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    EudraCT Number:2014-003960-20
    Sponsor's Protocol Code Number:SHP-610-201
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-11-05
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2014-003960-20
    A.3Full title of the trial
    An 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
    Estensione in aperto dello Studio HGT-SAN-093 per valutare la sicurezza e l'efficacia della somministrazione di HGT-1410 (Eparan N-solfatasi umana ricombinante) attraverso un dispositivo di rilascio intratecale del farmaco in pazienti pediatrici affetti da mucopolisaccaridosi di 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
    Uno studio di estensione per valutare la sicurezza e l'efficacia della somministrazione di HGT-1410 in pazienti pediatrici con sindrome San Filippo di tipo A
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberSHP-610-201
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN00000000
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00000000
    A.5.3WHO Universal Trial Reference Number (UTRN)U0000-0000-0000
    A.5.4Other Identifiers
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/027/2015
    B. Sponsor Information
    B.Sponsor: 1
    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, Inc.
    B.5.2Functional name of contact pointAlkisti Rouvas
    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 number0017814821736
    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 Nsulfatase (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 INNNA
    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 Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 typeProteina umana prodotta in linea cell con ing gen.
    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)
    Sindrome di Sanfilippo di Tipo A o Mucopolisaccaridosi (MPS IIIA)
    E.1.1.1Medical condition in easily understood language
    Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Sindrome di Sanfilippo di Tipo A o Mucopolisaccaridosi (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 20.0
    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 20.1
    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-
    Valutare la sicurezza a lungo termine in pazienti affetti da mucopolisaccaridosi di tipo IIIA (MPS IIIA o Sanfilippo tipo A) che hanno ricevuto 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.
    • La funzione cognitiva a lungo termine misurata dalle Scale di Bayley per lo sviluppo infantile, 3^ edizione (BSID-III) o dalla Batteria di valutazione di Kaufman per i bambini, 2^ edizione (KABC-II), attraverso i punteggi equivalenti per età e del quoziente di sviluppo (DQ) in pazienti affetti da MPS IIIA che hanno ricevuto HGT-1410
    • La funzione di comportamento adattivo a lungo termine, valutata dalle Scale Vineland per il comportamento adattivo, seconda edizione (VABS-II) in pazienti che hanno ricevuto HGT-1410
    • Il volume totale di materia grigia corticale, valutata con RM volumetrica del cervello, in pazienti che hanno ricevuto 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.
    Per essere considerati eleggibili per l'arruolamento, i pazienti devono soddisfare tutti i criteri elencati di seguito:
    1. Il paziente ha completato almeno la visita della Settimana 48 dello Studio HGT SAN 093.
    2. Il genitore o i genitori del paziente o il/i suo/suoi tutore/i legale/i devono aver firmato volontariamente un modulo di consenso informato approvato da un Comitato di revisione istituzionale (IRB) / Comitato etico indipendente (IEC) dopo che sono stati spiegati e discussi tutti gli aspetti rilevanti. Deve essere ottenuto il consenso del genitore o dei genitori del paziente o del suo tutore o dei suoi tutori legali e l'assenso del paziente, se rilevante.
    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. Patients with documented infusion-related reactions that are clinically manageable (for example, with pre-medication or slowing infusion rate) are not necessarily excluded based on the assessment of the investigator.
    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 SOPHA-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.
    1. Il paziente, se randomizzato al trattamento nello Studio HGT-SAN-093, ha avuto un calo di più di 20 punti nel punteggio DQ cognitivo del BSID-III tra il baseline e la visita della Settimana 48 nello Studio HGT-SAN-093, E, secondo la valutazione individuale dello Sperimentatore, è stato ritenuto un insuccesso del trattamento*.
    2. Il paziente ha avuto, secondo il parere dello Sperimentatore, un problema di sicurezza o medico che costituisce una controindicazione al trattamento con HGT 1410, compresi, a titolo esemplificativo ma non esaustivo, ipertensione intracranica clinicamente rilevante, reazioni gravi legate all'infusione dopo il trattamento con HGT-1410, disturbo epilettico incontrollabile.
    3. Il paziente presenta un'ipersensibilità nota a uno dei componenti di HGT 1410. I pazienti che presentano reazioni legate all'infusione documentate che sono clinicamente trattabili (per esempio, con farmaci preparatori o riduzione della velocità di infusione) non sono necessariamente esclusi sulla base della valutazione dello Sperimentatore.
    4. Il paziente viene arruolato in un altro studio clinico, diverso dall'HGT-SAN-093, che comporta sperimentazioni cliniche o l'uso di qualsiasi prodotto sperimentale (farmaco o dispositivo [intratecale/spinale]) entro i 30 giorni precedenti all'arruolamento nello studio o in qualsiasi momento durante lo studio.
    5. Il paziente presenta un'ipersensibilità nota o sospetta all'anestesia o si ritiene che sia a rischio inaccettabilmente elevato per l'anestesia a causa di vie respiratorie compromesse o altre condizioni.
    6. Il paziente presenta una condizione che è controindicata come descritto nelle Istruzioni d'uso dell'IDDD SOPH-A-PORT ® Mini S, compreso:
    a Il paziente ha avuto, o potrebbe avere, una reazione allergica ai materiali di costruzione del dispositivo SOPH-A-PORT ® Mini S
    b La corporatura del paziente è troppo piccola per sopportare le dimensioni della porta di accesso di SOPH-A-PORT ® Mini S, secondo il giudizio dello Sperimentatore
    c La terapia farmacologica del paziente richiede sostanze note per essere incompatibili con i materiali di costruzione
    d Il paziente presenta un'infezione locale o generale nota o sospetta
    e Il paziente è a rischio di sanguinamento anomalo a causa di una condizione medica o terapia
    f Il paziente presenta una o più anomalie spinali che potrebbero complicare l'impianto o il fissaggio in sicurezza
    g Il paziente ha un sistema di derivazione per CSF funzionante
    h Il paziente ha mostrato un'intolleranza a un dispositivo impiantato
    7. Il paziente non è in grado di attenersi al protocollo (ad es. non è in grado di tornare per valutazioni di sicurezza o verosimilmente non è altrimenti in grado di completare lo studio) secondo il giudizio dello Sperimentatore.
    *Tutti i pazienti trattati nello Studio HGT-SAN-093 saranno sottoposti a valutazione del proprio sviluppo cognitivo alla Visita della Settimana 48 nello Studio HGT-SAN-093. Qualora venga osservato un calo dal baseline di 20 punti o meno nel punteggio DQ BSID-III, allora il paziente potrà procedere nello Studio SHP 610 201 senza ulteriore valutazione. Qualora venga osservato un calo dal baseline maggiore di 20 punti nel punteggio DQ, allora avrà luogo una valutazione individuale da parte dello Sperimentatore al fine di stabilire se il paziente rappresenta un fallimento del trattamento. Tale valutazione individuale prenderà in considerazione i punteggi DQ, il punteggio VABS-II, lo stato fisico ed eventuali altre informazioni disponibili su questo paziente in quel momento. Se lo Sperimentatore considererà il paziente un fallimento del trattamento, allora il paziente non potrà entrare nello Studio 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 sicurezza è l'obiettivo primario dello studio e durante lo studio sarà valutata mediante:
    • raccolta di eventi avversi (per tipo, gravità e relazione con il trattamento [HGT-1410, IDDD, procedura chirurgica per il dispositivo o processo di somministrazione IT])
    • variazioni nelle analisi cliniche di laboratorio (analisi chimiche del siero, analisi ematologiche, analisi delle urine)
    • esame obiettivo
    • funzioni vitali
    • registrazioni di elettrocardiogramma a 12 derivazioni (ECG)
    • parametri di laboratorio del CSF (analisi chimiche, conte ematiche)
    • anticorpi anti-rhHNS nel CSF e nel siero, compresa la determinazione di anticorpi aventi attività enzimatica neutralizzante
    E.5.1.1Timepoint(s) of evaluation of this end point
    120 weeks
    120 settimane
    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 variazione dalla baseline dei punteggi BSID-III o KABC-II equivalenti per età, DQ e ritardo dello sviluppo
    • La variazione dalla baseline dei domini della funzione di comportamento adattivo, valutata da VABS II, mediante punteggi grezzi, punteggi equivalenti per età e punteggi del DQ
    • La variazione dalla baseline del volume totale della materia grigia corticale, valutata mediante RM
    E.5.2.1Timepoint(s) of evaluation of this end point
    120 weeks
    120 settimane
    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
    Qualità della vita e aspetti economici sanitari
    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 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 Yes
    E. trial design description
    Randomizzato, in aperto
    Randomized, open label
    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.8.4.1Number 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 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 Information not present in EudraCT
    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
    The LPLV will be the safety follow-up visit done 14 days after the final device (partial and/or full) is removed from that last patient during the safety follow up period.
    La LPLV sarà la visita di follow-up per la sicurezza effettuata 14 giorni dopo che l'ultimo dispositivo (parziale e/o completo) sarà stato rimosso dall'ultimo paziente durante il periodo di follow-up per la sicurezza.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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.1.1.1In 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: 17
    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
    Bambini di età compresa tra 24 e 60 mesi
    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 17
    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)
    The program has discontinued, the patient should revert back to their physician. At present, there is no approved therapy for San A so the treatment decision will be made by the physician and patient. Patients who do not have the IDDD removed (partial or full device) at the end of the treatment period will continue to be observed during a safety follow-up period with visits at the site every 6 months to evaluate patient safety of the device until the IDDD has been fully explanted
    Programma è stato interrotto, paziente deve tornare dal proprio medico. Ad oggi non esiste una terapia approvata per San A, la decisione relativa al trattamento verrà presa da medico e paziente. I pazienti che non si sottopongono a rimozione di IDDD al termine del periodo di trattamento continueranno ad essere osservati durante il periodo di follow-up per la sicurezza con visite al centro ogni 6 mesi per valutare la sicurezza per il paziente finché l'IDDD non sarà stato completamente espiantato
    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 Decision2015-08-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-16
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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