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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 below
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I have RDEB or DDEB with a specific genetic mutation.
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I am between 4 and 17 years old with RDEB, or at least 4 years old with DDEB.
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I have a wound between 5 to 30 cm2, without infection or cancer, suitable for treatment.
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I agree to use effective birth control or practice abstinence for 3 months after my last dose.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through week 32
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through week 32 for reporting.

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 more

Trial 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

PTW-002 (Topical Agent) Clinical Trial Eligibility Overview. Trial Name: NCT05529134 — Phase 1 & 2
Epidermolysis Bullosa Research Study Groups: PTW-002 10 mg/g gel, Placebo
Epidermolysis Bullosa Clinical Trial 2023: PTW-002 Highlights & Side Effects. Trial Name: NCT05529134 — Phase 1 & 2
PTW-002 (Topical Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05529134 — Phase 1 & 2

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."

Answered by AI
~1 spots leftby Apr 2024