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Clinical trials for Mucopolysaccharidosis

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    The EU Clinical Trials Register currently displays   44294   clinical trials with a EudraCT protocol, of which   7351   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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    63 result(s) found for: Mucopolysaccharidosis. Displaying page 1 of 4.
    1  2  3  4  Next»
    EudraCT Number: 2004-002743-27 Sponsor Protocol Number: TKT024EXT Start Date*: 2004-12-23
    Sponsor Name:Shire Human Genetic Therapies INC.
    Full Title: An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
    Medical condition: Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
    Disease: Version SOC Term Classification Code Term Level
    6.1 10056889 PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: DE (Completed) GB (Completed) SE (Completed) IT (Completed) ES (Completed)
    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: 2014-004143-13 Sponsor Protocol Number: SHP-609-302 Start Date*: 2015-03-19
    Sponsor Name:Shire Human Genetic Therapies, Inc.
    Full Title: An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction with Elaprase® in Patients with Hunter Syndrome...
    Medical condition: Hunter syndrome and cognitive impairment
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    20.0 10010331 - Congenital, familial and genetic disorders 10056917 Hunter's syndrome LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Completed) FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-000212-25 Sponsor Protocol Number: HGT-HIT-046 Start Date*: 2012-01-05
    Sponsor Name:Shire HGT Inc
    Full Title: An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Pediatric Pati...
    Medical condition: Treatment of Hunter syndrome and cognitive impairment
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2007-006044-22 Sponsor Protocol Number: HGT-ELA-038 Start Date*: Information not available in EudraCT
    Sponsor Name:Shire Human Genetic Therapies, Inc. (Shire HGT)
    Full Title: A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy
    Medical condition: Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
    Disease: Version SOC Term Classification Code Term Level
    10.1 10056889 PT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: PL (Completed)
    Trial results: View results
    EudraCT Number: 2010-021348-16 Sponsor Protocol Number: HGT-SAN-067 Start Date*: 2011-02-09
    Sponsor Name:Shire Human Genetic Therapies, Inc
    Full Title: An Open-Label Extension of Study HGT-SAN-055 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Administration of rhHNS in Patients with Sanfilippo Syndrome Type A (MPS IIIA)
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) NL (Completed)
    Trial results: View results
    EudraCT Number: 2014-003960-20 Sponsor Protocol Number: SHP-610-201 Start Date*: 2015-02-18
    Sponsor Name:Shire human Genetic Therapies, Inc.
    Full Title: A Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in...
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    17.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed) DE (Prematurely Ended) NL (Completed) GB (Prematurely Ended) FR (Completed) IT (Prematurely Ended)
    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: 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: 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: 2015-000753-20 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: 2020-003200-14 Sponsor Protocol Number: JR-141-GS31 Start Date*: 2022-05-16
    Sponsor Name:JCR Pharmaceuticals Co., Ltd.
    Full Title: A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients.
    Medical condition: Mucopolysaccharidosis type II
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    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: 2007-001163-30 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: 2013-003450-24 Sponsor Protocol Number: HGT-SAN-093 Start Date*: 2014-04-09
    Sponsor Name:Shire Human Genetic Therapies, Inc
    Full Title: A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in ...
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    17.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) ES (Completed) NL (Completed) IT (Completed) FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-001479-18 Sponsor Protocol Number: GENiSIS2013 Start Date*: 2014-06-09
    Sponsor Name:Manchester University NHS Foundation Trust
    Full Title: A Phase III, Double Blinded, Randomised, Placebo Controlled Clinical Trial of High Dose Oral Genistein Aglycone in Patients with Sanfilippo Syndrome (Mucopolysaccharidosis III)
    Medical condition: Sanfilippo syndrome (Mucopolysaccharidosis III)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    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: 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: 2022-002762-33 Sponsor Protocol Number: AX250-401 Start Date*: 2022-12-12
    Sponsor Name:Allievex Corporation
    Full Title: 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 I...
    Medical condition: Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    20.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    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
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