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    EudraCT Number:2017-002806-10
    Sponsor's Protocol Code Number:SOBI003-001
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-11-08
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2017-002806-10
    A.3Full title of the trial
    An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients
    A.4.1Sponsor's protocol code numberSOBI003-001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03423186
    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 SponsorSwedish Orphan Biovitrum AB (publ)
    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 supportSwedish Orphan Biovitrum AB
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSwedish Orphan Biovitrum AB (publ)
    B.5.2Functional name of contact pointAnders Bröijersen MD
    B.5.3 Address:
    B.5.3.1Street AddressSwedish Orphan Biovitrum
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post code11276
    B.5.4Telephone number+4686972000
    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/142/16
    D.3 Description of the IMP
    D.3.1Product nameChemically modified recombinant human sulfamidase
    D.3.2Product code SOBI003
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous drip use (Noncurrent)
    Intravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot applicable
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeSOBI003
    D.3.9.4EV Substance CodeSUB192574
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 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 IIIA or Sanfilippo Syndrome
    MPS III is a progressive disorder that affects the brain and spinal cord , other systems may also be involved
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis Type IIIA (MPS IIIA) or Sanfilippo Syndrome
    MPS III is a progressive disorder that affects the brain and spinal cord , other systems may also be involved
    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 LLT
    E.1.2Classification code 10028094
    E.1.2Term Mucopolysaccharidosis IH
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary objective:
    To evaluate the safety and tolerability of SOBI003 at different dose levels.
    E.2.2Secondary objectives of the trial
    Secondary objectives:

    1. To characterize tthe pharmacokinetics (PK) properties of SOBI003 following single and repeated administration by the use of non-compartmental analysis (NCA)
    2. To assess the immunogenicity of SOBI003
    3. To assess the pharmacodynamic (PD) effect of different dose levels and treatment duration of SOBI003 on heparan sulfate (HS) levels in
    cerebrospinal fluid (CSF), serum and urine
    4. To assess the effect of SOBI003 at different dose levels on neurocognition
    5. To assess the effect of SOBI003 at different dose levels on adaptive behavior
    6. To assess the effect of SOBI003 at different dose levels on gray matter volume
    7. To assess the effect of SOBI003 at different dose levels on Quality of Life
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Patients/parents will optionally consent for future research and future genetic research. At Week 22, blood is collected for future genetic research. At Baseline and Week 22, blood and urine are collected for future biomarker research. CSF is collected at Baseline and Week 24 for future biomarker research. In case of Early Termination of SOBI003 treatment, blood and urine is collected for storage for future biomarker and genetic research.
    MPS IIIA is a very rare and severe disease for which there is a lot of research ongoing to further increase the understanding of disease pathology as well as diagnosis. The purpose of the sample storage is to allow future exploration/confirmation of new research findings. This includes:
     SGSH genotypes
     Geno- and/or phenotype associated correlations with possible impact on safety, tolerability, immunogenicity, PK and PD related to SOBI003 treatment
     Biomarkers in serum, urine and/or CSF with possible relation to the safety, tolerability, immunogenicity, PK and PD of SOBI003
    E.3Principal inclusion criteria
    MPS IIIA patients, as confirmed by both a documented deficiency in sulfamidase enzyme activity in concordance with a diagnosis of MPS IIIA and normal enzyme activity level of at least one other sulfatase measured in leukocytes, that are ≥12 to ≤72 months of age and that have a developmental age ≥12 months.
    E.4Principal exclusion criteria
    Patients with at least one S298P mutation in the SGSH gene are excluded, as well as patients that have received prior stem cell or gene therapy, or enzyme replacement therapy for MPS IIIA.
    E.5 End points
    E.5.1Primary end point(s)
    Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Adverse events are recorded from the time of first investigational product administration until last study visit (Week 24).

    Blood and urine samples for central laboratory analyses of hematology, clinical chemistry and coagulation are collected at Screening and prior to each infusion at Weeks 1 to 8. Pre-infusion sampling also applies biweekly from Week 10 to 22.

    12-lead ECGs are obtained at Screening and prior to and post the infusions on Weeks 1 to 4, 6, 8, 12 and 24.

    A physical examination will be performed at Screening, Baseline and at Week 24. General appearance and skin are examined prior to and within 1 hour of completion of each infusion.
    E.5.2Secondary end point(s)
    Secondary endpoints:

    The secondary endpoints to evaluate the primary objective of the safety and tolerability of SOBI003 are:
    - Vital signs (blood pressure, heart rate, body temperature, respiratory rate and oxygen saturation)
    - Laboratory safety variables (hematology, coagulation, clinical chemistry and urine analysis)

    The endpoints relating to the 1st secondary objective are:
    - Serum SOBI003 PK parameters at Weeks 1, 4, 12, and 24; tEnd of inf,
    CEnd of inf, Cmax, tmax, CPre-dose, CTrough, CL, AUC0-168h, t1/2
    - CSF SOBI003 concentration at Weeks 12 and 24

    The endpoints relating to the 2nd secondary objective are:
    - Occurrence of anti-drug antibodies (ADAs) against SOBI003 in serum
    (seroconversion rate, time to seroconversion, transient/persistent). For patients with confirmed ADA positive serum samples, the following additional endpoints apply; ADA titers and IgG subclasses in serum and presence of neutralizing antibodies (NAb) in serum.
    - Occurrence of ADAs against SOBI003 in CSF (conversion rate, time to
    occurrence, transient/persistent). For patients with confirmed ADA
    positive CSF samples, the following additional endpoints apply; ADA
    titers and presence of NAb in CSF.

    The endpoints relating to the 3rd secondary objective are:
    - Change from baseline in CSF HS at Weeks 12 and 24
    - Change from baseline in serum HS at Weeks 2, 3, 4, 8, 12 and 24
    - Change from baseline in urine HS at Weeks 2, 3, 4, 8, 12 and 24

    The endpoints relating to the 4th secondary objective are:
    - neurocognitive Development Quotient (DQ) and age-equivalence score (AEq) as assessed by the Bayley Scales of Infant and Toddler Development®, third edition (BSID-III)
    - cognitive subtest or the Kaufman Assessment Battery for Children, Second edition (KABC™-II); change from baseline at Week 24.

    The endpoint relating to the 5th secondary objective is
    - adaptive behavior ageequivalence score (AEq) as assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II); change from baseline at Week 24.

    The endpoint relating to the 6th secondary objective is gray matter volume as assessed by brain volumetric magnetic resonance imaging (MRI); change from baseline at Week 24.

    The endpoint relating to the 7th secondary objective is Pediatric Quality of Life Inventory (PedsQL™) total score and PedsQL™ Family Impact Module total score; change from baseline at Week 24.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timespoints for evaluations:
    - Serum concentration vs time profiles are obtained for determination of SOBI003 PK variables at Weeks 1, 4, 12 and 24.
    - In addition, serum samples are collected when ADA samples are obtained. SOBI003 concentration in CSF is assessed at Weeks 12 and 24.
    - HS is assessed in CSF at Baseline and Weeks 12 and 24.
    - HS in serum at Baseline and at Weeks 2 to 4, 8, 12 and 24.
    - HS in urine at Screening, Baseline, and at Weeks 2 to 4, 8, 12, and 24.
    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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    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 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.8.3 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
    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
    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 months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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: 9
    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) Yes
    F. of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 7
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 2
    F.4.2.2In the whole clinical trial 9
    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)
    All patients that have completed treatment in SOBI003-001 study will be offered participation in extension study SOBI003-002. The total duration of the extension study for an individual patient is 80 weeks, resulting in a total of 104 weeks (2 years) of SOBI003 treatment.
    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 Decision2018-11-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-29
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-02-17
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