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    Summary
    EudraCT Number:2022-002762-33
    Sponsor's Protocol Code Number:AX250-401
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2022-09-21
    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 ConcernedGermany - BfArM
    A.2EudraCT number2022-002762-33
    A.3Full title of the trial
    A Phase 3B/4 Open-Label Multicenter Study Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase3B/4 extension study to Further Evaluate Safety, Tolerability and Efficacy of AX 250 in Patients with MPS Type IIIB
    A.4.1Sponsor's protocol code numberAX250-401
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05492799
    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 INNTralesinidase alfa
    D.3.9.2Current sponsor codeAX 250
    D.3.9.3Other descriptive namerhNAGLU-IGF2
    D.3.9.4EV Substance CodeSUB193855
    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
    - Further evaluate the long-term safety and tolerability of AX 250 administered by an implanted ICV device to subjects with MPS IIIB who have completed AX 250-202.
    - Further evaluate the impact of long-term AX 250 treatment on various skills and function as assessed in patients with MPS IIIB who have completed AX 250-202
    E.2.2Secondary objectives of the trial
    - to Characterize immunogenicity of AX 250 in CSF and serum
    - to evaluate the impact of AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by development quotient (DQ)
    - 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)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be eligible to participate individuals must meet all of the following criteria:
    - Must have completed 240 weeks of study 250-202 and enter study 250-401 with the first infusion occurring within 8 weeks of completing study 250-202.
    - Provided 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.
    - 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 250-202 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 since completing Study 250-202 prior to starting Study 250-401.
    - Has a history of poorly controlled seizure disorder.
    - Is prone to complications from ICV drug administration including patients with hydrocephalus or ventricular shunts.
    - Has received any investigational medication 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.
    E.5 End points
    E.5.1Primary end point(s)
    - To evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted intracerebroventricular (ICV) reservoir and catheter.
    - To evaluate the impact of AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by age equivalent score (AEq).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety evaluation includes
    -continuous monitoring of AEs and concomitant medications
    -complete physical examination (prior to 1º dose and Q24W thereafter through W144/Dosing Completion) includes an assessment of general appearance,cardiovascular,dermatologic,lymphatic,respiratory,gastrointestinal,genitourinary,musculoskeletal,neurologic systems and measurements of body weight,height and head circumference
    -brief physical exams are performed at dosing visit when complete physical exams not performed
    -clinical laboratory assessments at Baseline and Q24W thereafter
    -ECG and EEG performed at Screening/Baseline and at EoD or EoT
    -Brain imaging (MRI or CT) to monitor for asymptomatic subdural hygroma formation
    Efficacy evaluation includes
    -Neurocognitive testing using BSID-III or KABC-II
    E.5.2Secondary end point(s)
    - Secondary Efficacy evaluation includes:
    -- measures of communication assessment (BSID-III)
    -- measures of communication, motor skills and socialization skills as determined by VABS-II testing
    -- quality of life by ITQoL or CHQ-PF50
    - Liver and spleen volumes assessed by abdominal magnetic resonance imaging (MRI)
    - Regional brain volumes assessed by brain MRI
    - Pharmacodynamic Biomarkers: Glycosaminoglycans (GAGs)
    - Immunogenicity tests (validated immunogenicity assays on serum and CSF samples)
    E.5.2.1Timepoint(s) of evaluation of this end point
    - VABS-II , ITQOL or CHQ-PF50, MRI (under anesthesia) of the brain and abdomen performed every 48 weeks (± 2 weeks).
    - GAG:
    -- serum Week 4, Q4W thereafter
    -- plasma Week 12, Q12W thereafter
    - Immunogenicity tests : Samples of serum for TAb and CSF for TAb and NAb collected Q48W through Week 144/Dosing Completion. Samples of serum for NAb collected prior to the first dose and Q48W through Week 144/Dosing Completion.
    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 No
    E.6.7Pharmacodynamic Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    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 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 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 EEA2
    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
    United Kingdom
    United States
    Germany
    Türkiye
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    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 months0
    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 months0
    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: 15
    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: 11
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 4
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 15
    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 treatment 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 Decision2022-12-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-12
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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