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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03752905
Other study ID # PTR-01-001
Secondary ID
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
Start date January 9, 2019
Est. completion date November 30, 2020

Study information

Verified date March 2021
Source Phoenix Tissue Repair, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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 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).


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
PTR-01
Recombinant human collagen 7 (rC7)
Normal saline
Saline control

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Phoenix Tissue Repair, Inc.

Country where clinical trial is conducted

United States, 

Outcome

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
See also
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Not yet recruiting NCT03632265 - Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Phase 3
Recruiting NCT05944250 - A Pilot Study to Evaluate a Temporary Skin Substitute (Spincare® Matrix) for Wound Healing in RDEB Patients N/A
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Not yet recruiting NCT04285294 - Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
Active, not recruiting NCT04213261 - A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa Phase 3
Active, not recruiting NCT02323789 - Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa Phase 1/Phase 2
Recruiting NCT01874769 - Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) N/A
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Terminated NCT02984085 - Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. Phase 1/Phase 2
Completed NCT04227106 - Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 3
Completed NCT02698735 - Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients Phase 1/Phase 2
Completed NCT03012191 - Gentamicin for RDEB Phase 1/Phase 2
Completed NCT03529877 - Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa Phase 1/Phase 2
Completed NCT05143190 - Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01) Phase 2
Recruiting NCT04177498 - Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC Early Phase 1
Completed NCT04491604 - Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB Phase 3

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