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    Summary
    EudraCT Number:2015-004438-93
    Sponsor's Protocol Code Number:CT-ORZY-NPC002
    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:2016-07-15
    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 number2015-004438-93
    A.3Full title of the trial
    Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C
    Estudio clínico prospectivo, randomizado, doble ciego, controlado con placebo, de
    arimoclomol en pacientes con diagnóstico de enfermedad de Niemann-Pick tipo C
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to investigate the study drug Arimoclomol in a double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C.
    Estudio clínico prospectivo, randomizado, doble ciego, controlado con placebo, de
    arimoclomol en pacientes con diagnóstico de enfermedad de Niemann-Pick tipo C
    A.3.2Name or abbreviated title of the trial where available
    Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C.
    Investigar Arimoclomol con Niemann Pick tipo C pacientes.
    A.4.1Sponsor's protocol code numberCT-ORZY-NPC002
    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 SponsorOrphazyme ApS
    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 supportOrphazyme ApS
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOrphazyme ApS
    B.5.2Functional name of contact pointClinical Trial Manager
    B.5.3 Address:
    B.5.3.1Street AddressOle Maaløes Vej 3
    B.5.3.2Town/ cityCopenhagen N
    B.5.3.3Post codeDK-2200
    B.5.3.4CountryDenmark
    B.5.4Telephone number00452160 6341
    B.5.6E-mailkam@orphazyme.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/3/14/1376
    D.3 Description of the IMP
    D.3.1Product nameArimoclomol
    D.3.2Product code _
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNArimoclomol
    D.3.9.1CAS number _
    D.3.9.2Current sponsor codeOR0003
    D.3.9.3Other descriptive name_
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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.IMP: 2
    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/3/14/1376
    D.3 Description of the IMP
    D.3.1Product nameArimoclomol
    D.3.2Product code _
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNArimoclomol
    D.3.9.1CAS number _
    D.3.9.2Current sponsor codeOR0003
    D.3.9.3Other descriptive name_
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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.IMP: 3
    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/3/14/1376
    D.3 Description of the IMP
    D.3.1Product nameArimoclomol
    D.3.2Product code _
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNArimoclomol
    D.3.9.1CAS number _
    D.3.9.2Current sponsor codeOR0003
    D.3.9.3Other descriptive name_
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Niemann Pick disease type C
    Niemann Pick tipo C
    E.1.1.1Medical condition in easily understood language
    Niemann-Pick Type C is a rare and inherited disease in which quantities of fatty substances accumulate in the brain and other organs. The brain, central nervous system, liver and spleen are affected.
    Estudio clínico prospectivo, randomizado, doble ciego, controlado con placebo, de
    arimoclomol en pacientes con diagnóstico de enfermedad de Niemann-Pick tipo C
    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 19.0
    E.1.2Level PT
    E.1.2Classification code 10029403
    E.1.2Term Niemann-Pick disease
    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 therapeutic response to arimoclomol versus placebo, both in addition best available standard of care, at 12 months.
    Evaluar la respuesta terapéutica del arimoclomol frente al placebo, ambos
    sumados al mejor tratamiento de referencia habitual disponible, a los 12 meses.
    E.2.2Secondary objectives of the trial
    ?To evaluate the therapeutic response to arimoclomol through clinical, biological and imaging assessments at 6 and 12 months;
    ?To evaluate the long term therapeutic response (clinical and biological assessments) at 18 and 24 months and hereafter annually until study end;
    ?To evaluate the safety of arimoclomol.
    • Evaluar la respuesta terapéutica al arimoclomol a través de evaluaciones
    clínicas, biológicas y de imagen a los 6 y 12 meses.
    • Evaluar la respuesta terapéutica a largo plazo (evaluaciones clínicas y
    biológicas) a los 18 y 24 meses y, partir de ahí, anualmente hasta la conclusión
    del estudio.
    • Evaluar la seguridad del arimoclomol.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    In order to be eligible for inclusion into this study, each patient must fulfil all of the following inclusion criteria at enrolment of the study.
    ?EITHER
    NPC patients who have entered the CT ORZY NPC 001 study when aged from 2 years to 18 years and 11 months; and who have completed Visit 2 (EOS) of the CT ORZY NPC 001 study.
    OR
    NPC patients who did not enter or complete the CT ORZY NPC 001 study but are fulfilling all of criteria listed below:
    oDiagnosis of NPC1 or NPC2;
    oNPC diagnosis confirmed by:
    -Genetically confirmed (deoxyribonucleic acid [DNA] sequence analysis) by mutations in both alleles of NPC1 or NPC2,
    OR
    -Mutation in only one allele of NPC1 or NPC2 plus either positive filipin staining or elevated cholestane triol/oxysterols (>2 x upper limit of normal).
    oMales and females aged from 2 years to 18 years and 11 months;
    oTreated or not treated with miglustat;
    oIf a patient is under prescribed treatment with miglustat, it has to be under stable dose of the medication for at least 6 continuous months prior to inclusion in the CT ORZY NPC 002 study;
    oIf a patient has been discontinued from prescribed treatment with miglustat, they must have been discontinued for at least 3 continuous months prior to inclusion in the CT-ORZY-NPC-002 study;
    oBody mass index (BMI) Z score ? -2 SD (standard deviation) for age, according to the World Health Organisation (WHO) standards;
    oPresenting at least one neurological symptom of the disease (for example, but not limited to, hearing loss, vertical supranuclear gaze palsy, ataxia, dementia, dystonia, seizures, dysarthria, or dysphagia);
    oAbility to walk either independently or with assistance.
    ?Written informed consent (and assent if appropriate to local laws and regulations) prior to any study-related procedures;
    ?Willing to participate in all aspects of trial design including blood sampling (PK, blood biomarkers and safety labs), skin biopsies and imaging (ultrasonography of the liver and spleen);
    ?Ability to travel to the corresponding clinical trial site at the scheduled visit times for evaluation and follow-up;
    ?All sexually active female patients of child-bearing potential (post-menarchal) must use highly effective contraception during the study and until 1 week after the last dose of IMP.
    Highly effective birth control methods include: Combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable); intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; and vasectomised partner.

    All sexually active male patients with female partners of child-bearing potential (post-menarchal) must use a condom with or without spermicide in addition to the birth control used by their partners during the study and until 3 months after the last dose of IMP.
    Sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the study and for 1 week after the last dose of IMP (for female patients of child-bearing potential) and for 3 months after the last dose of IMP (for male patients with female partners of child-bearing potential). The reliability of sexual abstinence needs to be evaluated by the Investigator in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient.
    ?Ability to comply with the protocol-specified procedures/evaluations and scheduled visits.
    Para ser candidato a la inclusión en este estudio, todos los pacientes deben cumplir
    con los siguientes criterios de inclusión en el momento de la inclusión en el estudio.
    • O BIE
    Pacientes con NP-C que han participado en el estudio CT-ORZY-NPC-001
    cuando tenían una edad de entre 2 años y 18 años y 11 meses, y que han
    completado la Visita 2 (FE) del estudio CT-ORZY-NPC-001.
    O
    Pacientes con NP-C que no participaron o completaron el estudio CTORZY-
    NPC-001, pero cumplen todos los criterios que se indican a
    continuación:
    o Diagnóstico de NPC1 o NPC2.
    o Diagnóstico de NPC confirmado:
    - Genéticamente (análisis de secuencias de ácido
    desoxirribonucleico [ADN]) mediante mutaciones en
    ambos alelos del NPC1 o del NPC2.
    O BIEN
    - Mediante mutación de un solo alelo del NPC1 o del
    NPC2 más tinción con filipina positiva o
    concentraciones elevadas de
    oxisteroles/colestanotriol (>2 x el límite superior de
    normalidad).
    o Pacientes de ambos sexos con edades de entre 2 años y 18 años y
    11 meses.
    o Con o sin tratamiento con miglustat.
    o Si un paciente se encuentra en tratamiento prescrito con miglustat,
    deberá haberse mantenido con una dosis estable de la medicación
    durante al menos 6 meses consecutivos antes de su inclusión en el
    estudio CT-ORZY-NPC-002.
    o Si un paciente suspende el tratamiento prescrito con miglustat,
    deberá haberlo suspendido durante al menos 3 meses consecutivos
    antes de su inclusión en el estudio CT-ORZY-NPC-002.
    o Índice de masa corporal (IMC) con la puntuación Z R -2 de desviación estándar (DE) para la edad de acuerdo con los
    estándares de la Organización Mundial de la Salud (OMS).
    o Presenta al menos un síntoma neurológico de la enfermedad (por
    ejemplo, entre otros, pérdida de audición, parálisis visual
    supranuclear vertical, ataxia, demencia, distonía, ataques
    epilépticos, disartria o disfagia).
    o Capacidad para caminar de forma autónoma o con ayuda.
    • Consentimiento informado por escrito (y asentimiento si corresponde de
    acuerdo con la legislación local) previo a cualquier procedimiento
    relacionado con el estudio.
    • Voluntad de participar en todos los aspectos del diseño del ensayo,
    incluidas muestras de sangre (FC, biomarcadores sanguíneos y pruebas de
    seguridad de laboratorio), biopsias de piel y técnicas de imagen
    (ultrasonografía de hígado y bazo).
    • Posibilidad de desplazarse hasta el centro clínico correspondiente en el
    momento de las visitas programadas para la evaluación y el seguimiento.
    • Todas las mujeres fértiles (post-menarquía), sexualmente activas deben
    utilizar métodos anticonceptivos eficaces durante el estudio y hasta 1
    semana después de tomar la última dosis del PEI.
    Entre los métodos anticonceptivos eficaces se incluyen los siguientes:
    anticonceptivos hormonales combinados (con estrógenos y progestágenos)
    asociados a la inhibición de la ovulación (orales, intravaginales o
    transdérmicos); anticonceptivos hormonales solo de progestágenos
    asociados a inhibición de la ovulación (orales, inyectables o implantables);
    dispositivo intrauterino (DIU); sistema intrauterino liberador de hormonas,
    oclusión tubárica bilateral o vasectomía de la pareja.
    Todos los pacientes varones sexualmente activos con parejas fértiles (postmenarquía)
    deben utilizar preservativos con o sin espermicida, además del
    método anticonceptivo que utilicen sus parejas, durante el estudio y hasta 3
    meses después de tomar la última dosis del PEI.
    La abstinencia sexual se considera un método anticonceptivo muy eficaz
    solo si se define como la abstención de mantener relaciones heterosexuales
    durante el estudio y durante 1 semana después de tomar la última dosis del
    PEI (para las mujeres fértiles), y durante 3 meses después de tomar la
    última dosis del PEI (para hombres con parejas fértiles). El investigador
    deberá evaluar la fiabilidad de la abstinencia sexual en relación con la
    duración del ensayo clínico y el estilo de vida habitual y preferido del
    paciente.
    • Capacidad para cumplir con los procedimientos/evaluaciones y las visitas
    programadas especificados en el protocolo.
    E.4Principal exclusion criteria
    Each of the following criteria will be taken as reason for exclusion from participation in the trial:
    ?Recipient of a liver transplant or planned liver transplantation;
    ?Severe liver insufficiency (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal for age and gender (central laboratory assessment);
    ?Renal insufficiency, with serum creatinine level greater than 1.5 times the upper limit of normal (central laboratory assessment);
    ?Known or suspected allergy or intolerance to the IMP (arimoclomol or constituents);
    ?In the opinion of the Investigator, the patient's clinical condition does not allow for the required blood collection and/or skin biopsies as per the protocol-specified procedures;
    ?Treatment with any investigational drug during the study or in the 4 weeks prior to entering the study.
    This includes treatment with any investigational drug during the study in an attempt to treat NP-C;
    ?Pregnancy or breastfeeding;
    ?Current participation in another trial is not permitted unless it is a non-interventional study and the sole purpose of the trial is for long-term follow up/survival data (registry);
    ?For patients who have not completed the CT ORZY NPC 001 study, fulfilling any of the criteria listed below:
    oPatients with uncontrolled severe epileptic seizures period (at least 3 consecutive severe epileptic seizures that required medication) within 2 months prior to the written consent. This includes patients with ongoing seizures that are not stable in frequency or type or duration over a 2 month period prior to enrolment, requiring change in dose of antiepileptic medication (other than adjustment for weight) over a 2 month period prior to enrolment, or requiring 3 or more antiepileptic medications to control seizures;
    oNeurologically asymptomatic patients;
    oSevere manifestations of NP-C disease that would interfere with the patient?s ability to comply with the requirements of this protocol;
    oTreatment with any IMP within 4 weeks prior to the study enrolment.
    Cada uno de los siguientes criterios se considerará un motivo de exclusión de la
    participación en el ensayo:
    • Receptor de un trasplante de hígado o trasplante de hígado programado.
    • Insuficiencia hepática grave (definida como parámetros de laboratorio
    hepáticos, AST o ALT superiores a tres veces el límite superior normal para
    la edad y el sexo (evaluación del laboratorio central).
    • Insuficiencia renal con un nivel de creatinina sérica superior a 1,5 veces el
    límite superior normal (eva
    • En opinión del Investigador, el estado clínico del paciente no permite la
    recogida de sangre necesaria o las biopsias de piel de los procedimientos
    especificados en el protocolo.
    • Tratamiento con cualquier fármaco en fase de investigación durante el
    estudio o en las 4 semanas previas al comienzo del estudio.
    Se incluye aquí el tratamiento con cualquier fármaco en fase de
    investigación durante el estudio en un intento de tratar la NPC.
    • Embarazo o lactancia.
    • No se permite la participación actual en otro ensayo salvo que se trate de un
    estudio no intervencional y el único propósito del ensayo sea el seguimiento
    a largo plazo/datos de supervivencia (registro).
    • En el caso de los pacientes que no hayan completado el estudio CT-ORZYNPC-
    001, el cumplimiento de cualquiera de los criterios indicados a
    continuación:
    o Los pacientes con un período de ataques epilépticos graves sin
    control (al menos 3 ataques epilépticos graves consecutivos que
    requirieron medicación) en los 2 meses previos al consentimiento
    por escrito. Se incluye aquí a los pacientes con ataques
    continuados que no son estables en su frecuencia, tipo o duración
    durante un período de 2 meses previo a la inclusión, que requieren
    un cambio en la dosis de la medicación antiepiléptica (que no sea
    un ajuste por el peso) durante un período de 2 meses previo a la
    inclusión, o que requieren 3 o más medicaciones antiepilépticas
    para controlar los ataques.
    o Pacientes asintomáticos desde el punto de vista neurológico.
    o Manifestaciones graves de la enfermedad NP-C que podrían
    interferir con la capacidad del paciente para cumplir con los
    requisitos de este protocolo.
    o Tratamiento con cualquier PEI en las 4 semanas previas a la
    inclusión en el estudio.
    luación del laboratorio central).
    • Alergia o intolerancia conocida o sospechada al PEI (arimoclomol o sus
    componentes).
    E.5 End points
    E.5.1Primary end point(s)
    ?Change in the NPC disease severity based on the NPCCSS scores (Yanjanin et al., 2010) from baseline (Visit 1) to 12 months.
    Cambio en la gravedad de la enfermedad NPC basado en las puntuaciones
    NPCCSS (Yanjanin et al., 2010) desde el período inicial (Visita 1) hasta los 12
    meses.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Visit 6 (12 months)
    Visita 6 (12 meses)
    E.5.2Secondary end point(s)
    ?Change in NPCCSS at 6 months (placebo versus arimoclomol), 18 and 24 months and annually thereafter;
    ?Changes in each individual domain of the NPCCSS (Yanjanin et al., 2010) at 6 and 12 months (placebo versus arimoclomol), 18 and 24 months and annually thereafter;
    ?Change in the Niemann Pick type C Clinical Database (NPC-cdb) score (modified "Stampfer Score" [Stampfer et al., 2013]) at 6 and 12 months placebo versus arimoclomol), 18 and 24 months and annually thereafter;
    ?Change in Quality of Life (EQ 5D Y) at 6 and 12 months (placebo versus arimoclomol), 18 and 24 months and annually thereafter;
    ?Change in the SARA score at 6 and 12 months (placebo versus arimoclomol), 18 and 24 months and annually thereafter;
    ?Change in the 9HPT time at 6 and 12 months (placebo versus arimoclomol), 18 and 24 months and annually thereafter.
    • Cambio en NPCCSS a los 6 meses (placebo frente a arimoclomol), 18 y 24
    meses, y anualmente con posterioridad.
    • Cambios en cada dominio individual de NPCCSS (Yanjanin et al., 2010) a los 6
    y 12 meses (placebo frente a arimoclomol), 18 y 24 meses, y anualmente con
    posterioridad.
    • Cambio en la puntuación de la base de datos clínica de Niemann Pick tipo C
    (NPC-cdb) (puntuación "Stampfer" modificada [Stampfer et al., 2013] a los 6 y
    12 meses, placebo frente a arimoclomol), 18 y 24 meses, y anualmente con
    posterioridad.
    • Cambio en la calidad de vida (EQ-5D-Y) a los 6 y 12 meses (placebo frente a
    arimoclomol), 18 y 24 meses, y anualmente con posterioridad.
    • Cambio en la puntuación SARA a los 6 y 12 meses (placebo frente a
    arimoclomol), 18 y 24 meses, y anualmente con posterioridad.
    • Cambio en el tiempo de 9HPT a los 6 y 12 meses (placebo frente a
    arimoclomol), 18 y 24 meses, y anualmente con posterioridad.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoints will be at 6 months, 12 months, 18 Months and 24 months
    Puntos de tiempo estarán en 6 meses, 12 meses, 18 meses y 24 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 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 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 trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Switzerland
    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 years0
    E.8.9.1In the Member State concerned months15
    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 months15
    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: 30
    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: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Patients on this study will involve minors and adult patients where brain/ central nervous
    system are affected and could impact their ability to give consent.
    F.3.3.7Others No
    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 36
    F.4.2.2In the whole clinical trial 46
    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)
    Following this study, all patients will be offered to continue into the extension phase of the study where every patient will receive arimoclomol and be followed up on an annual basis. The extension phase runs until arimoclomol has received European Union (EU) marketing authorisation (MA) or until the analysis of data from the controlled, blinded phase 12 month study period does not support the efficacy and/or safety of arimoclomol.
    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 Decision2016-08-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-08-19
    P. End of Trial
    P.End of Trial StatusOngoing
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