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    EudraCT Number:2014-003960-20
    Sponsor's Protocol Code Number:SHP-610-201
    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:2015-01-26
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2014-003960-20
    A.3Full title of the trial
    A Open-Label Extension of Study HGT-SAN-093 Evaluating the
    Safety and Efficacy Study of HGT-1410 (Recombinant Human
    Heparan N Sulfatase) Administration via an Intrathecal Drug
    Delivery Device in Pediatric Patients with Mucopolysaccharidosis
    Type IIIA Disease
    Estudio de extensión abierto de HGT-SAN-093 para evaluar la seguridad y la eficacia de la administración de HGT-1410 (heparán N sulfatasa humana recombinante) mediante un dispositivo de administración intratecal de medicación en pacientes pediátricos con mucopolisacaridosis de tipo IIIA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Extension Study to Evaluate the Safety and Efficacy of HGT-1410 Administration in Pediatric Patients with Sanfilippo Syndrome Type A
    Estudio de extensión para evaluar la seguridad y la eficacia de la administración de HGT-1410 en pacientes pediátricos con Sindrome de Sanfilipo de tipo A
    A.4.1Sponsor's protocol code numberSHP-610-201
    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 SponsorShire human Genetic Therapies, 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 supportShire human Genetic Therapies, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Human Genetic Therapies Inc
    B.5.2Functional name of contact pointKenia Baez
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34914229372
    B.5.5Fax number0017814821819
    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/3/08/582
    D.3 Description of the IMP
    D.3.1Product nameRecombinant human heparan N-sulfatase (rhHNS)
    D.3.2Product code HGT-1410
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot available
    D.3.9.2Current sponsor codeHGT-1410
    D.3.9.3Other descriptive nameRecombinant human heparan N-sulfatase
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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 Yes
    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 Yes
    D. medicinal product typeHuman protein heparan N-sulfatase produced in a human cell line by genetic engineering technology.
    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
    Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Síndrome de Sanfilippo de tipo A o mucopolisacaridosis (MPS IIIA)
    E.1.1.1Medical condition in easily understood language
    Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Síndrome de Sanfilippo de tipo A o mucopolisacaridosis (MPS IIIA)
    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 17.1
    E.1.2Level LLT
    E.1.2Classification code 10056918
    E.1.2Term Sanfilippo's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10056890
    E.1.2Term Mucopolysaccharidosis III
    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 long-term safety in patients with mucopolysaccharidosis type IIIA disease (MPS IIIA or Sanfilippo Type A) who received HGT-1410
    Evaluar la seguridad a largo plazo en pacientes con mucopolisacaridosis de tipo IIIA (MPS IIIA o síndrome de Sanfilippo de tipo A) que hayan recibido HGT-1410
    E.2.2Secondary objectives of the trial
    To evaluate the following: The long-term cognitive function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Kaufman Assessment Battery for Children, 2nd Edition (KABC-II), age-equivalent and developmental quotient (DQ) scores in patients with MPS IIIA who received HGT-1410; the long-term adaptive behavioral function, assessed by Vineland Adaptive Behavior Scales, Second Edition (VABS-II) in patients who received HGT-1410; the total cortical grey matter volume, as assessed by volumetric MRI of the
    brain, in patients who received HGT-1410.
    Evaluar lo siguiente:
    La función cognitiva a largo plazo determinada mediante las Escalas de Bayley de desarrollo infantil, tercera edición (BSID-III), o de la Batería de evaluación de Kaufman para niños, segunda edición (KABC-II), las puntuaciones de equivalentes de edad y coeficiente de desarrollo (CD) en pacientes con MPS IIIA que hayan recibido HGT-1410; la conducta adaptativa a largo plazo, evaluada mediante las Escalas de conducta adaptativa de Vineland, segunda edición (VABS-II), en pacientes que hayan recibido HGT-1410; El volumen total de sustancia gris cortical, evaluado mediante RM volumétrica del cerebro, en pacientes que hayan recibido HGT-1410.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria to be considered eligible for enrollment:
    1. Patient has completed through at least the Week 48 visit of Study HGT-SAN-093.
    2. The patient?s parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board- (IRB-)/Independent Ethics Committee- (IEC-) approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient?s parent(s) or legally authorized guardian(s) and the patient?s assent, as relevant, must be obtained.
    Los pacientes deberán cumplir todos los criterios siguientes para poder ser incluidos en el estudio:
    1. El paciente ha llegado hasta, como mínimo, la visita 48 del estudio HGT-SAN-093.
    2. Los padres o tutores legales del paciente deberán haber firmado voluntariamente un documento de consentimiento informado, aprobado por el comité ético de investigación clínica, una vez explicados y comentados todos los aspectos pertinentes del estudio. Será obligatorio obtener el consentimiento de los padres o tutores legales y el asentimiento del paciente, cuando proceda.
    E.4Principal exclusion criteria
    Patients will be excluded from the study if any of the following criteria are met:
    1. The patient, if randomized to treatment in Study HGT-SAN-093, has experienced a decline of more than 20 points in the BSID-III cognitive DQ score between Baseline and the Week 48 visit in Study HGT-SAN-093, AND, upon individual evaluation by the Investigator, has been deemed a treatment failure*.
    2. The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with HGT-1410, including but not limited to clinically relevant intracranial hypertension, severe infusion-related reactions after treatment with HGT-1410, uncontrollable seizure disorder.
    3. The patient has a known hypersensitivity to any of the components of HGT-1410.
    4. The patient is enrolled in another clinical study, other than HGT-SAN-093, that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study.
    5. The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions.
    6. The patient has a condition that is contraindicated as described in the SOPH-APORT ® Mini S IDDD Instructions for Use, including:
    a.- The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT ® Mini S device
    b.- The patient?s body size is too small to support the size of the SOPH-A-PORT ® Mini S Access Port, as judged by the Investigator
    c.- The patient?s drug therapy requires substances known to be incompatible with the materials of construction
    d.- The patient has a known or suspected local or general infection
    e.- The patient is at risk of abnormal bleeding due to a medical condition or therapy
    f.- The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    g.- The patient has a functioning CSF shunt device
    h.- The patient has shown an intolerance to an implanted device
    7. The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.
    *All treated patients in Study HGT-SAN-093 will have their cognitive development assessed at the Week 48 Visit in Study HGT-SAN-093. If a decline from Baseline of 20 points or less in the BSID-III DQ score is observed, then the patient may proceed into
    the Study SHP-610-201 without further evaluation. If a decline from Baseline of more than 20 points in DQ score is observed, then an
    individual evaluation by the Investigator will occur to determine if the patient is a treatment failure. This individual evaluation will
    take into account the DQ scores, VABS-II score, physical status, and any other information available for that patient at that time. If the Investigator deems the patient to
    be a treatment failure, then the patient may not enter the Study SHP-610-201.
    Se excluirá del estudio a los pacientes que cumplan alguno de los criterios siguientes:
    1. El paciente, si se le asignó un tratamiento en el estudio HGT-SAN-093, ha presentado un descenso de más de 20 puntos en el CD cognitivo de la escala BSID-III entre la visita basal y la semana 48 en el estudio HGT-SAN-093 Y, según la evaluación individual realizada por el investigador, se ha considerado que representa un fracaso del tratamiento*.
    2. El paciente ha experimentado, en opinión del investigador, un problema médico o de seguridad que contraindica el tratamiento con HGT-1410, por ejemplo, hipertensión intracraneal de importancia clínica, una reacción grave a la infusión después del tratamiento con HGT-1410 o un trastorno comicial no controlable.
    3. El paciente tiene hipersensibilidad conocida a alguno de los componentes de HGT- 1410.
    4. El paciente participa en otro estudio clínico, aparte de HGT-SAN-093, que suponga investigaciones clínicas o el uso de un producto en investigación (fármaco o dispositivo [intratecal/vertebral]) en los 30 días previos a la inclusión en este estudio o en algún momento durante el mismo.
    5. El paciente tiene hipersensibilidad confirmada o supuesta a los anestésicos o se considera que existe un riesgo inaceptablemente alto para la anestesia debido a un deterioro de las vías respiratorias u otras enfermedades.
    6. El paciente presenta alguna de las contraindicaciones que se detallan en las instrucciones de uso del sistema de administración de medicación por vía intratecal (SAMI) SOPH-A-PORT® Mini S, como por ejemplo:
    a. El paciente ha sufrido o podría sufrir una reacción alérgica a los materiales empleados en la fabricación del dispositivo SOPH-A-PORT® Mini S.
    b. El paciente tiene un tamaño corporal demasiado pequeño para soportar el tamaño del puerto de acceso SOPH-A-PORT® Mini S, según el criterio del investigador.
    c. El paciente necesita tratamiento farmacológico con sustancias incompatibles con los materiales de fabricación.
    d. El paciente sufre una infección local o general confirmada o supuesta.
    e. El paciente corre riesgo de sufrir una hemorragia anormal debida a una enfermedad o tratamiento.
    f. El paciente tiene una o varias anomalías vertebrales que podrían complicar la implantación o la fijación de forma segura.
    g. El paciente lleva un dispositivo de derivación del líquido cefalorraquídeo (LCR) en funcionamiento.
    h. El paciente ha demostrado intolerancia a un dispositivo implantado.
    7. El paciente no puede cumplir el protocolo (p. ej., no puede acudir a las evaluaciones de la seguridad o es improbable que finalice el estudio por otros motivos), en opinión del investigador.
    *Se hará una evaluación del desarrollo cognitivo a todos los pacientes tratados en el estudio HGT-SAN-093 en la visita de la semana 48 del estudio HGT-SAN-093. Si se observa un descenso de 20 puntos o menos en la puntuación del CD de la BSID-III con respecto al valor basal, el paciente podrá incorporarse al estudio SHP-610-201 sin necesidad de más evaluaciones. Si se observa un descenso de más de 20 puntos de la puntuación del CD con respecto al valor basal, el investigador realizará una evaluación individual para determinar si el paciente representa un fracaso del tratamiento. Esta evaluación individual tendrá en cuenta las puntuaciones del CD, la puntuación de las escalas VABS-II, el estado físico y todos los demás datos del paciente disponibles en ese momento. Si el investigador considera que el paciente representa un fracaso del tratamiento, el paciente no podrá incorporarse al estudio SHP-610-201.
    E.5 End points
    E.5.1Primary end point(s)
    Safety is the primary objective of the study and will be assessed during the study by the following:
    - collection of adverse events (by type, severity, and relationship to treatment [HGT-1410, the IDDD, device surgical procedure, or IT administration process])
    - changes in clinical laboratory testing (serum chemistry, hematology, urinalysis)
    - physical examination
    - vital signs
    - 12-lead electrocardiogram (ECG) recordings
    - CSF laboratory parameters (including chemistries, cell counts)
    - anti-rhHNS antibodies in CSF and serum, including determination of antibodies having enzyme neutralizing activity
    La seguridad es el objetivo primario del estudio y se evaluará mediante las determinaciones siguientes:
    - recopilación de los acontecimientos adversos (por tipo, intensidad y relación con el tratamiento [HGT-1410, SAMI, implantación quirúrgica del dispositivo o proceso de administración IT])
    - alteraciones en los análisis clínicos (bioquímica sérica, hematología, análisis de orina)
    - exploración física
    - constantes vitales
    - electrocardiogramas (ECG) de 12 derivaciones
    - parámetros de análisis del LCR (bioquímica, recuentos celulares)
    - anticuerpos anti-rhHNS en el LCR y el suero, incluida la determinación de anticuerpos con actividad neutralizante enzimática
    E.5.1.1Timepoint(s) of evaluation of this end point
    120 weeks
    120 semanas
    E.5.2Secondary end point(s)
    - The change from Baseline in BSID-III or KABC-II age-equivalent, DQ, and developmental delay scores
    - The change from Baseline in adaptive behavioral function domains, assessed by VABS II, using raw scores, age-equivalent scores, and DQ scores
    - The change from Baseline in total cortical grey matter volume, as assessed by MRI
    - La variación con respecto a los valores basales de las Escalas de Bayley (BSID-III) o en la Batería de evaluación de Kaufman para niños (KABC-II), las puntuaciones de equivalentes de edad, coeficiente de desarrollo (CD) y escalas de retraso de desarrollo.
    - La variación con respecto a los valores basales de la conducta adaptativa, evaluada mediante las Escalas de conducta adaptativa de Vineland (VABS-II), las puntuaciones de equivalentes de edad y coeficiente de desarrollo (CD).
    - La variación con respecto a los valores basales del volumen total de sustancia gris cortical, evaluado mediante resonancia magnética (RM) volumétrica del cerebro
    E.5.2.1Timepoint(s) of evaluation of this end point
    120 weeks
    120 semanas
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Quality of Life and Health Economics
    Calidad de vida y valoración de economía sanitaria.
    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) 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 Yes
    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 No
    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 EEA6
    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
    Last visit of the last patient in the clinical study
    Última visita del último paciente en el ensayo clínico
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 18
    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: 18
    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 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
    Children between 24 and 60 months
    Niños entre 24 y 60 meses
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 11
    F.4.2.2In the whole clinical trial 18
    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 Decision2015-02-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-01-30
    P. End of Trial
    P.End of Trial StatusCompleted
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