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Topical Agent
PTW-002 Gel for Epidermolysis Bullosa
Phase 1 & 2
Waitlist Available
Research Sponsored by Phoenicis Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
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.
Male or female, ≥ 4 - 17 years of age at Screening for RDEB patients, and ≥ 4 years of age at Screening for DDEB patients.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline through week 32
Awards & highlights
Study Summary
This trial will test the safety and effectiveness of PTW-002 in patients with DDEB or RDEB. Up to 8 patients will be enrolled, and will receive multiple doses of PTW-002. The trial will evaluate the safety, proof of mechanism, preliminary efficacy, and systemic exposure of PTW-002 in these patients.
Who is the study for?
This trial is for children and adults with Dystrophic Epidermolysis Bullosa (DEB) due to specific gene mutations. Participants must have a caregiver, no signs of local infection in the target wound area, and agree to use contraception if applicable. Excluded are those with certain treatments or conditions that could affect safety or study results.Check my eligibility
What is being tested?
The study tests PTW-002 gel against a placebo in patients with DEB. It's double-blind, meaning neither participants nor researchers know who gets the real treatment versus placebo. The goal is to assess safety, how well it works, and its effects on the body.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions at the application site such as redness or irritation since PTW-002 is a topical medication being tested for skin conditions.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have RDEB or DDEB with a specific genetic mutation.
Select...
I am between 4 and 17 years old with RDEB, or at least 4 years old with DDEB.
Select...
I have a wound between 5 to 30 cm2, without infection or cancer, suitable for treatment.
Select...
I agree to use effective birth control or practice abstinence for 3 months after my last dose.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline through week 32
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through week 32
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Assessment of exon 73 exclusion in COL7A1 mRNA, measured by droplet digital polymerase chain reaction (ddPCR)
Number of adverse events (AEs)/serious adverse events (SAEs)
Secondary outcome measures
Basement membrane
Effect of PTW-002 on skin strength by onset of (re)blistering of a healed wound
Effect of PTW-002 on the presence of anchoring fibrils measured by electron microscopy
+3 moreTrial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: PTW-002 10 mg/g gelExperimental Treatment1 Intervention
PTW-002 poloxamer hydrogel for topical administration (cutaneous use), 10 mg/g gel
Group II: PlaceboPlacebo Group1 Intervention
Matching placebo poloxamer hydrogel for topical administration (cutaneous use)
Find a Location
Who is running the clinical trial?
Phoenicis TherapeuticsLead Sponsor
2 Previous Clinical Trials
52 Total Patients Enrolled
1 Trials studying Epidermolysis Bullosa
2 Patients Enrolled for Epidermolysis Bullosa
Hal Landy, MDStudy DirectorPhoenicis Therapeutics
6 Previous Clinical Trials
88 Total Patients Enrolled
2 Trials studying Epidermolysis Bullosa
2 Patients Enrolled for Epidermolysis Bullosa
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had gene therapy applied to my skin.I have not had cancer in the TWA or any cancer in the last 5 years, except for treated skin cancer.I haven't taken aminoglycoside antibiotics, except as eye drops, for 7 days or 5 half-lives before my baseline visit.I have a bleeding disorder and need anticoagulants, confirmed by a recent test.I have RDEB or DDEB with a specific genetic mutation.I am between 4 and 17 years old with RDEB, or at least 4 years old with DDEB.I needed a blood transfusion for my hemoglobin level at screening.I agree to use effective birth control or practice abstinence for 3 months after my last dose.I have a wound between 5 to 30 cm2, without infection or cancer, suitable for treatment.I do not have serious liver or kidney diseases that could affect my safety in the study.I haven't used any experimental drugs or devices recently, nor plan to during the study.I have had cell therapy that needed specific medication.
Research Study Groups:
This trial has the following groups:- Group 1: PTW-002 10 mg/g gel
- Group 2: Placebo
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there still vacancies for participants in this trial?
"Confirming the info on clinicaltrials.gov, this particular medical trial has ceased recruitment activities since September 1st 2022. Nevertheless, there are currently 28 other studies actively seeking patients for participation."
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