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

PHASE1/PHASE2TerminatedINTERVENTIONAL
Enrollment

2

Participants

Timeline

Start Date

July 2, 2018

Primary Completion Date

December 17, 2018

Study Completion Date

December 17, 2018

Conditions
Epidermolysis Bullosa Dystrophica, RecessiveEpidermolysis Bullosa Dystrophica, Dominant
Interventions
DRUG

QR-313

QR-313 will be applied topically once daily for 8 weeks of treatment.

DRUG

Placebo

Placebo will be applied topically once daily for 8 weeks of treatment.

Trial Locations (6)

15005

Cincinnati Children's Hospital, Cincinnati

28046

Hospital Universitario La Paz, Madrid

55454

Journey Clinic, Center for Pediatric Blood and Marrow Transplantation, Minneapolis

75015

Hopital Necker Enfants Malades, Paris

80045

Children's Hospital Colorado, Aurora

94305

Stanford University School of Medicine, LPCH, Palo Alto

Sponsors
All Listed Sponsors
lead

Phoenicis Therapeutics

INDUSTRY

NCT03605069 - 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 | Biotech Hunter | Biotech Hunter