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    Summary
    EudraCT Number:2021-002532-23
    Sponsor's Protocol Code Number:ER004-CLIN01/F60082AI201
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-09-09
    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 number2021-002532-23
    A.3Full title of the trial
    A prospective, open-label, genotype-match controlled, multicenter clinical trial to investigate the efficacy and safety of intra-amniotic ER004 as a prenatal treatment for male subjects with X-linked hypohidrotic ectodermal dysplasia (XLHED)
    Ensayo clínico prospectivo, multicéntrico, abierto, controlado por compatibilidad genotípica para investigar la eficacia y la seguridad de ER004 intraamniótico como tratamiento prenatal en sujetos varones con displasia ectodérmica hipohidrótica ligada al cromosoma X (DEHLX)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open-label Phase 2 trial to investigate efficacy and safety of intra-amniotic administrations of ER004 in male subjects with X-linked hypohidrotic ectodermal dysplasia (XLHED)
    Ensayo clínico en fase II, abierto, para investigar la eficacia y la seguridad de la administración de ER004 intraamniótico en participantes varones con displasia ectodérmica hipohidrótica ligada al cromosoma X (DEHLX)
    A.4.1Sponsor's protocol code numberER004-CLIN01/F60082AI201
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/284/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorEspeRare Foundation
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPierre Fabre Médicament
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEspeRare Foundation
    B.5.2Functional name of contact pointER004-Clinical Use Information
    B.5.3 Address:
    B.5.3.1Street AddressCampus Biotech, Avenue Secheron 15
    B.5.3.2Town/ cityGeneva
    B.5.3.3Post code1202
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+34 968 92 94 29
    B.5.6E-mailinfo.er004@esperare.org
    B.Sponsor: 2
    B.1.1Name of SponsorPierre Fabre Médicament
    B.1.3.4CountryFrance
    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 supportPierre Fabre Médicament
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEspeRare Foundation
    B.5.2Functional name of contact pointER004-Clinical Use Information
    B.5.3 Address:
    B.5.3.1Street AddressCampus Biotech, Avenue Secheron 15
    B.5.3.2Town/ cityGeneva
    B.5.3.3Post code1202
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+34 968 92 94 29
    B.5.6E-mailinfo.er004@esperare.org
    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/3/05/334
    D.3 Description of the IMP
    D.3.1Product nameER004
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraamniotic use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot Available
    D.3.9.2Current sponsor codeER004
    D.3.9.3Other descriptive nameHuman immunoglobulin G1 constant region - human ectodysplasin-A1 receptor-binding domain fusion protein
    D.3.9.4EV Substance CodeSUB196780
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    X-linked hypohidrotic ectodermal dysplasia (XLHED)
    Displasia ectodérmica hipohidrótica ligada al cromosoma X (DEHLX)
    E.1.1.1Medical condition in easily understood language
    X-linked hypohidrotic ectodermal dysplasia (XLHED)
    Displasia ectodérmica hipohidrótica ligada al cromosoma X (DEHLX)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10072592
    E.1.2Term Hypohidrotic ectodermal dysplasia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of intra-amniotic administrations of ER004 on sweating ability in male XLHED subjects with a null mutation in EDA at 6 months of age, compared to untreated matched control subjects.
    Evaluar la eficacia de la administración intraamniótica de ER004 sobre la capacidad de sudoración en varones con DEHLX con una mutación anuladora en EDA a la edad de 6 meses, en comparación con sujetos testigo compatibles no tratados.
    E.2.2Secondary objectives of the trial
    To assess the efficacy of intra-amniotic administrations of ER004 on sweat pore density and dentition in male subjects with XLHED at 6 months of age
    To assess the efficacy of intra-amniotic administrations of ER004 in male subjects with XLHED up to 5 years of age
    To assess the safety and tolerability of intra-amniotic administrations of ER004 in male subjects with XLHED up to 6 months of age and in mothers up to 1 month after delivery
    To assess the long-term safety of intra amniotic administrations of ER004 in male subjects with XLHED up to 5 years of age
    To assess safety parameters in untreated relatives (controls)
    Evaluar la eficacia de la administración intraamniótica de ER004 en la densidad de poros sudoríparos y en la dentadura en varones con DEHLX a la edad de 6 meses
    Evaluar la eficacia de la administración intraamniótica de ER004 en varones con DEHLX hasta la edad de 5 años
    Evaluar la seguridad y tolerabilidad de la administración intraamniótica de ER004 en varones con DEHLX hasta la edad de 6 meses y en las madres hasta 1 mes después del parto
    Evaluar la seguridad a largo plazo de la administración intraamniótica de ER004 en varones con hasta la edad de 5 años
    Evaluar los parámetros de seguridad en familiares no tratados (testigos)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For mother : Adult mother with confirmed pregnancy no later than week 23+6 and genetically confirmed as carrier of an EDA mutation

    For fetus subject: Male Fetal subject with confirmed diagnosis of XLHED

    Untreated relative : Untreated male relative subject ages between 6 months and 60 years with the same EDA mutation as the treated subject
    Para la madre: Madre adulta con embarazo confirmado como máximo en la semana 23+6 y portadora de una mutación en EDA confirmada mediante prueba genética
    Para el sujeto fetal: Feto masculino con diagnóstico confirmado de DEHLX
    Familiar no tratado: familiar cercano no tratado con edades comprendidas entre los 6 meses y los 60 años con la misma mutación EDA que el sujeto tratado
    E.4Principal exclusion criteria
    For mother:
    Any evidence of active maternal infection associated with a risk of preterm birth and/or congenital anomalies of prenatal and postnatal risk to the child Documented maternal HIV infection.
    Any pre-existing maternal medical condition that increases the risk of preterm birth or increases the risk of a serious untoward event occurring to the mother during pregnancy.
    Any pregnancy disorder associated with an increased risk of preterm birth, and/or maternal, fetal or neonatal morbidity/mortality

    For fetal subject
    1.Second major anatomic anomaly (not related to the underlying XLHED) that contributes to a significant morbidity or mortality risk, or echocardiogram or ultrasonography or other findings that indicate a high risk of fetal demise or risk of preterm birth.
    2.Any condition other than XLHED (i.e., other forms of ectodermal dysplasia, large orofacial clefts) that is likely to have an impact on the number of tooth germs.
    3.Any other medical condition which in the opinion of the investigator would not allow for safe conduct of the study for the subject, or that would interfere with efficacy assessments (e.g., any disorders that lead to reduced fetal swallowing).

    Untreated Relative
    1.Carrier of an hypomorphic EDA mutation.
    2.Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists.
    3.Presence of an implanted device (e.g., defibrillator, neurostimulator, pacemaker).
    4.Previous treatment with the study intervention by any route of administration prior to study start.
    Para la madre:
    Cualquier prueba de infección materna activa asociada con un riesgo de parto prematuro o anomalías congénitas con riesgo prenatal y posnatal para el bebé. Infección por VIH materna confirmada.
    Cualquier enfermedad anterior de la madre que aumente el riesgo de parto prematuro o de que esta sufra un acontecimiento adverso grave durante el embarazo.
    Cualquier trastorno del embarazo asociado con un aumento del riesgo de parto prematuro o morbilidad/mortalidad materna, fetal o neonatal

    Para el sujeto fetal
    1.Otra anomalía anatómica de importancia (no secundaria a DEHLX) que contribuya a un riesgo notable de morbilidad o mortalidad, o un ecocardiograma o ecografía u otros hallazgos que indiquen un elevado riesgo de muerte fetal o riesgo de parto prematuro.
    2.Cualquier enfermedad aparte de DEHLX (p. ej., otras formas de displasia ectodérmica, labio leporino con fisura grande) que puedan influir en la cantidad de gérmenes dentales.
    3.Cualquier otra enfermedad que, en opinión del investigador, no permitiera la realización segura del estudio para el sujeto, o que pudiera interferir con la evaluación de la eficacia (p. ej., cualquier desorden que reduzca el tránsito fetal).

    Familiar no tratado
    1.Portador de una mutación hipomórfica en EDA.
    2.Hipersensibilidad conocida a la pilocarpina o a los agonistas muscarínicos similares a la pilocarpina.
    3. Presencia de un dispositivo implantado (p. ej., desfibrilador, neuroestimulador, marcapasos).
    4.Tratamiento previo con la intervención del estudio por cualquier vía de administración antes del inicio del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Mean sweat volume collected on both forearms after local stimulation with pilocarpine (pilocarpine-induced sweating).
    Volumen medio de sudor recogido en ambos antebrazos después de la estimulación local con pilocarpina (sudoración provocada por pilocarpina).
    E.5.1.1Timepoint(s) of evaluation of this end point
    For treated subject, mean sweat volume is collected at 6 months of age (V8) (corrected age for subjects born at < 37 weeks)
    Para el sujeto tratado, la media del volumen de sudor se recoge a los 6 meses de edad (V8) (edad corregida para los sujetos que nazcan con < 37 semanas)
    E.5.2Secondary end point(s)
    Key secondary endpoints:
    •Mean sweat pore density (number/cm2) determined by direct visualization with a VivaScope® at 6 months of age (V8) at 2 different sites on the soles of the feet
    •Dental development evaluated by the number of erupted teeth and tooth buds (palpable alveolar structures in the alveolar ridge) as determined by oral examination at 6 months of age (V8)

    Other secondary endpoints:
    •Mean sweat volume on both forearms measured from a pilocarpine-induced sweat test (at other timepoints than 6 months).
    •Sweat pore density (at other timepoints than 6 months).
    •Dentition (at other timepoints than 6 months).
    •Dry eye
    •Salivation assessment
    •Number of XLHED related hospitalizations
    •Assessment of eczema
    •Safety and tolerability assessments
    •Health-related quality of life assessments
    Criterios de valoración secundarios clave:
    •Densidad media de poros sudoríparos (número/cm2) determinada por visualización directa con VivaScope® a la edad de 6 meses (V8) en dos áreas diferentes en la zona plantar
    •Desarrollo dental evaluado por el número de dientes emergentes y brotes dentarios (estructuras alveolares palpables en el borde alveolar) determinado durante la exploración bucal a la edad de 6 meses (V8)

    Otros criterios de valoración secundarios:
    •Volumen medio de sudor en ambos antebrazos, medido durante una prueba de sudor provocada por pilocarpina (en momentos no correspondientes a los 6 meses).
    •Densidad de poros sudoríparos (en otros momentos no correspondientes a los 6 meses).
    •Xeroftalmia
    •Evaluación de la salivación
    •Número de hospitalizaciones relacionadas con la DEHLX
    •Evaluación de eccemas
    •Evaluaciones de la seguridad y la tolerabilidad
    •Evaluaciones de la calidad de vida relacionada con la salud
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 6 month of age for primary and key secondary endpoints
    A los 6 meses de edad para los criterios de valoración principal y secundarios clave
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    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) No
    E.7.1.1First administration to humans No
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Genotype-matched controlled
    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 EEA5
    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
    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
    The End of study is defined as the time point when the last treated subject has completed the last visit (V14) or the premature withdrawal visit.
    El final del estudio se define como el momento en que el último sujeto tratado haya completado la última visita (V14) o la visita de retirada anticipada.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years8
    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: 20
    F.1.1.1In Utero Yes
    F.1.1.1.1Number of subjects for this age range: 20
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Fetus and new born incapable to give their consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 11
    F.4.2.2In the whole clinical trial 20
    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)
    ER004 will not be administered to participants after the end of the study. Upon completion of the last study visit, participants will return to the care of their treating physician.
    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 Decision2021-10-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-28
    P. End of Trial
    P.End of Trial StatusOngoing
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