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    Summary
    EudraCT Number:2017-003083-13
    Sponsor's Protocol Code Number:250-202
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-01-11
    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 ConcernedUK - MHRA
    A.2EudraCT number2017-003083-13
    A.3Full title of the trial
    A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2 study to evaluate Long-Term Safety and Efficacy of AX 250 in Patients with MPS Type IIIB
    A.4.1Sponsor's protocol code number250-202
    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 SponsorAllievex Corporation
    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 supportAllievex Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAllievex Corporation
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressP.O. Box 1056
    B.5.3.2Town/ cityMarblehead
    B.5.3.3Post codeMA, 01945
    B.5.3.4CountryUnited States
    B.5.6E-mailinquiries@allievex.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 numberEMA/OD/213/14
    D.3 Description of the IMP
    D.3.1Product nameN/A
    D.3.2Product code AX 250
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraventricular use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot available
    D.3.9.2Current sponsor codeAX 250
    D.3.9.3Other descriptive nameRHNAGLU-IGF2
    D.3.9.4EV Substance CodeSUB177868
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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
    Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS
    IIIB)
    E.1.1.1Medical condition in easily understood language
    NAGLU deficiency
    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 20.1
    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.1.2 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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •Evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter
    • Evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by development quotient (DQ)
    E.2.2Secondary objectives of the trial
    • Evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by age equivalent score (AEq)
    • Characterize immunogenicity of long-term AX 250 treatment in CSF and serum
    • Evaluate the impact of long-term AX 250 treatment on CSF, serum and urine GAGs
    • Evaluate the impact of long-term AX 250 treatment on brain structure assessed by magnetic resonance imaging (MRI)
    • Evaluate the impact of long-term AX 250 treatment on adaptive function derived from the Vineland Adaptive Behavior Scales, 2nd edition (VABS-II)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Must have completed 48 weeks in Part 2 of Study 250-201 and enter
    250-202 within 8 weeks of study completion
    • Written informed consent from parent or legal guardian and assent from subject, if required
    • Has the ability to comply with protocol requirements, in the opinion of the investigator
    • Males and females who are of reproductive age should practice true abstinence, defined as no sexual activity, during the study and for 6 months after the study has been completed (or withdrawal from the study). If sexually active and not practicing true abstinence, males and females of reproductive age must use a highly effective method of contraception while participating in the study.
    • If female with childbearing potential, must have a negative pregnancy test at the Screening visit and be willing to have additional pregnancy tests during the study.
    E.4Principal exclusion criteria
    Individuals who meet any of the following exclusion criteria are ineligible to participate in this study:
    • Has (1) a cognitive AEq score ≤ 18 months, (2) a DQ score ≤ 20 and (3) no evidence of improvement during the 250-201 study in secondary or exploratory efficacy endpoints.
    • Would not benefit from enrolling in the study in the opinion of the investigator
    • Has received stem cell, gene therapy or ERT (other than AX 250) for MPS IIIB
    • Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities)
    • Has contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain)
    • Has a history of poorly controlled seizure disorder
    • Is prone to complications from intraventricular drug administration,
    including patients with hydrocephalus or ventricular shunts
    • Has received any investigational medication other than AX 250
    within 30 days prior to the Baseline visit or is scheduled to receive
    any investigational drug during the course of the study
    • Has a medical condition or extenuating circumstance that, in the
    opinion of the investigator, might compromise the subject’s ability
    to comply with protocol requirements, the subject’s well-being or
    safety, or the interpretability of the subject’s clinical data.
    • Is pregnant at any time during the study
    E.5 End points
    E.5.1Primary end point(s)
    To evaluate the lon-term safety and tolerability of AX 250 administered to
    subjects with MPS IIIB by an implanted intracerebroventricular (ICV)
    reservoir and catheter.

    To evaluate the lon-term impact of AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety evaluation will include continuous monitoring of AEs and concomitant medications. Clinical laboratory assessments at Baseline and every 4W the first 96W, Q12W thereafter. CSF for cell count, protein, and glucose collected prior to each weekly infusion. A complete physical examination performed at Baseline and every 24W thereafter. The exam includes assessment of general appearance, vital signs, measurements of height, weight and head circumference, a detailed neurological examination, and assessment of cardiovascular, respiratory,and gastrointestinal systems. Brief physical exams weekly dosing visits when complete exams are not performed. ECG and EEG performed at the EoT. Brain imaging (MRI or CT) to monitor for asymptomatic subdural hygroma formation at a frequency of up to Q4W
    E.5.2Secondary end point(s)
    The secondary objectives of this study are:
    *to evaluate the long-term impact of AX 250 treatment on cognitive function in patients
    with MPS IIIB as assessed by age equivalent score (AEq)
    *to characterize single- and repeated-dose pharmacokinetics (PK) of
    AX 250 in cerebrospinal fluid (CSF) and plasma
    *to characterize immunogenicity of long-term AX 250 treatment in CSF and serum
    *to evaluate the impact of AX 250 treatment on CSF, serum and urine GAGs
    *to evaluate the impact of AX 250 treatment on brain structure
    assessed by magnetic resonance imaging (MRI)
    *To evaluate the impact of AX 250 treatment on adaptive function
    derived from the Vineland Adaptive Behavior Scales, 2nd edition (VABSII).
    E.5.2.1Timepoint(s) of evaluation of this end point
    The CSF, as well as blood collected at less frequent intervals, will be used for GAG analyses and/or exploratory analyses of the biochemical, molecular, cellular and genetic/genomic aspects of MPS IIIB.

    CSF and blood (serum) will be drawn throughout the study to analyze immunogenicity

    Urine will be collected for GAG and creatinine analyses as well as for exploratory analyses at Baseline and every 24 weeks thereafter.

    Brain structure will be evaluated by MRI during Baseline visits and at Weeks 24 and 48.
    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 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 Yes
    E.8.1.1Randomised No
    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 Yes
    E.8.1.7.1Other trial design description
    Baseline Control
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Baseline Control
    E.8.2.4Number of treatment arms in the trial1
    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 EEA3
    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
    Colombia
    Germany
    Spain
    Taiwan
    Turkey
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial 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: 33
    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: 33
    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.3.3.6.1Details of subjects incapable of giving consent
    The subjects included in this trial are between 1 to 10 years of age.
    Therefore before any study-related procedures are performed, parents
    or legal guardians of the subjects must provide informed consent, and
    if required, subjects must assent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 33
    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)
    Expected normal tratment for the 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 Decision2018-01-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-02
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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