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Clinical trials for Hurler Syndrome (MPS I)

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
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    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    11 result(s) found for: Hurler Syndrome (MPS I). Displaying page 1 of 1.
    EudraCT Number: 2006-005216-27 Sponsor Protocol Number: 78993095 Start Date*: 2007-03-26
    Sponsor Name:HUS, Hospital for Children and Adolescents
    Full Title: Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord c...
    Medical condition: Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respir...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10056887 Mucopolysaccharidosis IH/S LLT
    Population Age: Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-000350-11 Sponsor Protocol Number: 2032/MUL Start Date*: 2014-05-28
    Sponsor Name:Multiplex Pharma Holdings LLC
    Full Title: An open label, randomized study to investigate the safety of weekly pentosan polysulfate injections in adult patients with Mucopolysaccharidosis Type I receiving enzyme replacement therapy.
    Medical condition: Mucopolysaccharidosis type I (alpha-L-Iduronidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10056888 Mucopolysaccharidosis IS LLT
    17.0 100000004850 10056887 Mucopolysaccharidosis IH/S LLT
    17.0 100000004850 10028094 Mucopolysaccharidosis IH LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2015-000585-61 Sponsor Protocol Number: ALID02003 Start Date*: 2015-05-18
    Sponsor Name:Genzyme, a Sanofi Company
    Full Title: A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients with Mucopolysaccaridosis I (MPS I) Being Treated with Aldurazyme® (laronidase)
    Medical condition: Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie's Syndrome
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Infants and toddlers, Children, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2007-001453-26 Sponsor Protocol Number: MPS protocol 08-06-07 Start Date*: 2007-07-02
    Sponsor Name:
    Full Title: Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving com...
    Medical condition: Mucopolysaccharidosis type I, II and VI.
    Disease: Version SOC Term Classification Code Term Level
    12.0 10028095 Mucopolysaccharidosis IV LLT
    12.0 10056886 Mucopolysaccharidosis I LLT
    12.0 10056889 Mucopolysaccharidosis II PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2007-001163-30 Sponsor Protocol Number: ALID02307 Start Date*: 2015-05-11
    Sponsor Name:Genzyme, a Sanofi Company
    Full Title: A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase)
    Medical condition: Mucopolysaccharidosis I
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-003031-35 Sponsor Protocol Number: R04049 Start Date*: 2015-11-20
    Sponsor Name:Central Manchester University Hospitals NHS Foundation Trust
    Full Title: A Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitigating the Alloimmune Response to Intravenous Laronidase in Infants With Severe Mucopolysaccharido...
    Medical condition: Severe Mucopolysaccharidosis Type I (Hurler syndrome, MPS IH)
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10028094 Mucopolysaccharidosis IH LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2010-020048-36 Sponsor Protocol Number: HGT-HIT-045 Start Date*: 2011-01-31
    Sponsor Name:Shire HGT, Inc
    Full Title: A Phase I/II, Randomized, Safety and Ascending Dose Ranging Study of Intrathecal Idursulfase-IT administered in conjunction with intravenous Elaprase in Pediatric Patients with Hunter Syndrome and ...
    Medical condition: Treatment of Hunter syndrome and cognitive impairment
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2009-017026-39 Sponsor Protocol Number: UCBT-002 Start Date*: 2009-12-28
    Sponsor Name:Aldagen, Inc
    Full Title: A PHASE III TRIAL OF ALD-101 ADUVANT THERAPY of UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) IN PATIENTS WITH INBORN ERRORS OF METABOLISM
    Medical condition: Inborn errors of metabolism
    Disease: Version SOC Term Classification Code Term Level
    9.1 10062018 Inborn error of metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2009-011921-15 Sponsor Protocol Number: UCBT-002 Start Date*: 2009-06-22
    Sponsor Name:Aldagen, Inc
    Full Title: A PHASE III TRIAL OF ALD-101 ADUVANT THERAPY of UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) IN PATIENTS WITH INBORN ERRORS OF METABOLISM
    Medical condition: Inborn errors of metabolism
    Disease: Version SOC Term Classification Code Term Level
    9.1 10062018 Inborn error of metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Not Authorised)
    Trial results: (No results available)
    EudraCT Number: 2018-000206-28 Sponsor Protocol Number: SB-318-1502 Start Date*: 2018-08-14
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase 1/2, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis I (MPS I)
    Medical condition: Mucopolysaccharidosis type I (MPS I)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-004909-27 Sponsor Protocol Number: DNLI-E-0002 Start Date*: 2020-07-22
    Sponsor Name:Denali Therapeutics Inc.
    Full Title: A PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO DETERMINE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME
    Medical condition: Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II])
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10056917 Hunter's syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) NL (Ongoing)
    Trial results: (No results available)
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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