why choose us

First received: September 01, 2022

Clinical Trial: Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene

Study Status: NOT_YET_RECRUITING

Recruit Status: NOT_YET_RECRUITING

Condition: Dystrophic Epidermolysis Bullosa

Study Type: INTERVENTIONAL


Official Title: 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 Domina

Brief 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.

Read more