PTW-002-001

• Research type

  Research Study

• Full title

  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 Or Recessive Dystrophic Epidermolysis Bullosa (DDEB / RDEB) Due To Mutations(s) In Exon 73 Of The COL7A1 Gene

• IRAS ID

  1007270

• Contact name

  Hal Landy

• Contact email

  hal.landy@phoenicistx.com

• Sponsor organisation

  Phoenicis Therapeutics

• Eudract number

  2023-000017-40

• Research summary

  Dystrophic epidermolysis bullosa (DEB) is one of the major forms of epidermolysis bullosa. The signs and symptoms can vary widely among affected people. In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases often involve widespread blistering that can lead to vision loss, disfigurement, and other serious medical problems. DEB is caused by a mutation in the COL7A1 gene. The study medication in this trial is an antisense oligonucleotide that leads to the normal function of C7 Protein and is likely to improve the symptoms of the disease. The safety, proof of mechanism of the study medication, preliminary efficacy, and systemic exposure in patients with Dystrophic Epidermolysis Bullosa shall be investigated in this trial.

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  23/SW/0064

• Date of REC Opinion

  25 Sep 2023

• REC opinion

  Further Information Unfavourable Opinion