- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05529134
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
March 29, 2023 updated by: Phoenicis Therapeutics
A Double-blind, Randomized, Intra-patient Placebo- Controlled, Multiple Dose Study of PTW-002 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy, and Systemic Exposure in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa (DDEB / RDEB) Due to Mutation(s) in Exon 73 of the COL7A1 Gene
A double-blind, randomized, intra-patient placebo- controlled, multiple dose study of PTW-002 evaluating safety, proof of mechanism, preliminary efficacy, and systemic exposure in patients with Dominant Dystrophic Epidermolysis Bullosa (DDEB) or Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene.
Up to two RDEB patients 4 to 17 years of age and up to 6 DDEB patients 4 years of age and older will be enrolled.
Study Overview
Status
Not yet recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Anticipated)
8
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Ramsey Johnson
- Phone Number: 978-726-1478
- Email: ramsey@phoenicistx.com
Study Locations
-
-
California
-
Stanford, California, United States, 94305
- Stanford Health Care
-
Principal Investigator:
- Peter Marinkovich, MD
-
Contact:
- Kunju J Sridhar
- Email: kunju@stanford.edu
-
-
Massachusetts
-
Worcester, Massachusetts, United States, 01655
- UMass Memorial Medical Center
-
Contact:
- Celia Hartigan
- Email: Celia.Hartigan@umassmed.edu
-
-
Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital
-
Principal Investigator:
- Anne Lucky, MD
-
Contact:
- Bret Augsburger
- Email: Bret.Augsburger@cchmc.org
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
4 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Patients, and/or their legal guardian(s), if the patient is under the legal age of consent, must provide written Informed Consent or Assent, in accordance with national and/or local laws, prior to the conduct of any study related procedures. In addition, if applicable, a minor child must provide informed Assent in accordance with national and/or local laws and in compliance with the recommendations of the approving Institutional Review Board.
- Male or female, ≥ 4 - 17 years of age at Screening for RDEB patients, and ≥ 4 years of age at Screening for DDEB patients.
- Have a confirmed diagnosis of RDEB or DDEB and at least one pathogenic mutation in exon 73 of the COL7A1 gene. Historical genetic data may be acceptable with Medical Monitor approval.
Have at least one TWA that shows no signs of local infection, and contains a target lesion that is either new or has shown dynamic healing in the past and complies with the following additional criteria:
- surface area of the target lesion ranging from 5 to 30 cm2, located centrally in the selected TWA.
- exposed sub-epidermal tissue to allow absorption of the investigational medicinal product (IMP).
- no suspicion of current squamous cell carcinoma (SCC) upon visual inspection.
- Have a caregiver or support person available, who can follow study instructions in compliance with the protocol and attend study site visits with the patient as required, in the opinion of the Investigator.
- Female patients who have reached menarche and male patients must either practice true abstinence in accordance with their preferred and usual lifestyle, or agree to use acceptable, effective methods of contraception for up to 3 months following their last dose of IMP.
Exclusion Criteria:
- Pregnant or breast-feeding female.
- Hemoglobin level at Screening requiring transfusion. The patient may be rescreened when the condition is considered stable.
- Use of aminoglycosides, by any route of administration, except eye drops, 7 days or 5 half-lives, whichever is longer, prior to Baseline visit.
- Untreated carcinoma of the TWA or history of carcinoma within 5 years prior to Screening, except adequately treated cutaneous squamous or basal cell carcinoma.
- Life expectancy less than 6 months, as assessed by the Investigator.
- Current or known history of clinically significant hepatic or renal disease that in the opinion of the Investigator, could impact patient safety or study participation.
- Bleeding disorder or condition, requiring the use of anticoagulants to be confirmed by activated partial thromboplastin time (aPTT) by local lab within 48 hours of first treatment.
- Use of any investigational drug or device within 28 days or 5 half-lives of the Baseline visit, whichever is longer, or plans to participate in another study of a drug or device during the study period. The washout of 5 half-lives does not apply to gene and cell therapy.
- History of cell therapy requiring treatment with exclusionary medication.
- History of skin-based gene therapy to the TWA.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: PTW-002 10 mg/g gel
PTW-002 poloxamer hydrogel for topical administration (cutaneous use), 10 mg/g gel
|
poloxamer hydrogel for topical administration
|
Placebo Comparator: Placebo
Matching placebo poloxamer hydrogel for topical administration (cutaneous use)
|
placebo poloxamer hydrogel for topical administration
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Number of adverse events (AEs)/serious adverse events (SAEs)
Time Frame: Baseline through Week 32
|
Baseline through Week 32
|
Assessment of exon 73 exclusion in COL7A1 mRNA, measured by droplet digital polymerase chain reaction (ddPCR)
Time Frame: Week 4
|
Week 4
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change in uptake of PTW-002 by cells at the basement membrane by fluorescent in situ hybridization (FISH) analysis
Time Frame: Week 4
|
Week 4
|
Effect of PTW-002 on wound healing by change in wound size (surface area)
Time Frame: Baseline through Week 16
|
Baseline through Week 16
|
Effect of PTW-002 on skin strength by onset of (re)blistering of a healed wound
Time Frame: Baseline through Week 16
|
Baseline through Week 16
|
Systemic exposure through serum levels of PTW-002 after topical administration to the target wound area (TWA)
Time Frame: Baseline through Week 32
|
Baseline through Week 32
|
Effect of PTW-002 on the presence of collagen type VII protein measured by immunofluorescent staining
Time Frame: Week 8
|
Week 8
|
Effect of PTW-002 on the presence of anchoring fibrils measured by electron microscopy
Time Frame: Week 8
|
Week 8
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Hal Landy, MD, Phoenicis Therapeutics
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Anticipated)
April 30, 2023
Primary Completion (Anticipated)
April 30, 2024
Study Completion (Anticipated)
July 31, 2024
Study Registration Dates
First Submitted
September 1, 2022
First Submitted That Met QC Criteria
September 1, 2022
First Posted (Actual)
September 6, 2022
Study Record Updates
Last Update Posted (Actual)
March 31, 2023
Last Update Submitted That Met QC Criteria
March 29, 2023
Last Verified
March 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- PTW-002-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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