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    EudraCT Number:2017-002164-41
    Sponsor's Protocol Code Number:END-GH-2017
    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:2017-06-21
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2017-002164-41
    A.3Full title of the trial
    Growth hormone therapy in adults with Prader-Willi syndrome: Effect on muscle tone assessed by functional magnetic resonance imaging (fMRI) and its relation to muscle strenght and body composition.
    Tratamiento con hormona de crecimiento en el adulto con Síndrome de Prader-Willi: efecto sobre el tono muscular valorado mediante resonancia magnética funcional (RMf) y su relación con la fuerza muscular y la composición corporal
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study with adult patients with Prader-Willi syndrome, which evaluate the effect of growth hormone therapy on muscle tone and its relation with muscle strength and body composition assessed with imaging procedures.
    Estudio con pacientes adultos con síndrome de Prader-Willi, que pretende valorar el efecto del tratamiento con hormona del crecimiento sobre el tono muscular y su relación con la fuerza muscular y la composición corporal valorado mediante técnicas de imagen.
    A.4.1Sponsor's protocol code numberEND-GH-2017
    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 SponsorFundació Parc Taulí
    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 supportFundació Parc Taulí
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHospital Universitari Parc Taulí
    B.5.2Functional name of contact pointOficina de Recerca
    B.5.3 Address:
    B.5.3.1Street AddressParc Taulí, 1
    B.5.3.2Town/ citySabadell
    B.5.3.3Post code08202
    B.5.4Telephone number34937458451
    B.5.5Fax number34937175067
    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 Yes
    D. name Genotonorm Miniquick 0,2 mg
    D. of the Marketing Authorisation holderPFIZER, S.L.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Powder and solution for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN GROWTH HORMONE
    D.3.9.4EV Substance CodeSUB20678
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.2
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with Prader-Willi Syndrome (SPW) with Growth hormone deficit.
    Pacientes con síndrome de Prader-Willi con déficit de hormona de crecimiento
    E.1.1.1Medical condition in easily understood language
    Patients with Prader-Willi Syndrome (SPW) with Growth hormone deficit.
    Pacientes con síndrome de Prader-Willi con déficit de hormona de crecimiento
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10036476
    E.1.2Term Prader-Willi syndrome
    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 assess the muscle tone with functional magnetic resonance imaging (fMRI) in adult patients undergoing SPW with hormone deficit, before and after 12 months of GH treatment.
    Evaluar el tono muscular mediante resonancia magnética funcional (RMf) en adultos con SPW con déficit de hormona de crecimiento, antes y después de 12 meses de tratamiento con GH.
    E.2.2Secondary objectives of the trial
    1. To assess the effect of GH treatment on the muscle strength before and after 12 months of treatment.
    2. To assess the effect of GH treatment on the body composition before and after 12 months of treatment.
    3. To study if there are changes on different cognitives aspects before and after 12 months of treatment.
    4. To study if there are changes in some of the metabolism regulatory hormones (leptina and kisspeptina) and if they correlate with body composition changes.
    5. To study the sleeping architecture, the
    presence of respiratory sleep disorder and the sleeping quality, before and after 12 months of GH treatment.
    1. Evaluar el efecto del tratamiento con GH sobre la fuerza muscular antes y a los 12 meses de tratamiento.
    2. Evaluar el efecto del tratamiento con GH sobre la composición corporal antes y a los 12 meses de tratamiento.
    3. Estudiar si hay cambios en diferentes aspectos cognitivos antes y a los 12 m de tratamiento
    4. Estudiar si se producen cambios en algunas hormonas reguladoras del metabolismo (leptina y kisspeptina) y si se correlacionan con los cambios en la composición corporal.
    5. Estudiar la arquitectura del sueño, la presencia de trastorno respiratorio del sueño y la calidad del sueño, antes y a los 12 m de tratamiento con GH.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Patients with PWS ≥ 18 years
    • Patients who have demonstrated a GH deficiency in at least one of the
    two GH stimuli tests performed in routine clinical practice
    • Patients (or their legal guardians if they are legally incapacitated) who
    have signed informed consent to participate in the study. In the case of
    patients legally incapacitated, they will give their assent in participating.
    • Pacientes con SPW ≥ 18 años
    • Pacientes en los que se haya demostrado un déficit de GH en al menos una de estas dos pruebas de estímulo de GH realizadas en la práctica clínica habitual
    Se considera que existe un déficit de GH si:
    Pico de GH en prueba de estimulación con GHRH+ arginina:
    18 a 25 años: <19 ng/ml
    >25 años: según IMC.
    IMC <25: <11 ng/ml
    IMC 25-30:<8 ng/ml
    IMC >30: < 4 ng/ml
    Pico de GH en la prueba de estimulación con glucagón < 3 ng/ml
    • Pacientes (o sus tutores legales en el supuesto que estén incapacitados legalmente) que hayan firmado su consentimiento informado para participar en el estudio. En el caso de los pacientes incapacitados legalmente, darán su asentimiento en participar.
    E.4Principal exclusion criteria
    • Contraindications for MRI
    • Inability to stand still during MRI
    • Impossibility to understand the paradigm to be realized during the MRI
    • Severe obesity, uncontrolled diabetes, severe untreated obstructive
    sleep apnea, active cancer and active psychosis
    • Patients who present any other contraindication to treatment with GH
    according to the data sheet.
    • Any circumstance that at the discretion of the physician may pose a
    risk or clinical harm to the patient's participation in the study or
    interfere with the assessments of the same.
    • Contraindicaciones para RMf
    • Imposibilidad de estarse quieto durante la realización de la RMf
    • Imposibilidad de entender el paradigma a realizar durante la RMf
    • Obesidad severa, diabetes no controlada, apnea obstructiva grave del sueño no tratada, cáncer activo y psicosis activa
    • Pacientes que presenten cualquier otra contraindicación para recibir tratamiento con GH según la ficha técnica.
    • Cualquier circunstancia que a criterio del médico le pueda suponer un riesgo o perjuicio clínico la participación del paciente en el estudio o que interfiera en las valoraciones del mismo.
    E.5 End points
    E.5.1Primary end point(s)
    Muscle tone with functional magnetic resonance imaging (fMRI)
    Tono muscular mediante resonancia magnética funcional (RMf)
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    12 meses
    E.5.2Secondary end point(s)
    1. Study of muscle strength
    2. Study of body composition
    3. Study of bone mineral density
    4. Mental and cognitive assessment
    5. Polysomnography
    6. Hormonal study
    7. Safety
    1. Estudio de la fuerza muscular
    2. Estudio de composición corporal
    3. Estudio de densidad mineral ósea
    4. Evaluación mental y cognitiva
    5. Polisomnografía
    6. Estudio hormonal
    7. Seguridad
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    12 meses
    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 No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind Yes
    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 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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    Last patient last visit
    Ultima visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    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 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. of subjects incapable of giving consent
    Some patients are not capable of giving nformed consent
    Algunos pacientes no están capacitados
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    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)
    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 Decision2017-07-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-27
    P. End of Trial
    P.End of Trial StatusOngoing
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