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    EudraCT Number:2010-020199-45
    Sponsor's Protocol Code Number:MOR-005
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-04-09
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2010-020199-45
    A.3Full title of the trial
    A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)

    Extensión de un estudio multicéntrico, multinacional para evaluar la eficacia y seguridad a largo plazo de BMN 110 en pacientes con mucopolisacaridosis IVA (síndrome de Morquio A)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    A.4.1Sponsor's protocol code numberMOR-005
    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 SponsorBioMarin Pharmaceutical Inc.
    B.1.3.4CountryUnited States
    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 supportBioMarin Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Pharmaceutical Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address105 Digital Drive
    B.5.3.2Town/ cityNovato, CA
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number080023436421960043420
    B.5.5Fax number+1415884 6944
    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/3/09/657
    D.3 Description of the IMP
    D.3.1Product namerecombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
    D.3.2Product code BMN 110
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot Available
    D.3.9.1CAS number 9025-60-9
    D.3.9.2Current sponsor coderhGALNS
    D.3.9.3Other descriptive namerecombinant human N-acetylgalactosamine-6-sulfatase, BMN 110
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Mucopolysaccharidosis Type IVA
    Mucopolisacaridosis IVA
    E.1.1.1Medical condition in easily understood language
    Morquio A Syndrome
    Síndrome de Morquio A
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level PT
    E.1.2Classification code 10028095
    E.1.2Term Mucopolysaccharidosis IV
    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 the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA.
    Evaluar la seguridad y la eficacia a largo plazo de la administración de 2,0 mg/kg/todas las
    semanas y 2,0 mg/kg/cada dos semanas de BMN 110 en pacientes con MPS IVA.
    E.2.2Secondary objectives of the trial
    - To evaluate the long-term effect of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow on changes in biochemical markers of inflammation and bone and cartilage metabolism, in patients with MPS IVA.
    - To evaluate patient perception of impairment and improvement at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA.
    - Evaluar el efecto a largo plazo de la administración de 2,0 mg/kg/todas las semanas y de 2,0 mg/kg/cada dos semanas de BMN 110 sobre los cambios en los marcadores
    bioquímicos de inflamación y el metabolismo de los huesos y cartílagos en pacientes con
    MPS IVA.
    - Evaluar la percepción de los pacientes en cuanto a deterioro o mejoría con 2,0 mg/kg todas
    las semanas y 2,0 mg/kg/cada dos semanas en pacientes con MPS IV A.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ? Must have completed MOR-004.
    ? Is willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorized representative, after the nature of the study has been explained, and prior to performance of research-related procedures.
    ? If sexually active, must be willing to use an acceptable method of contraception while participating in the study.
    ? If female, of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests done during the study.
    - Debe haber completado MOR-004.
    - Estar dispuesto y poder otorgar un consentimiento informado firmado por escrito. O, en el caso de los pacientes menores de 18 (u otra edad según lo defina la ley o normativa regional), otorgar el asentimiento por escrito (si fuera necesario) y hacer que un representante legalmente autorizado firme el consentimiento informado, después de que se haya explicado la naturaleza del estudio y antes de la realización de los procedimientos relacionados con la investigación.
    - Si lleva una vida sexual activa, debe estar dispuesto a usar un método aceptado de
    anticoncepción mientras participa en el estudio.
    - Si es mujer con capacidad de concebir debe tener un resultado negativo en la prueba de embarazo al inicio y estar dispuesta a realizarse pruebas de embarazo adicionales durante el estudio.
    E.4Principal exclusion criteria
    ? Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or partner) at any time during the study.
    ? Has used any investigational product (other than BMN 110 in MOR-004), or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments.
    ? Was enrolled in a previous BMN 110 study, other than MOR-004.
    ? Has a concurrent disease or condition, including but not limited to, symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation, or pose a safety risk, as determined by the Investigator.
    ? Has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
    - Está embarazada o en periodo de lactancia, al inicio, o tiene planes de embarazo (personal
    o su pareja) en cualquier momento durante el estudio.
    - Ha usado cualquier producto en investigación (que no sea BMN 110 en MOR-004), o un
    dispositivo médico en investigación, dentro de los 30 días previos al inicio; o se requiere
    que use un agente en investigación antes de completar todas las evaluaciones del estudio
    - Participó en un estudio de BMN 110 anterior, distinto de MOR-004.
    - Tiene una enfermedad o afección concurrente, por ejemplo, inestabilidad sintomática de la
    columna cervical, compresión clínicamente significativa de la columna dorsal o
    enfermedad cardíaca grave que interferiría con la participación en el estudio, o representaría un riesgo para la seguridad, según la determinación del investigador.
    - Tiene una afección que, en opinión del investigador, coloca al paciente en alto riesgo de
    cumplir con el tratamiento de manera deficiente o de no completar el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Variables:
    ? endurance tests:
    ? 6 minute walk (6MW) test
    ? 3 minute stair climb (3MSC) test
    ? urine KS concentration (normalized to creatinine)
    ? respiratory function tests:
    ? forced expiratory time (FET)
    ? forced expiratory volume in 1 second (FEV1)
    ? forced inspiratory vital capacity (FIVC)
    ? forced vital capacity (FVC)
    ? maximum voluntary ventilation (MVV)
    ? anthropometric measurements (standing height, length, sitting height, and weight)
    ? skeletal X-rays of lumbar spine and lower extremity
    ? MPS Health Assessment Questionnaire
    ? audiometry examinations
    Safety Variables:
    ? adverse events (AEs)
    ? standard clinical laboratory tests (serum chemistry, hematology, and urinalysis)
    ? vital signs
    ? echocardiograms
    ? electrocardiograms (ECGs)
    ? routine physical examinations (including standard neurologic examination)
    ? concomitant medications
    ? immunogenicity tests
    ? cervical spine (flexion?extension) X-rays
    Variables de eficacia:
    - pruebas de resistencia: prueba de caminata de 6 minutos (6MW) y prueba de subida de escaleras de 3 minutos (3MSC)
    - concentración de KS en orina (normalizada por creatinina)
    - pruebas de la función respiratoria:
    – tiempo espiratorio forzado (TEF)
    – volumen espiratorio forzado en 1 segundo (VEF1)
    – capacidad vital inspiratoria forzada (CVIF)
    – capacidad vital forzada (CVF)
    – ventilación voluntaria máxima (VVM)
    mediciones antropométricas (estatura de pie, longitud, estatura sentado y peso)
    - radiografías del esqueleto de la columna lumbar y las extremidades inferiores (las radiografías de las extremidades inferiores se realizan solamente para pacientes de ≤ 20 años de edad)
    - Cuestionario de evaluación de salud de MPS
    exámenes de audiometría
    Variables de seguridad:
     acontecimientos adversos (AA)
     análisis de laboratorio clínico estándar (bioquímica sérica, hematología y análisis de orina)
     constantes vitales
     ecocardiogramas
     electrocardiogramas (ECG)
     exploraciones físicas de rutina, incluido examen neurológico estándar
     medicación concomitante
     pruebas de inmunogenicidad
     radiografías de columna cervical (flexión–extensión)
    E.5.1.1Timepoint(s) of evaluation of this end point
    ? 6MW test, 3MSC test: baseline, wk 12, every 24 wks in Part 1, every 48 wks in Part 2
    ? respiratory function tests: baseline, every 24 wks in Part 1, every 48 wks in Part 2
    ? anthropometric measurements, MPS Health Assessment Questionnaire, audiometry examinations: baseline, every 24 wks in Part 1 and Part 2
    ? adverse events, vital signs, concomitant medications: baseline, weekly in Part 1, weekly or every other week in Part 2
    ? echocardiograms, electrocardiograms: baseline, every 48 wks in Part 2
    ? routine physical examinations, immunogenicity tests, standard clinical laboratory tests, urine KS concentration: baseline, every 12 wks in Part 1, every 24 wks in Part 2
    ? x-rays of cervical spine, lumbar spine and lower extremity: baseline, every 72 wks in Part 2
    -pruebas de 6MWy3MSC: línea de base,12 semanas(S),cada 24S en la Parte 1,cada 48S en la Parte 2
    -pruebas de función respiratoria: línea de base, cada 24S en la Parte 1,cada 48S en de la Parte 2
    -medidas antropométricas, MPSCuestionario de Evaluación de Salud, exámenes de audiometría: línea de base,cada 24S en la Parte 1yParte 2
    -eventos adversos, signos vitales, medicación concomitante: inicio, cada S en la Parte 1, cada S o cada 2S en la Parte 2
    -eco y electrocardiogramas: línea de base, cada 48S de la Parte 2
    -exámenes físicos de rutina, pruebas de inmunogenicidad y estándar de lab clínico, orina Concentración KS: línea de base, cada 12S en la Parte 1, cada 24S en la Parte 2
    -radiografías de la columna cervical,lumbar y extremidades inferiores: línea de base, cada 72S en la Parte 2
    E.5.2Secondary end point(s)
    Exploratory Variables:
    ? blood inflammatory biomarkers
    ? blood biochemical markers of bone and cartilage metabolism
    ? Patient Impression Questionnaire (PIQ)
    Variables exploratorias:
     biomarcadores inflamatorios en sangre
     marcadores bioquímicos sanguíneos del metabolismo de los huesos y cartílagos
     Cuestionario de Impresiones del paciente (CIP)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Exploratory Variables:
    ? blood inflammatory biomarkers, and blood biochemical markers of bone and cartilage metabolism: baseline, every 24 wks in Part 1 and Part 2
    ? Patient Impression Questionnaire (PIQ) will be done at Baseline and only at Week 24 within 1 hour (± 15 minutes) following the second 3MSC test.
    Variables exploratorias:
    biomarcadores inflamatorios en sangre, y la sangre marcadores bioquímicos del metabolismo óseo y cartílago: línea de base, cada 24 semanas en la Parte 1 y Parte 2
    ? Cuestionario Impression Paciente (PAA) se hará en el momento basal y sólo en la semana 24 en 1 hora (± 15 minutos) después de la segunda prueba 3MSC.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    Part 1: randomised double blind until optimal BMN 110 dose; Part 2: open-label BMN 110 treatment
    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. description
    2.0 mg/kg/qw or 2.0 mg/kg/qow BMN110
    E.8.2.4Number of treatment arms in the trial2
    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 EEA19
    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
    Korea, Republic of
    Saudi Arabia
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months8
    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: 110
    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. of subjects for this age range: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 70
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 Yes
    F. of subjects incapable of giving consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 90
    F.4.2.2In the whole clinical trial 162
    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)
    Treatment will be provided through this extension study for up to 240 weeks or until one of the following occurs: the patient withdraws consent and discontinues from the study, the patient is discontinued from the study at the discretion of the Investigator, or the study is terminated.
    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 Decision2013-07-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-04-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-06-16
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