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    Summary
    EudraCT Number:2017-004806-17
    Sponsor's Protocol Code Number:PQ-313-002
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-04-11
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2017-004806-17
    A.3Full title of the trial
    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
    Estudio inicial en humanos, doble ciego, aleatorizado, intra-sujeto, controlado con placebo y de dosis múltiples de QR 313 para evaluar la seguridad, el mecanismo, la eficacia preliminar y la exposición sistémica en sujetos con epidermólisis ampollosa distrófica recesiva (EADR) debido a una mutación o mutaciones en el exón 73 del gen COL7A1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
    Estudio para evaluar QR-313 en sujetos con epidermólisis ampollosa distrófica recesiva (EADR) debido a una mutación o mutaciones en el exón 73 del gen COL7A1
    A.3.2Name or abbreviated title of the trial where available
    WINGS
    A.4.1Sponsor's protocol code numberPQ-313-002
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorProQR Theraputics
    B.1.3.4CountryNetherlands
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportProQR Therapeutics
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationProQR Therapeutics
    B.5.2Functional name of contact pointClinical Trial Manager
    B.5.3 Address:
    B.5.3.1Street AddressZernikedreef 9
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333 CK
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31881667000
    B.5.6E-mailclinical@proqr.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1938
    D.3 Description of the IMP
    D.3.1Product nameQR-313 Gel for Topical (Cutaneous) Administration
    D.3.2Product code QR-313
    D.3.4Pharmaceutical form Gel
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPCutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNunavailable
    D.3.9.1CAS number unavailable
    D.3.9.2Current sponsor codeQR-313
    D.3.9.4EV Substance CodeSUB190801
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboGel
    D.8.4Route of administration of the placeboCutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
    Epidermólisis ampollosa distrófica recesiva (EADR) debido a una mutación o mutaciones en el exón 73 del gen COL7A1
    E.1.1.1Medical condition in easily understood language
    RDEB due to mutation(s) in exon 73 of the COL7A1 gene
    EADR debido a una mutación o mutaciones en el exón 73 del gen COL7A1
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10014989
    E.1.2Term Epidermolysis bullosa
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - 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
    - Evaluar la seguridad y la tolerabilidad tras la aplicación tópica de QR-313 en el área de herida diana (AHD)
    - Evaluar el efecto de QR-313 en la exclusión (salto) del exón 73 del ARNm del gen COL7A1
    E.2.2Secondary objectives of the trial
    - 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
    - Evaluar la eficacia preliminar en la curación de la herida y en la resistencia de la piel
    - Evaluar la exposición sistémica tras la administración tópica de QR-313 en el área de herida diana (AHD)
    - Evaluar el efecto de QR-313 en la presencia de la proteína del colágeno de tipo VII y de las fibrillas de anclaje
    E.2.3Trial contains a sub-study No
    E.3Principal 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
    1. Sujetos de ambos sexos, ≥2 años de edad en el momento de la selección con un diagnóstico clínico de EADR con confirmación de una o más mutaciones patogénicas en el exón 73 en al menos uno de los alelos del gen COL7A1.
    2. Tener al menos un AHD de 10 x 10 cm que muestre curación dinámica de la herida, sin signos de infección local y en la que se pueda seleccionar una herida que cumpla los criterios siguientes:
    a. área de superficie de 5 a 60 cm2
    b. herida abierta con potencial de curación
    c. tejido subepidérmico expuesto para permitir la absorción del PEI
    d. sin sospecha en el momento de la inspección visual de carcinoma escamocelular (CEC) simultáneo.
    E.4Principal 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.
    1. Mujeres embarazadas o en periodo de lactancia
    2. Nivel de hemoglobina en la selección que requiere transfusiones. El sujeto puede volverse a someter al procedimiento de selección cuando la afección se considere estable.
    3. Carcinoma sin tratar en el AHD o antecedentes de carcinoma en los 5 años anteriores a la selección, a excepción del carcinoma escamocelular o basocelular cutáneo adecuadamente tratado.
    4. Esperanza de vida inferior a 6 meses, según la evaluación del investigador.
    5. Presencia actual o antecedentes conocidos de enfermedad hepática o renal clínicamente significativa que, en opinión del investigador, pueda afectar a la seguridad del sujeto o a la participación en el estudio.
    6. Tratamiento sistémico con un inmunomodulador, inmunosupresor o quimioterapia citotóxica en los 2 meses anteriores a la visita basal.
    E.5 End points
    E.5.1Primary end point(s)
    - Assessment of adverse events/serious adverse events
    - Absence of exon 73 in COL7A1 mRNA, detected by polymerase chain reaction (PCR)
    - Evaluación de los acontecimientos adversos/acontecimientos adversos graves
    - Ausencia del exón 73 en el ARNm del gen COL7A1, detectada mediante la reacción en cadena de la polimerasa (RCP)
    E.5.1.1Timepoint(s) of evaluation of this end point
    - AEs are assessed throughout the study
    - Exon skip is assessed after 1 week of treatment with IMP
    - Los AAs son evaluados a lo largo del estudio
    - El salto del exón se evalúa después de 1 semana de tratamiento con el PEI
    E.5.2Secondary 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)
    - Tamaño de la herida (área de superficie)
    - Gravedad de la herida evaluada mediante la escala de valoración subjetiva de la gravedad por parte del médico (Physician Subjective Assessment of Severity, PSAS) y la escala de valoración subjetiva del cambio por parte del médico (Physician Subjective Assessment of Change, PSAC)
    - Estado de las heridas evaluado mediante un cuestionario breve específico de lesiones (Short Wound Specific Questionnaire, SWSQ)
    - Estado de la cicatrización de las heridas
    - Aparición de (nuevas) ampollas en una herida curada
    - Niveles séricos de QR-313
    - Presencia de expresión de la proteína del colágeno de tipo VII, determinada mediante microscopía de inmunofluorescencia indirecta (IFI)
    - Presencia de fibrillas de anclaje determinada mediante microscopía electrónica de transmisión (MET)
    E.5.2.1Timepoint(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)
    - se harán fotografías semanales a lo largo del estudio con el propósito de los parámetros de curación de la herida y la resistencia de la piel
    - se miden los niveles séricos de QR-313 después de 1 semana de tratamiento con el PEI
    - la expresión de la proteína del colágeno de tipo VII a las 8 semanas después de la última dosis de PEI (FdE)
    - medida de las fibrillas de anclaje a las 8 semanas después de la última dosis de PEI (FdE)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Austria
    Canada
    Czech Republic
    France
    Germany
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 7
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Women of child bearing potential using contraception; Children from 2 to 18 years
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 8
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-09-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-30
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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