PTW-002 10 mg/g gel for Dystrophic Epidermolysis Bullosa
Phase-Based Progress Estimates
Effectiveness
Safety
Stanford Health Care, Stanford, CA
Dystrophic Epidermolysis Bullosa+2 More
PTW-002 10 mg/g gel - DrugEligibility
Any Age
All Sexes
What conditions do you have?
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Eligibility
Any Age
All Sexes
What conditions do you have?
Select
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
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
Trial Design
2 Treatment Groups
PTW-002 10 mg/g gel
1 of 2
Placebo
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
Drug
Experimental Group · 1 Intervention: PTW-002 10 mg/g gel · Intervention Types: DrugPlacebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: DrugTrial 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 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.
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