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 of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
ClinicalTrials.gov ID: NCT05529134
Sponsor: Phoenicis Therapeutics
Information provided by: Phoenicis Therapeutics (Responsible Party)
Last Update Posted: 2023-03-31
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.
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 Dominant or Recessive Dystrophic Epidermolysis Bullosa (DDEB / RDEB) Due to Mutation(s) in Exon 73 of the COL7A1 Gene
Intervention / Treatment:
- Drug: PTW-002 10 mg/g gel
- Drug: Placebo
| Category | Value |
|---|---|
| Study Start (Actual) |
2023-04-30
|
| Primary Completion (Estimated) |
2024-04-30
|
| Study Completion (Estimated) |
2024-07-31
|
| Enrollment (Estimated) | 8 |
| Study Type | Interventional |
| Phase | Phase 1 Phase 2 |
| Other Study ID Numbers |
PTW-002-001
|