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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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5 April 2021 |
Main ID: |
EUCTR2017-004806-17-FR |
Date of registration:
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05/02/2018 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
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Scientific title:
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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 |
Date of first enrolment:
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Target sample size:
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14 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-004806-17 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 1
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Phase:
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Human pharmacology (Phase I): yes
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
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Countries of recruitment
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Austria
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Canada
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Czech Republic
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France
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Germany
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Spain
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trial Manager
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Address:
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Zernikedreef 9
2333 CK
Leiden
Netherlands |
Telephone:
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+31881667000 |
Email:
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clinical@proqr.com |
Affiliation:
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ProQR Therapeutics |
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Name:
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Clinical Trial Manager
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Address:
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Zernikedreef 9
2333 CK
Leiden
Netherlands |
Telephone:
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+31881667000 |
Email:
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clinical@proqr.com |
Affiliation:
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ProQR Therapeutics |
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Key inclusion & exclusion criteria
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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: 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.
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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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
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Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
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Intervention(s)
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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 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Gel Route of administration of the placebo: Cutaneous use
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Primary Outcome(s)
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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
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Primary end point(s): - Assessment of adverse events/serious adverse events - Absence of exon 73 in COL7A1 mRNA, detected by polymerase chain reaction (PCR)
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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
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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 Outcome(s)
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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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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)
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Secondary ID(s)
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PQ-313-002
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2017-004806-17-GB
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Source(s) of Monetary Support
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ProQR Therapeutics
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Ethics review
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Status:
Approval date:
Contact:
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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