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart.
Experimental: PTR-01 0.3 mg/kg
Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart.
Experimental: PTR-01 1.0 mg/kg
Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart.
Placebo Comparator: Normal Saline
Saline control to mimic PTR-01.
Experimental: PTR-01 3.0 mg/kg
Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart.
Drug: - PTR-01
Recombinant human collagen 7 (rC7)
Drug: - Normal saline
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
Redwood City, California, 94063
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Aurora, Colorado, 80045
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Philadelphia, Pennsylvania, 19107