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Clinical trials for Mucopolysaccharidosis type I

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    The EU Clinical Trials Register currently displays   44347   clinical trials with a EudraCT protocol, of which   7375   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).

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    38 result(s) found for: Mucopolysaccharidosis type I. Displaying page 1 of 2.
    1  2  Next»
    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: 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: 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: 2004-000642-21 Sponsor Protocol Number: ASB-03-06 Start Date*: Information not available in EudraCT
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI
    Medical condition: Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome)
    Disease: Version SOC Term Classification Code Term Level
    6.0 10056892 PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IE (Completed)
    Trial results: View results
    EudraCT Number: 2015-001983-20 Sponsor Protocol Number: NGLU-CL01-T Start Date*: 2015-08-06
    Sponsor Name:Alexion Pharmaceuticals, Inc
    Full Title: A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Int...
    Medical condition: Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2023-001027-16 Sponsor Protocol Number: LPS16578 Start Date*: 2023-11-22
    Sponsor Name:Genzyme Europe B. V
    Full Title: A Phase 4, single-arm, open-label safety and efficacy study of Aldurazyme® (laronidase) as enzyme replacement therapy in participants with Mucopolysaccharidosis I (MPS I) in China
    Medical condition: Mucopolysaccharidosis 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: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-000753-20 Sponsor Protocol Number: NGLU-CL06 Start Date*: 2016-08-16
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A PHASE I/II OPEN LABEL STUDY IN MPS IIIB SUBJECTS TO INVESTIGATE THE SAFETY, BIODISTRIBUTION, PHARMACOKINETICS, AND PHARMACODYNAMICS/EFFICACY OF SBC-103 ADMINISTERED INTRAVENOUSLY
    Medical condition: Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (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: 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: 2007-007003-33 Sponsor Protocol Number: ALID01803 Start Date*: 2010-09-16
    Sponsor Name:Genzyme Europe BV
    Full Title: A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme(laronidase) Treatment on Lactation in Women with Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
    Medical condition: mucocopolysaccharidoses I
    Disease: Version SOC Term Classification Code Term Level
    9.1 10056886 LLT
    Population Age: Adults Gender: Female
    Trial protocol: IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2014-002596-28 Sponsor Protocol Number: PTC124-GD-024-MPS Start Date*: 2015-06-01
    Sponsor Name:PTC Therapeutics Inc.
    Full Title: CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS)
    Medical condition: Nonsense mutation Mucopolysaccharidosis Type I
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-003105-41 Sponsor Protocol Number: PTC124-GD-031-MPS Start Date*: 2015-10-13
    Sponsor Name:PTC Therapeutics Inc.
    Full Title: CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS)
    Medical condition: Nonsense mutation Mucopolysaccharidosis Type I
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-015984-15 Sponsor Protocol Number: HGT-SAN-055 Start Date*: 2010-03-25
    Sponsor Name:Shire Human Genetic Therapies, Inc. (Shire HGT)
    Full Title: A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan-N-sulfatase (rhHNS) Intrathecal Administration via an Intrathecal Drug Delivery Device in Pat...
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    14.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2005-003512-30 Sponsor Protocol Number: ASB-008 Start Date*: 2007-04-05
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI)
    Medical condition: Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome)
    Disease: Version SOC Term Classification Code Term Level
    6.0 10056892 PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) PT (Completed)
    Trial results: View results
    EudraCT Number: 2012-000856-33 Sponsor Protocol Number: 2012-40 Start Date*: Information not available in EudraCT
    Sponsor Name:Institut Pasteur
    Full Title: A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase cDNA in children with Sanfilippo type B syndrome
    Medical condition: Mucopolysaccharidosis III B
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2010-019962-10 Sponsor Protocol Number: P1-SAF301 Start Date*: Information not available in EudraCT
    Sponsor Name:SANFILIPPO Therapeutics SAS
    Full Title: AN OPEN-LABEL, SINGLE ARM, MONOCENTRIC, PHASE I/II CLINICAL STUDY OF INTRACEREBRAL ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS SEROTYPE 10 CARRYING THE HUMAN SGSH AND SUMF1 cDNAS FOR THE TREAT...
    Medical condition: Sanfilippo type A syndrome (also named Mucopolysaccharidosis Type A)
    Disease: Version SOC Term Classification Code Term Level
    12.1 10056918 Sanfilippo's syndrome LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-003400-39 Sponsor Protocol Number: NGLU-CL02 Start Date*: 2014-07-15
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
    Medical condition: Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) ES (Temporarily Halted)
    Trial results: View results
    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: 2008-007365-23 Sponsor Protocol Number: MOR-002 Start Date*: 2009-03-12
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome)
    Medical condition: Mucopolysaccharidosis Type IV A
    Disease: Version SOC Term Classification Code Term Level
    9.1 10028095 Mucopolysaccharidosis IV LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2022-002314-17 Sponsor Protocol Number: JR-441-101 Start Date*: 2023-07-03
    Sponsor Name:JCR Pharmaceuticals Co., Ltd.
    Full Title: Phase I/II study of weekly infusions of JR-441 in patients with mucopolysaccharidosis type IIIA
    Medical condition: Mucopolysaccharidosis type IIIA (MPS IIIA) MPS III is an autosomal recessive disease characterized by the accumulation of HS in the tissues throughout the body due to a deficiency in enzymes involv...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    Trial results: (No results available)
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