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    The EU Clinical Trials Register currently displays   43861   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2011-004355-40
    Sponsor's Protocol Code Number:rhLAMAN-04
    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:2012-04-17
    Trial results View 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 number2011-004355-40
    A.3Full title of the trial
    A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.
    Ensayo multicéntrico y sin enmascaramiento de la seguridad y eficacia a largo plazo de Lamazym para el tratamiento de enfermos de alfamanosidosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.
    Ensayo multicéntrico y sin enmascaramiento de la seguridad y eficacia a largo plazo de Lamazym para el tratamiento de enfermos de alfamanosidosis
    A.3.2Name or abbreviated title of the trial where available
    Phase 2b
    Fase 2b
    A.4.1Sponsor's protocol code numberrhLAMAN-04
    A.5.4Other Identifiers
    Name:EudraCTNumber:2011-004355-40
    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 SponsorZymenex A/S
    B.1.3.4CountryDenmark
    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 supportEU FP7 ALPHA-MAN
    B.4.2CountryEuropean Union
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationZymenex A/S
    B.5.2Functional name of contact pointCEO
    B.5.3 Address:
    B.5.3.1Street AddressRoskildevej 12C
    B.5.3.2Town/ cityHillerod
    B.5.3.3Post codeDK-3400
    B.5.3.4CountryDenmark
    B.5.4Telephone number+4548250054
    B.5.5Fax number+4548251054
    B.5.6E-mailzxmail@zymenex.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/3/04/260
    D.3 Description of the IMP
    D.3.1Product nameLamazym
    D.3.2Product code rhLAMAN
    D.3.4Pharmaceutical form Powder for 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.3.9.2Current sponsor coderhLAMAN
    D.3.9.3Other descriptive namealfa-manosidadas humana recombinante
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1.8 to 2.2
    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.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
    Treatement of Alpha-Mannosidosis
    Tratamiento de alfa-manosidosis
    E.1.1.1Medical condition in easily understood language
    The scientific study is a treatment process lasting 6 months with patients suffering from the disease known as alpha-mannosidosis.
    Tratamiento de la alfa-manosidosis, una enfermedad sumamente rara
    causada por un defecto genético
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The overall objective of this trial is the evaluation of long-term efficacy, safety and tolerability of Lamazym treatment in patients with alpha-Mannosidosis.
    The primary objectives of the trial are:
    Evaluation of long-term efficacy from baseline of Lamazym on reduction of the biomarker Oligosaccharides in blood and CSF and an improvement in the 3 minute stair climb, 6 minute walk test and pulmonary function from baseline.
    El objetivo general de este ensayo es la evaluación de la tolerabilidad, la seguridad y la eficacia a largo plazo del tratamiento con Lamazym en enfermos de alfa-manosldosis.
    Los objetivos principales del ensayo son:
    Evaluación de la eficacia a largo plazo desde el Inicio de Lamazym sobre
    la reducción del blomarcador oligosacáridos en la sangre y el LCR, y una
    mejora en las pruebas de 3 minutos subiendo escaleras y 6 minutos
    caminando y la actividad pulmonar desde el inicio
    E.2.2Secondary objectives of the trial
    The secondary objectives of the trial are:
    To evaluate the long-term safety profile of Lamazym
    Evaluation of efficacy of Lamazym on:
    o The clinical parameters BOT2 and Hearing
    o The CSF biomarkers (Tau, NFLp, GFAp)
    o The cognitive development tested by Leiter R
    o The CNS improvement measured with MRI/MRS
    Los objetivos secundarios del ensayo son:
    - Evaluación de la toxicidad de Lamazym a largo plazo.
    - Evaluación de la eficacia de Lamazym en:
    *Los parámetros clínicos BOT2 y Hearing;
    *Los biomarcadores del LCR ( Tau, PNFL, PGFA);
    *El desarrollo cognitivo probado con Leiter R;
    *La mejora del SNC medida mediante RM/ERM
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)
    Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
    The subject and his/her guardian(s) must have the ability to comply with the protocol
    El paciente deberá haber participado en el ensayo de fase I (número EudraCT: 2010-022084-36) y en el de fase IIa (número EudraCT: 2010-022085-26).
    El paciente o su(s) tutor( es) autorizados legalmente deberán firmar y entregar el consentimiento informado antes de la realización de actividades relacionadas con el ensayo (las actividades relacionadas con el ensayo son todos aquellos procedimientos que no se habrían realizado durante el tratamiento normal del paciente).
    El paciente y su(s) tutor( es) deberán tener la capacidad para cumplir el protocolo
    E.4Principal exclusion criteria
    The subject cannot walk without support
    Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
    History of bone marrow transplantation
    Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
    Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial.
    Pregnancy
    Psychosis within the last 3 months
    Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial
    Participation in other interventional trials testing IMP except for studies with Lamazym
    El paciente no puede caminar sin apoyo.
    Presencia de anomalía cromosómica conocida y síndromes que afecten al desarrollo psicomotor distintos de la alfa-manosidosis.
    Antecedentes de trasplante de médula ósea.
    Presencia de enfermedades cardiovasculares, hepáticas, pulmonares o renales conocidas y clínicamente significativas u otras afecciones que, en opinión del investigador, descartarían la participación en el ensayo.
    Cualquier otra afección o enfermedad intercurrente grave o circunstancia atenuante que, en opinión del investigador, descartaría la participación en el ensayo.
    Embarazo.
    Psicosis en los últimos tres meses.
    Cirugía mayor prevista que, en opinión del investigador, descartaría la participación en el ensayo.
    Participación en otros ensayos de intervención que prueben el Producto en Investigación, excepto los éstudlos con Lamazym
    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoint evaluation as change from baseline on Reduction of Oligosaccharides in blood serum and CSF and an improvement in the 3 minute stair climb, 6 minute walk test and pulmonary function
    Evaluación del criterio principal de valoración como cambio desde el
    inicio de la Reducción de los oligosacáridos en el suero sanguíneo y el LCR y una
    mejora en las pruebas de 3 minutos subiendo escaleras y 6 minutos
    caminando, así como en la actividad pulmonar
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy will be assessed at the Danish site as interim (January/February 2012) and as end evaluation (minimum 26 weeks post trial initiation).
    La eficacia se evaluará en el centro danés en forma de evaluación
    intermedia (enero-febrero de 2012) y final (un mínimo de 26 semanas
    tras el inicio del ensayo).
    E.5.2Secondary end point(s)
    Safety Endpoints measured throughout the trial of:
    Development of adverse events
    Development of clinically significant changes in vital signs and change in physical examination
    Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
    Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies

    Secondary Efficacy Endpoints evaluation as change from baseline on:
    CNS parameters: Reduced concentration of mannose-rich oligosaccharides in brain tissue as measured by MRS visual score, reduction of MRI diffusion coefficient in white matter, gray matter and centrum semi oval and Improved Cerebrospinal fluid neuro-degeneration biomarker (Tau, NFLp, GFAp)
    Clinical parameters: Improved functional capacity on the BOT2 and reduced hearing loss
    Cognitive ability: Improved age equivalence with Leiter R
    In addition, the CHAQ, filled by the subject?s guardian, will be evaluated
    Criterios de valoración de la seguridad medidos a lo largo del ensayo de:
    aparición de acontecimientos adversos;
    aparición de cambios clínicamente significativos en las constantes vitales y cambio en la exploración física;
    aparición de cambios clínicamente significativos en las variables de laboratorio clínico (hematología, bioquímica y análisis de orina);
    aparición de anticuerpos contra rhLAMAN y de anticuerpos neutralizantes o inhibidores.

    Evaluación de los criterios secundarios de valoración de la eficacia como cambio desde el inicio en:
    Parámetros del SNC: reducción de la concentración de oligosacáridos ricos en manosa en el tejido cerebral medida mediante el índice visual de ERM, reducción del coeficiente de difusión de RM en la sustancia blanca, la sustancia gris y el centro semioval, y mejora del biomarcador de
    neurodegeneración del líquido cefalorraquídeo (Tau, PNFL, PGFA).
    Parámetros clínicos: mejora de la capacidad funcional de la BOT2 y reducción de la hipoacusia.
    Capacidad cognitiva: mejora de la equivalencia de edad con Leiter R.
    Además, se evaluará el CHAQ, cumplimentado por el tutor del paciente
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety will be assessed at every visit.
    Efficacy will be assessed at the Danish site as interim (January/February 2012) and as end evaluation (minimum 26 weeks post trial initiation).
    La eficacia se evaluará en el centro danés en forma de evaluación intermedia (enero-febrero de 2012) y final (un mínimo de 26 semanas tras el inicio del ensayo).
    La evaluación de los criterios de evaluación de seguridad se llevará a cabo a lo largo de la prueba
    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 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.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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 No
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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
    Última Visita del Último Paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months6
    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: 8
    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: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    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 Yes
    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
    One female of 15-17 year is tested monthly (HCG)
    Due to the disease related mental retardation the subjects are under gardianship of the parents
    Se evalúa mensualmente a una mujer de 15-17 años (HCG).
    Debido al retraso mental asociado a la enfermedad, los pacientes se
    hallan bajo la custodia de sus padres
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 9
    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)
    Given no safety concerns have arisen, the subjects will be offered to continue to receive the investigational medicinal product (IMP) on a post-study care base in the home country until the product is available on the market or until the sponsor decides to terminate the project (e.g. for safety reasons).
    Siempre que no se hayan producido problemas de seguridad, se
    ofrecerá a los pacientes la posibilidad de seguir recibiendo el producto
    en fase de investigación clínica en la modalidad de uso compasivo
    en el país de origen hasta que el producto esté disponible en el
    mercado o hasta que el promotor decida cancelar el proyecto (p. ej. por
    motivos de seguridad).
    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 Decision2012-04-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-07
    P. End of Trial
    P.End of Trial StatusCompleted
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