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Recessive Dystrophic Epidermolysis BullosaPhase 2NCT04599881

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001. Read more about this study

Who is it for?
Ages 12 years and older
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
6 participants
Where
2 sitesUnited States
Study dates
15 Oct 2020to 1 Sep 2021
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

  • Patients must meet all of the following criteria to be eligible for study participation in the three month run in period of the study:
  • 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 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.
  • 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

  • Patients with any of the following will be excluded from participation in the study:
  • 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.
  • 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

Trial locations and contacts

2 sites in 1 country.

United States2 sites
  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Stanford University

    Redwood City, California, 94063

Other trials in United States

About this study

Official title: A Phase 2 Open-Label Study of PTR-01 in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.

Read the detailed description

Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.

In Part 1, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 2 in which patients will receive a dose of 3.0 mg/kg every other week for a total of 7 doses. In Part 3, patients will be followed for 12 weeks. No investigational therapy will be administered during this time. At the end of each dosing period, an efficacy assessment will be performed. Safety will be assessed continuously throughout the study.

Following the end of Part 3, patients may be eligible for a potential long-term extension to further refine the dosing regimen, depending upon study drug availability.

Being tested
PTR-01
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 collagen 7, rC7

    IV recombinant collagen 7 at 3 mg/kg given weekly for 4 doses, followed by bi-weekly for 7 doses

Study groups in NCT04599881
Study groupRoleWhat they receive
PTR-01 3 mg/kgExperimentalAll 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 every other week for a total of 7 doses.
Receives: Drug: PTR-01

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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 162 days

  2. Incidence of treatment-emergent adverse events

    Safety and tolerability, as assessed by treatment-emergent adverse events

    Measured: Up to 162 days

  3. Incidence of infusion-associated reactions

    Safety and tolerability, as assessed by infusion-associated reactions (IAR)

    Measured: Up to 162 days

  4. Incidence of anti-drug antibodies (ADA)

    Safety and tolerability, as assessed by immunogenicity through anti-drug antibody (ADA) testing

    Measured: Up to 162 days

Secondary outcomes (39)
  1. Delivery of PTR-01 to skin

    PTR-01 incorporation by immunofluorescence using NC1 \& NC2 staining, by dose frequency period

    Measured: Up to 162 days

  2. Formation of anchoring fibrils

    Formation of new anchoring fibrils as measured by electron microscopy

    Measured: Up to 162 days

  3. Change in wound surface area, as assessed by wound imaging

    Wound area of target lesions, as assessed by wound imaging

    Measured: Up to 162 days

  4. Change in wound surface area, as assessed by Investigator Global Impression of Change (IGIC)

    Wound area of target lesions, as assessed by IGIC

    Measured: Up to 162 days

  5. Change in total body wound surface area

    Change in total body wound surface area, using Rule of Nines

    Measured: Up to 162 days

  6. Change in skin integrity, as assessed by suction blister time

    Change in skin integrity, as assessed by suction blister time

    Measured: Up to 162 days

  7. 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 162 days

  8. 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

    Measured: Up to 162 days

  9. Change in itch severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)

    Severity of itch, as assessed by Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB), maximum score of 234 (worst)

    Measured: Up to 162 days

  10. 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 162 days

  11. 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

    Measured: Up to 162 days

  12. 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 162 days

  13. Change in the impact of pain on quality of life

    Change in the impact of pain on quality of life, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB) instrument, maximum score of 234 (worst)

    Measured: Up to 162 days

  14. 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 162 days

  15. Change in dysphagia, as assessed by volume of oral nutritional intake

    Change of dysphagia, as assessed by volume of oral nutritional intake, using patient interview and diary, maximum score is 40 (worst)

    Measured: Up to 162 days

  16. Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Scale

    Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Scale

    Measured: Up to 162 days

  17. Stabilization of dysphagia, as assessed by volume oral nutritional intake

    Stabilization of dysphagia, as assessed by volume oral nutritional intake, using patient interview and diary

    Measured: Up to 162 days

  18. Change in corneal symptoms

    Change of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI)

    Measured: Up to 162 days

  19. Stabilization of corneal symptoms

    Stabilization of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI)

    Measured: Up to 162 days

  20. Rate of change in nutritional markers (hemoglobin/hematocrit)

    Change of nutritional markers, as assessed by hemoglobin/hematocrit

    Measured: Up to 162 days

  21. Rate of change in nutritional markers (total protein/albumin)

    Change of nutritional markers, as assessed by total protein/albumin

    Measured: Up to 162 days

  22. Rate of change in nutritional markers (iron/TIBC)

    Change of nutritional markers, as assessed by iron/TIBC

    Measured: Up to 162 days

  23. Rate of change in nutritional markers (C-reactive protein)

    Change of nutritional markers, as assessed by C-reactive protein

    Measured: Up to 162 days

  24. Rate of stabilization of nutritional markers (hemoglobin/hematocrit)

    Stabilization of nutritional markers, as assessed by hemoglobin/hematocrit

    Measured: Up to 162 days

  25. Rate of stabilization of nutritional markers (total protein/albumin)

    Stabilization of nutritional markers, as assessed by total protein/albumin

    Measured: Up to 162 days

  26. Rate of stabilization of nutritional markers (iron/TIBC)

    Stabilization of nutritional markers, as assessed by iron/TIBC

    Measured: Up to 162 days

  27. Rate of stabilization of nutritional markers (C-reactive protein)

    Stabilization of nutritional markers, as assessed by C-reactive protein

    Measured: Up to 162 days

  28. Change in Investigator Global Impressions of Change (IGIC)

    Global impressions of change, as assessed through IGIC (1-7), 7 being worst

    Measured: Up to 162 days

  29. Change in Investigator Patient Impressions of Change (PGIC)

    Global impressions of change, as assessed through PGIC (1-7), 7 being worst

    Measured: Up to 162 days

  30. Change in disease activity and scarring

    Change in disease activity and scarring, as assessed by the Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI)

    Measured: Up to 162 days

  31. 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

    Measured: Up to 162 days

  32. Change in overall health

    Change in overall disability, as assessed by the Health Assessment Questionnaire or Children's Health Assessment Questionnaire (HAQ/CHAQ)

    Measured: Up to 162 days

  33. Change in mental health

    Change in mental health and social functioning, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) mental health domains

    Measured: Up to 162 days

  34. Change in social function

    Change in mental health and social functioning, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) social function domains

    Measured: Up to 162 days

  35. Change in amount of wound care

    Change in amount of wound care, as assessed by patient interviews

    Measured: Up to 162 days

  36. Change in time for wound care

    Change in time for wound care, as assessed by patient interviews

    Measured: Up to 162 daysUp to 162 days

  37. Change in cost of wound care

    Change in cost of wound care, as assessed by patient interviews

    Measured: Up to 162 days

  38. Change in overall patient impression of quality of life

    Change in overall quality of life, as assessed by patient interviews

    Measured: Up to 162 days

  39. Change in overall patient impression of disability

    Change in overall disability, as assessed by patient interviews

    Measured: Up to 162 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Oct 2020
  2. Study start15 Oct 2020
  3. Primary completion28 Aug 2021
  4. Study completion1 Sep 2021

Resources

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
Open-label study
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04599881
Sponsor's protocol ID
PTR-01-002
Status last verified
Sep 2021
First submitted
8 Oct 2020
Last updated
16 Sep 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

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 8 inclusion and 7 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 16 Sep 2021. 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?

Wound healing (time frame: Up to 162 days). The study lists 4 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 16 Sep 2021.

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Clinical Trial NCT04599881 | AzTrials