Recessive Dystrophic Epidermolysis BullosaPhase 1/2PTR-01-001NCT03752905
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
A Phase 1/2 study testing PTR-01 and Normal saline in people with Recessive Dystrophic Epidermolysis Bullosa, run by Phoenix Tissue Repair, Inc. at 3 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- PTR-01 and Normal saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 3 sitesUnited States
- Study dates
- 9 Jan 2019to 30 Nov 2020
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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:
Show all 9Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Thomas Jefferson Univeristiy
Philadelphia, Pennsylvania, 19107
Stanford University
Redwood City, California, 94063
About this study
Official 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)
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.
Read more
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.
Read the 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 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).
- Being tested
- PTR-01, Normal saline
- Keywords
- RDEB
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTR-01Drug
Also called Recombinant human collagen 7 (rC7)
Recombinant human collagen 7 (rC7)
Normal salineDrug
Saline control
| Study group | Role | What they receive |
|---|---|---|
| PTR-01 0.1 mg/kg | Experimental | Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart. Receives: Drug: PTR-01 |
| PTR-01 0.3 mg/kg | Experimental | Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart. Receives: Drug: PTR-01 |
| PTR-01 1.0 mg/kg | Experimental | Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart. Receives: Drug: PTR-01 |
| Normal Saline | Placebo comparator | Saline control to mimic PTR-01. Receives: Drug: Normal saline |
| PTR-01 3.0 mg/kg | Experimental | Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart. Receives: Drug: PTR-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to Day 127
Secondary outcomes (8)
To measure the peak serum concentration (Cmax) of PTR-01
Pharmacokinetic parameter estimates of Cmax
Measured: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the time to peak concentration (Tmax) of PTR-01
Pharmacokinetic parameter estimates of Tmax
Measured: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the area under the curve (AUC) of PTR-01
Pharmacokinetic parameter estimates of AUC
Measured: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the clearance of PTR-01
Pharmacokinetic parameter estimates of clearance
Measured: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the half-life (t1/2) of PTR-01
Pharmacokinetic parameter estimates of t1/2
Measured: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
Change from Baseline in rC7
Change in rC7 on skin biopsy by immunofluorescence (IF)
Measured: Screening and Day 127
Change from Baseline in anchoring fibrils
Change in anchoring fibrils on skin biopsy by electron microscopy (EM)
Measured: Screening and Day 127
Duration of rC7 residence in tissue
Duration of rC7 residence in tissue by skin biopsy
Measured: Screening and Day 127
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2018
- Study start9 Jan 2019
- Primary completion30 Nov 2020
- Study completion30 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recessive Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Phoenix Tissue Repair, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Theresa Podrebarac, MD, Study director, Phoenix Tissue Repair
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03752905
- Sponsor's protocol ID
- PTR-01-001
- Acronym
- PTR-01-001
- Status last verified
- Mar 2021
- First submitted
- 19 Nov 2018
- Last updated
- 9 Mar 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There are currently no plans to share individual participant data with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT03752905
- Acronym / sponsor ID
- PTR-01-001 / PTR-01-001
- Keywords
- RDEB
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Phoenix Tissue Repair, Inc.Industry (pharma, biotech, devices)
- Study start
- 9 Jan 2019 (actual)
- Primary completion
- 30 Nov 2020 (actual)
- Study completion
- 30 Nov 2020 (actual)
- First posted
- 26 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
- Model description
- Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center
| Arm | Type | Receives |
|---|---|---|
| PTR-01 0.1 mg/kg | Experimental | Drug: PTR-01Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart. |
| PTR-01 0.3 mg/kg | Experimental | Drug: PTR-01Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart. |
| PTR-01 1.0 mg/kg | Experimental | Drug: PTR-01Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart. |
| Normal Saline | Placebo comparator | Drug: Normal salineSaline control to mimic PTR-01. |
| PTR-01 3.0 mg/kg | Experimental | Drug: PTR-01Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PTR-01Also: Recombinant human collagen 7 (rC7)Recombinant human collagen 7 (rC7) | Drug | PTR-01 0.1 mg/kg, PTR-01 0.3 mg/kg, PTR-01 1.0 mg/kg, PTR-01 3.0 mg/kg |
| Normal salineSaline control | Drug | Normal Saline |
Primary outcome
Incidence of treatment-emergent adverse events
Time frame: Up to Day 127
The primary endpoint of this study is safety and tolerability, as assessed by treatment-emergent adverse events, infusion-associated reactions (IAR) and immunogenicity
8 secondary outcomes
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
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
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
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
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
Change from Baseline in rC7
Time frame: Screening and Day 127
Change from Baseline in anchoring fibrils
Time frame: Screening and Day 127
Duration of rC7 residence in tissue
Time frame: Screening and Day 127
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2021.
- Sponsor websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No. There are currently no plans to share individual participant data with other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Theresa Podrebarac, MD study director
Phoenix Tissue Repair
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PTR-01 and Normal saline in people with Recessive Dystrophic Epidermolysis Bullosa, run by Phoenix Tissue Repair, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (time frame: Up to Day 127). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2021.