QR-313
- Generic Name
- QR-313
- Drug Type
- Biotech
- Background
QR-313 is an antisense oligonucleotide targeting exon 73. It is being investigated for the treatment of dystrophic epidermolysis bullosa.
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
Phase 1
Not yet recruiting
- Conditions
- Dystrophic Epidermolysis Bullosa
- Interventions
- Drug: Placebo
- First Posted Date
- 2022-09-06
- Last Posted Date
- 2023-03-31
- Lead Sponsor
- Phoenicis Therapeutics
- Target Recruit Count
- 8
- Registration Number
- NCT05529134
- Locations
- 🇺🇸
Stanford Health Care, Stanford, California, United States
🇺🇸UMass Memorial Medical Center, Worcester, Massachusetts, United States
🇺🇸Cincinnati Children's Hospital, Cincinnati, Ohio, United States
A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene
Phase 1
Terminated
- Conditions
- Epidermolysis Bullosa Dystrophica, RecessiveEpidermolysis Bullosa Dystrophica, Dominant
- Interventions
- Drug: Placebo
- First Posted Date
- 2018-07-30
- Last Posted Date
- 2021-08-25
- Lead Sponsor
- Phoenicis Therapeutics
- Target Recruit Count
- 2
- Registration Number
- NCT03605069
- Locations
- 🇺🇸
Journey Clinic, Center for Pediatric Blood and Marrow Transplantation, Minneapolis, Minnesota, United States
🇺🇸Stanford University School of Medicine, LPCH, Palo Alto, California, United States
🇺🇸Children's Hospital Colorado, Aurora, Colorado, United States
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