Recessive Dystrophic Epidermolysis Bullosa Clinical Trial
— PTR-01-001Official 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)
Verified date | March 2021 |
Source | Phoenix Tissue Repair, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 12 |
Est. completion date | November 30, 2020 |
Est. primary completion date | November 30, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Thomas Jefferson Univeristiy | Philadelphia | Pennsylvania |
United States | Stanford University | Redwood City | California |
Lead Sponsor | Collaborator |
---|---|
Phoenix Tissue Repair, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Change from Baseline in suction blister time | Change from Baseline in suction blister time (as compared to placebo and historical controls) | Baseline and Day 127 | |
Other | Change from Baseline in target wound size | Change from Baseline in target wound size (percent healing from Baseline) | Baseline and Day 127 | |
Other | Change from Baseline in healing of up to 5 chronic wounds | Change in healing of up to 5 wounds that chronically heal and reopen | Baseline and Day 127 | |
Other | Change from Baseline in wound surface area | Change from Baseline in wound surface area | Screening and Day 127 | |
Other | Change from Baseline in patient reported outcomes as assessed by the Leuven Itch Scale (LIS) | Change from Baseline in patient reported outcomes | Baseline and Day 127 | |
Other | Change from Baseline in patient reported outcomes as assessed by the pruritus-specific quality-of-life instrument ("ItchyQoL") | Change from Baseline in patient reported outcomes | Baseline and Day 127 | |
Other | Change from Baseline in patient reported outcomes as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) Questionnaire | Change from Baseline in patient reported outcomes | Baseline and Day 127 | |
Other | Change from Baseline in patient reported outcomes as assessed by the full Health Assessment Questionnaire (HAQ) | Change from Baseline in patient reported outcomes | Baseline and Day 127 | |
Other | Change from Baseline in the Investigator Global Assessment | Change from Baseline in the Investigator Global Assessment (IGA) | Screening and Day 127 | |
Other | Change from Baseline in the biochemical marker albumin | Change from Baseline in biochemical markers of disease (albumin) | Screening and Day 127 | |
Other | Change from Baseline in the biochemical marker iron | Change from Baseline in biochemical markers of disease (iron) | Screening and Day 127 | |
Other | Change from Baseline in the biochemical marker total iron binding capacity | Change from Baseline in biochemical markers of disease (total iron binding capacity) | Screening and Day 127 | |
Other | Change from Baseline in the biochemical marker hemoglobin | Change from Baseline in biochemical markers of disease (hemoglobin) | Screening and Day 127 | |
Other | Change from Baseline in the biochemical marker hematocrit | Change from Baseline in biochemical markers of disease (hematocrit) | Screening and Day 127 | |
Other | Change from Baseline in the biochemical marker total protein | Change from Baseline in biochemical markers of disease (total protein) | Screening and Day 127 | |
Primary | 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 | To measure the peak serum concentration (Cmax) of PTR-01 | Pharmacokinetic parameter estimates of Cmax | Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose | |
Secondary | To measure the time to peak concentration (Tmax) of PTR-01 | Pharmacokinetic parameter estimates of Tmax | Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose | |
Secondary | To measure the area under the curve (AUC) of PTR-01 | Pharmacokinetic parameter estimates of AUC | Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose | |
Secondary | To measure the clearance of PTR-01 | Pharmacokinetic parameter estimates of clearance | Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose | |
Secondary | To measure the half-life (t1/2) of PTR-01 | Pharmacokinetic parameter estimates of t1/2 | Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose | |
Secondary | Change from Baseline in rC7 | Change in rC7 on skin biopsy by immunofluorescence (IF) | Screening and Day 127 | |
Secondary | Change from Baseline in anchoring fibrils | Change in anchoring fibrils on skin biopsy by electron microscopy (EM) | Screening and Day 127 | |
Secondary | Duration of rC7 residence in tissue | Duration of rC7 residence in tissue by skin biopsy | Screening and Day 127 |
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