Dystrophic Epidermolysis BullosaPhase 1/2NCT05529134
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
A Phase 1/2 study testing PTW-002 10 mg/g gel in people with Dystrophic Epidermolysis Bullosa, run by Phoenicis Therapeutics at 3 sites in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- What is being tested?
- PTW-002 10 mg/g gel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 3 sitesUnited States
- Study dates
- 30 Apr 2023to 31 Jul 2024
- 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 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Bret Augsburger, Bret.Augsburger@cchmc.org
Anne Lucky, MD
Stanford Health Care
Stanford, California, 94305
Kunju J Sridhar, kunju@stanford.edu
Peter Marinkovich, MD
UMass Memorial Medical Center
Worcester, Massachusetts, 01655
Celia Hartigan, Celia.Hartigan@umassmed.edu
Karen Wiss, MD
About this study
Official title: 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.
- Conditions
- Dystrophic Epidermolysis Bullosa
- Being tested
- PTW-002 10 mg/g gel, Placebo
- Keywords
- DDEB, RDEB, exon 73, Epidermolysis Bullosa, COL7A1 gene, DEB, collagen type VII protein, C7, Dominant Dystrophic Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- 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.
PTW-002 10 mg/g gelDrug
poloxamer hydrogel for topical administration
PlaceboDrug
placebo poloxamer hydrogel for topical administration
| Study group | Role | What they receive |
|---|---|---|
| PTW-002 10 mg/g gel | Experimental | PTW-002 poloxamer hydrogel for topical administration (cutaneous use), 10 mg/g gel Receives: Drug: PTW-002 10 mg/g gel |
| Placebo | Placebo comparator | Matching placebo poloxamer hydrogel for topical administration (cutaneous use) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events (AEs)/serious adverse events (SAEs)
Measured: Baseline through Week 32
Assessment of exon 73 exclusion in COL7A1 mRNA, measured by droplet digital polymerase chain reaction (ddPCR)
Measured: Week 4
Secondary outcomes (6)
Change in uptake of PTW-002 by cells at the basement membrane by fluorescent in situ hybridization (FISH) analysis
Measured: Week 4
Effect of PTW-002 on wound healing by change in wound size (surface area)
Measured: Baseline through Week 16
Effect of PTW-002 on skin strength by onset of (re)blistering of a healed wound
Measured: Baseline through Week 16
Systemic exposure through serum levels of PTW-002 after topical administration to the target wound area (TWA)
Measured: Baseline through Week 32
Effect of PTW-002 on the presence of collagen type VII protein measured by immunofluorescent staining
Measured: Week 8
Effect of PTW-002 on the presence of anchoring fibrils measured by electron microscopy
Measured: Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2022
- Study start30 Apr 2023 (estimated)
- Primary completion30 Apr 2024 (estimated)
- Study completion31 Jul 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Phoenicis Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hal Landy, MD, Study director, Phoenicis Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05529134
- Sponsor's protocol ID
- PTW-002-001
- Status last verified
- Mar 2023
- First submitted
- 1 Sep 2022
- Last updated
- 31 Mar 2023
- Results posted
- 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 31 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05529134
- Acronym / sponsor ID
- PTW-002-001
- Conditions
- Dystrophic Epidermolysis Bullosa
- Keywords
- DDEB, RDEB, exon 73, Epidermolysis Bullosa, COL7A1 gene, DEB, collagen type VII protein, C7
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Phoenicis TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Apr 2023 (estimated)
- Primary completion
- 30 Apr 2024 (estimated)
- Study completion
- 31 Jul 2024 (estimated)
- First posted
- 6 Sep 2022 (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 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PTW-002 10 mg/g gel | Experimental | Drug: PTW-002 10 mg/g gelPTW-002 poloxamer hydrogel for topical administration (cutaneous use), 10 mg/g gel |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo poloxamer hydrogel for topical administration (cutaneous use) |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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:
- 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.
Key exclusion criteria (10 listed)
- 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.
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 |
|---|---|---|
| PTW-002 10 mg/g gelpoloxamer hydrogel for topical administration | Drug | PTW-002 10 mg/g gel |
| Placeboplacebo poloxamer hydrogel for topical administration | Drug | Placebo |
Primary outcome
Number of adverse events (AEs)/serious adverse events (SAEs)
Time frame: Baseline through Week 32
Assessment of exon 73 exclusion in COL7A1 mRNA, measured by droplet digital polymerase chain reaction (ddPCR)
Time frame: Week 4
6 secondary outcomes
Change in uptake of PTW-002 by cells at the basement membrane by fluorescent in situ hybridization (FISH) analysis
Time frame: Week 4
Effect of PTW-002 on wound healing by change in wound size (surface area)
Time frame: 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
Systemic exposure through serum levels of PTW-002 after topical administration to the target wound area (TWA)
Time frame: Baseline through Week 32
Effect of PTW-002 on the presence of collagen type VII protein measured by immunofluorescent staining
Time frame: Week 8
Effect of PTW-002 on the presence of anchoring fibrils measured by electron microscopy
Time frame: Week 8
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2023.
- 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.
Hal Landy, MD study director
Phoenicis Therapeutics
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 PTW-002 10 mg/g gel in people with Dystrophic Epidermolysis Bullosa, run by Phoenicis Therapeutics 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 10 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: status not confirmed, as of the sponsor's last update on 31 Mar 2023. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ramsey Johnson (978-726-1478, ramsey@phoenicistx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse events (AEs)/serious adverse events (SAEs) (time frame: Baseline through Week 32). 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 31 Mar 2023.