PTW-002 10 mg/g gel for Dystrophic Epidermolysis Bullosa

Phase-Based Progress Estimates
Stanford Health Care, Stanford, CA
Dystrophic Epidermolysis Bullosa+2 More
PTW-002 10 mg/g gel - Drug
Any Age
All Sexes
What conditions do you have?

Study 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 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.

Eligible Conditions

  • Dystrophic Epidermolysis Bullosa

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Dystrophic Epidermolysis Bullosa

Study Objectives

2 Primary · 6 Secondary · Reporting Duration: Baseline through Week 32

Baseline through Week 16
Effect of PTW-002 on skin strength by onset of (re)blistering of a healed wound
Effect of PTW-002 on wound healing by change in wound size (surface area)
Baseline through Week 32
Number of adverse events (AEs)/serious adverse events (SAEs)
Systemic exposure through serum levels of PTW-002 after topical administration to the target wound area (TWA)
Week 4
Assessment of exon 73 exclusion in COL7A1 mRNA, measured by droplet digital polymerase chain reaction (ddPCR)
Change in uptake of PTW-002 by cells at the basement membrane by fluorescent in situ hybridization (FISH) analysis
Week 8
Effect of PTW-002 on the presence of anchoring fibrils measured by electron microscopy
Effect of PTW-002 on the presence of collagen type VII protein measured by immunofluorescent staining

Trial Safety

Safety Progress

1 of 3

Other trials for Dystrophic Epidermolysis Bullosa

Trial Design

2 Treatment Groups

PTW-002 10 mg/g gel
1 of 2
1 of 2
Experimental Treatment
Non-Treatment Group

8 Total Participants · 2 Treatment Groups

Primary Treatment: PTW-002 10 mg/g gel · Has Placebo Group · Phase 1 & 2

PTW-002 10 mg/g gel
Experimental Group · 1 Intervention: PTW-002 10 mg/g gel · Intervention Types: Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline through week 32
Closest Location: Stanford Health Care · Stanford, CA
2019First Recorded Clinical Trial
1 TrialsResearching Dystrophic Epidermolysis Bullosa
25 CompletedClinical Trials

Eligibility Criteria

Age Any Age · All Participants · 8 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are male or female, or are in the 4-17 age group
You have a confirmed diagnosis of RDEB or DDEB and at least one pathogenic mutation in exon 73 of the COL7A1 gene
Surface area of the target lesion must be between 5 and 30 cm2.
The investigational medicinal product (IMP) is absorbed into the sub-epidermal tissue.
You have no suspicion of current SCC upon visual inspection.
You 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.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.