Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene

A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-004806-17-FR

Enrollment

Registered

2018-02-05

Start date

Unknown

Completion date

Unknown

Last updated

2021-04-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: QR-313 Gel for Topical (Cutaneous) Administration Product Code: QR-313 Pharmaceutical Form: Gel INN or Proposed INN: unavailable CAS Number: unavailable Current Sponsor code: QR-313 Conc

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Male or female, = 2 years of age at Screening with a clinical diagnosis of RDEB with confirmation of at least one of the alleles of the COL7A1 gene containing one or more pathogenic mutations in exon 73. 2. Have at least one TWA of 10 x 10 cm that shows dynamic wound healing, no signs of local infection and in which a wound can be selected with the following criteria: a. a surface area ranging from 5 to 60 cm2 b. open wound that has healing potential c. exposed sub-epidermal tissue to allow absorption of the IMP d. no suspicion of current squamous cell carcinoma (SCC) upon visual inspection Are the trial subjects under 18? yes Number of subjects for this age range: 7 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 7 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Pregnant or breast-feeding female 2. Hemoglobin level at Screening requiring transfusion. The subject may be rescreened when the condition is considered stable 3. Untreated carcinoma of the TWA or history of carcinoma within 5 years prior to Screening, except adequately treated cutaneous squamous or basal cell carcinoma. 4. Life expectancy less than 6 months, as assessed by the Investigator 5. Current or known history of clinically significant hepatic or renal disease, that in the opinion of the Investigator, could impact subject safety or study participation. 6. Treatment with any systemic immunomodulators, immunosuppressants or cytotoxic chemotherapy within 2 months prior to the Baseline visit.

Design outcomes

Primary

Measure	Time frame
Main Objective: - To evaluate safety and tolerability following topical administration of QR-313 to the target wound area (TWA) - To assess the effect of QR-313 on the exclusion (skipping) of exon 73 from COL7A1 mRNA ;Secondary Objective: - To assess the preliminary efficacy on wound healing and skin strength - To assess systemic exposure after topical administration of QR-313 to the Target Wound Area (TWA) - To assess the effect of QR-313 on the presence of collagen type VII protein and anchoring fibrils ;Primary end point(s): - Assessment of adverse events/serious adverse events - Absence of exon 73 in COL7A1 mRNA, detected by polymerase chain reaction (PCR) ;Timepoint(s) of evaluation of this end point: - AEs are assessed throughout the study - Exon skip is assessed after 1 week of treatment with IMP	—

Measure

Time frame

Main Objective: - To evaluate safety and tolerability following topical administration of QR-313 to the target wound area (TWA) - To assess the effect of QR-313 on the exclusion (skipping) of exon 73 from COL7A1 mRNA ;Secondary Objective: - To assess the preliminary efficacy on wound healing and skin strength - To assess systemic exposure after topical administration of QR-313 to the Target Wound Area (TWA) - To assess the effect of QR-313 on the presence of collagen type VII protein and anchoring fibrils ;Primary end point(s): - Assessment of adverse events/serious adverse events - Absence of exon 73 in COL7A1 mRNA, detected by polymerase chain reaction (PCR) ;Timepoint(s) of evaluation of this end point: - AEs are assessed throughout the study - Exon skip is assessed after 1 week of treatment with IMP

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Secondary

Measure	Time frame
Secondary end point(s): - Wound size (surface area) - Wound severity as assessed by Physician Subjective Assessment of Severity (PSAS) and Physician Subjective Assessment of Change (PSAC) - Wound status as assessed by Short Wound Specific Questionnaire (SWSQ) - Status of wound closure - Onset of (re)blistering of a healed wound - Serum levels of QR-313 - Presence of collagen type VII protein expression, measured by indirect immunofluorescence (IIF) microscopy - Presence of anchoring fibrils measured by transmission electron microscopy (TEM);Timepoint(s) of evaluation of this end point: - weekly images are taken throughout the study for the purpose of the wound healing and skin strength parameters - serum level of QR-313 is measured after 1 week of treatment with IMP - collagen type VII protein expression at 8 weeks after last dose of IMP (EOS) - anchoring fibrils measured at 8 weeks after last dose of IMP (EOS)	—

Measure

Time frame

Secondary end point(s): - Wound size (surface area) - Wound severity as assessed by Physician Subjective Assessment of Severity (PSAS) and Physician Subjective Assessment of Change (PSAC) - Wound status as assessed by Short Wound Specific Questionnaire (SWSQ) - Status of wound closure - Onset of (re)blistering of a healed wound - Serum levels of QR-313 - Presence of collagen type VII protein expression, measured by indirect immunofluorescence (IIF) microscopy - Presence of anchoring fibrils measured by transmission electron microscopy (TEM);Timepoint(s) of evaluation of this end point: - weekly images are taken throughout the study for the purpose of the wound healing and skin strength parameters - serum level of QR-313 is measured after 1 week of treatment with IMP - collagen type VII protein expression at 8 weeks after last dose of IMP (EOS) - anchoring fibrils measured at 8 weeks after last dose of IMP (EOS)

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Countries

Austria, Canada, Czech Republic, France, Germany, Spain, United Kingdom, United States

Contacts

Public ContactClinical Trial Manager

ProQR Therapeutics

clinical@proqr.com+31881667000

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026