Recessive Dystrophic Epidermolysis BullosaPhase 2NCT05143190
Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)
A Phase 2 study testing PTR-01 in people with Recessive Dystrophic Epidermolysis Bullosa, run by Phoenix Tissue Repair, Inc. at 2 sites in United States.
Trial summary
A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- PTR-01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 2 sitesUnited States
- Study dates
- 2 Dec 2021to 9 Aug 2022
- Phase
- Phase 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willing to provide informed consent form, or if 12 to <18 years of age, legal guardian has provided informed consent form and the minor has signed an assent form acknowledging that they understand and agree to study procedures.
- Has satisfactorily completed participation in PTR-01-002.
- 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.
- 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.
- Has previously had an anaphylactic reaction to 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 (other than PTR-01).
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Columbia University Irving Medical Center
New York, New York, 10032
Stanford University
Redwood City, California, 94063
About this study
Official title: A Phase 2b Open-Label Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01 in Study PTR-01-002
A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.
Read the detailed description
Protocol PTR-01-003 is a 4-part Phase 2, open-label study of PTR-01 in patients who satisfactorily completed study PTR-01-002 and meet current enrollment criteria.
In Part 1, patients will be monitored monthly for up to 2 months with patient-reported and Investigator assessments. In Part 2, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 3 in which patients will receive a dose of 3.0 mg/kg monthly for a total of 5 additional doses. At the end of each dosing period, efficacy assessments will be performed. During Part 4, patients will be evaluated at Months 1 and 3 after completion of dosing to assess the durability of wound healing and other efficacy parameters. Safety will be assessed continuously throughout the study.
- Being tested
- PTR-01
- Keywords
- Dystrophic Epidermolysis Bullosa, Epidermolysis Bullosa, RDEB, EB
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTR-01Drug
Also called Recombinant collagen 7 (rC7)
Intravenous recombinant collagen 7
| Study group | Role | What they receive |
|---|---|---|
| PTR-01 | Experimental | All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg once monthly for a total of 5 doses. Receives: Drug: PTR-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sustained wound healing
Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)
Measured: Up to 246 days
Incidence of treatment-emergent adverse events
Safety and tolerability, as assessed by treatment-emergent adverse events
Measured: Up to 246 days
Secondary outcomes (34)
Delivery of recombinant collagen 7 (PTR-01) to skin
Amount of recombinant collagen 7 (PTR-01) incorporation to skin as compared to normal human skin assessed by immunofluorescence using NC1 \& NC2 antibody staining
Measured: Up to 246 days
Formation of anchoring fibrils
Formation of new anchoring fibrils as measured by electron microscopy
Measured: Up to 246 days
Change in wound surface area
Wound surface area of lesions as assessed by medical photography using the Canfield RUBI 3D imaging system
Measured: Up to 246 days
Change in skin integrity, as assessed by suction blister time
Change in skin integrity, as assessed by suction blister time
Measured: Up to 246 days
Change in skin integrity, as assessed by time to re-blistering
Change in skin integrity, as assessed by time to re-blistering
Measured: Up to 246 days
Change in itch severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains
Severity of itch, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains, maximum score of 5 (worst)
Measured: Up to 246 days
Change in the impact of itch on quality of life
Change in the impact of itch on quality of life, as assessed by the Pruritus-Specific Quality of Life Instrument (ItchyQoL), maximum score of 110 (worst)
Measured: Up to 246 days
Change in pain severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) pain domains
Change in pain severity, as assessed by Patient-Reported Outcome Measurement Information System (PROMIS) pain domains, maximum score of 5 (worst)
Measured: Up to 246 days
Change in pain severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)
Change in pain severity, as assessed by the Instrument for Scoring Clinical, maximum score of 234 (worst)
Measured: Up to 246 days
Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire
Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire, maximum score is 40 (worst)
Measured: Up to 246 days
Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument
Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument, maximum score of 40 (worst)
Measured: Up to 246 days
Change in corneal symptoms
Change of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI), maximum score of 100 (worst)
Measured: Up to 246 days
Stabilization of corneal symptoms
Stabilization of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI), maximum score of 100 (worst)
Measured: Up to 246 days
Rate of change in nutritional markers (hemoglobin)
Change of nutritional markers, as assessed by hemoglobin
Measured: Up to 246 days
Rate of change in nutritional markers (hematocrit)
Change of nutritional markers, as assessed by hematocrit
Measured: Up to 246 days
Rate of change in nutritional markers (total protein/albumin)
Change of nutritional markers, as assessed by total protein/albumin
Measured: Up to 246 days
Rate of change in nutritional markers (Fe/TIBC)
Change of nutritional markers, as assessed by total Fe/TIBC
Measured: Up to 246 days
Rate of change in nutritional markers (C-reactive protein)
Change of nutritional markers, as assessed by total C-reactive protein
Measured: Up to 246 days
Change in Investigator Global Impressions of Change (IGIC)
Global impressions of change, as assessed through IGIC (1-7), 7 being worst
Measured: Up to 246 days
Change in Investigator Patient Impressions of Change (PGIC)
Global impressions of change, as assessed through PGIC (1-7), 7 being worst
Measured: Up to 246 days
Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire maximum score is 67 (worst)
Measured: Up to 246 days
Change in overall health
Change in overall disability, as assessed by the Health Assessment Questionnaire or Children's Health Assessment Questionnaire (HAQ/CHAQ) maximum score is 3 (worst)
Measured: Up to 246 days
Change in mental health
Change in mental health, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) mental health domains, maximum score is 5 (worst)
Measured: Up to 246 days
Change in social function
Change in social functioning, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) social function domains, maximum score is 4 (worst)
Measured: Up to 246 days
Change in amount of wound care
Change in amount of wound care, as assessed by patient interviews
Measured: Up to 246 days
Change in time for wound care
Change in time for wound care, as assessed by patient interviews
Measured: Up to 246 days
Change in cost of wound care
Change in cost of wound care, as assessed by patient interviews
Measured: Up to 246 days
Change in overall patient impression of quality of life
Change in overall anecdotal quality of life, as assessed by one-on-one patient interviews
Measured: Up to 246 days
Change in overall patient impression of disability
Change in overall anecdotal disability, as assessed by one-on-one patient interviews
Measured: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Cmax
Correlate ADA with Cmax
Measured: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Tmax
Correlate ADA with Tmax
Measured: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Area Under the Curve (AUC)
Correlate ADA with Area Under the Curve (AUC)
Measured: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of clearance of PTR-01
Correlate ADA with clearance of PTR-01
Measured: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of PTR-01 half-life
Correlate ADA with PTR-01 half-life
Measured: Up to 246 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2021
- Study start2 Dec 2021
- Primary completion20 Jun 2022
- Study completion9 Aug 2022
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Phase 2 open-label
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Phoenix Tissue Repair, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Phoenix Tissue Repair, a BridgeBio company
- Responsible party
- Sponsor
- Investigators
- Dan Rudin, MD, Study director, Phoenix Tissue Repair
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05143190
- Sponsor's protocol ID
- PTR-01-003
- Status last verified
- Nov 2022
- First submitted
- 9 Nov 2021
- Last updated
- 21 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b Open-Label Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01 in Study PTR-01-002
- ClinicalTrials.gov ID
- NCT05143190
- Acronym / sponsor ID
- PTR-01-003
- Keywords
- Dystrophic Epidermolysis Bullosa, Epidermolysis Bullosa, RDEB, EB
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Phoenix Tissue Repair, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Phoenix Tissue Repair, a BridgeBio company
- Study start
- 2 Dec 2021 (actual)
- Primary completion
- 20 Jun 2022 (actual)
- Study completion
- 9 Aug 2022 (actual)
- First posted
- 3 Dec 2021 (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.
A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.
Detailed description
Protocol PTR-01-003 is a 4-part Phase 2, open-label study of PTR-01 in patients who satisfactorily completed study PTR-01-002 and meet current enrollment criteria.
In Part 1, patients will be monitored monthly for up to 2 months with patient-reported and Investigator assessments. In Part 2, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 3 in which patients will receive a dose of 3.0 mg/kg monthly for a total of 5 additional doses. At the end of each dosing period, efficacy assessments will be performed. During Part 4, patients will be evaluated at Months 1 and 3 after completion of dosing to assess the durability of wound healing and other efficacy parameters. Safety will be assessed continuously throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Phase 2 open-label
| Arm | Type | Receives |
|---|---|---|
| PTR-01 | Experimental | Drug: PTR-01All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg once monthly for a total of 5 doses. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Willing to provide informed consent form, or if 12 to <18 years of age, legal guardian has provided informed consent form and the minor has signed an assent form acknowledging that they understand and agree to study procedures.
- Has satisfactorily completed participation in PTR-01-002.
- Agrees to use contraception as follows:
- Be willing and able to comply with this protocol.
Key exclusion criteria (6 listed)
- Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.
- Has previously had an anaphylactic reaction to 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 (other than PTR-01).
- Is anticipated to receive new regimens of antibiotics or other anti-infectives during the trial.
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 collagen 7 (rC7)Intravenous recombinant collagen 7 | Drug | PTR-01 |
Primary outcome
Sustained wound healing
Time frame: Up to 246 days
Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)
Incidence of treatment-emergent adverse events
Time frame: Up to 246 days
Safety and tolerability, as assessed by treatment-emergent adverse events
34 secondary outcomes
Delivery of recombinant collagen 7 (PTR-01) to skin
Time frame: Up to 246 days
Formation of anchoring fibrils
Time frame: Up to 246 days
Change in wound surface area
Time frame: Up to 246 days
Change in skin integrity, as assessed by suction blister time
Time frame: Up to 246 days
Change in skin integrity, as assessed by time to re-blistering
Time frame: Up to 246 days
Change in itch severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains
Time frame: Up to 246 days
Change in the impact of itch on quality of life
Time frame: Up to 246 days
Change in pain severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) pain domains
Time frame: Up to 246 days
Change in pain severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)
Time frame: Up to 246 days
Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire
Time frame: Up to 246 days
Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument
Time frame: Up to 246 days
Change in corneal symptoms
Time frame: Up to 246 days
Stabilization of corneal symptoms
Time frame: Up to 246 days
Rate of change in nutritional markers (hemoglobin)
Time frame: Up to 246 days
Rate of change in nutritional markers (hematocrit)
Time frame: Up to 246 days
Rate of change in nutritional markers (total protein/albumin)
Time frame: Up to 246 days
Rate of change in nutritional markers (Fe/TIBC)
Time frame: Up to 246 days
Rate of change in nutritional markers (C-reactive protein)
Time frame: Up to 246 days
Change in Investigator Global Impressions of Change (IGIC)
Time frame: Up to 246 days
Change in Investigator Patient Impressions of Change (PGIC)
Time frame: Up to 246 days
Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Time frame: Up to 246 days
Change in overall health
Time frame: Up to 246 days
Change in mental health
Time frame: Up to 246 days
Change in social function
Time frame: Up to 246 days
Change in amount of wound care
Time frame: Up to 246 days
Change in time for wound care
Time frame: Up to 246 days
Change in cost of wound care
Time frame: Up to 246 days
Change in overall patient impression of quality of life
Time frame: Up to 246 days
Change in overall patient impression of disability
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Cmax
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Tmax
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Area Under the Curve (AUC)
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of clearance of PTR-01
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of PTR-01 half-life
Time frame: Up to 246 days
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 21 Nov 2022.
- Individual participant dataSharing planned: No
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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.
Dan Rudin, MD study director
Phoenix Tissue Repair
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PTR-01 in people with Recessive Dystrophic Epidermolysis Bullosa, run by Phoenix Tissue Repair, Inc. at 2 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 6 inclusion and 6 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 21 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
Sustained wound healing (time frame: Up to 246 days). The study lists 2 primary outcomes in all. 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 21 Nov 2022.