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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
8
poloxamer hydrogel for topical administration
placebo poloxamer hydrogel for topical administration
Stanford Health Care
Stanford, California, United States
UMass Memorial Medical Center
Worcester, Massachusetts, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
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
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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.