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Clinical trials for Dysostosis

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
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44341   clinical trials with a EudraCT protocol, of which   7370   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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    13 result(s) found for: Dysostosis. Displaying page 1 of 1.
    EudraCT Number: 2013-000597-29 Sponsor Protocol Number: LTS13619 Start Date*: 2015-09-30
    Sponsor Name:sanofi-aventis recherche & développement
    Full Title: An Open-Label Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients with Usher Syndrome type 1B
    Medical condition: Usher syndrome type 1B
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2022-001092-14 Sponsor Protocol Number: TIGEM5_USH Start Date*: 2023-10-12
    Sponsor Name:FONDAZIONE TELETHON
    Full Title: A phase I/II open label, dose escalation study of sub-retinal administration of a “mixture of two adeno-associated viral vectors of serotype 8 containing the 5 ´- half sequence of the human MYO7A ...
    Medical condition: Usher syndrome (USH) is characterized by the association of sensorineural hearing loss, Retinitis Pigmentosa (RP), and, in some cases, vestibular dysfunction. It is the most frequent cause of deaf-...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    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: 2010-020198-18 Sponsor Protocol Number: MOR-004 Start Date*: 2010-12-30
    Sponsor Name:BioMarin Pharmaceutical Inc
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with M...
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) NL (Completed) FR (Ongoing) DE (Completed) PT (Completed) IT (Completed) NO (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2010-020199-45 Sponsor Protocol Number: MOR-005 Start Date*: 2011-06-08
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) IT (Completed) PT (Completed) DK (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2011-003197-84 Sponsor Protocol Number: MOR-007 Start Date*: 2011-11-22
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-005703-33 Sponsor Protocol Number: MOR-006 Start Date*: 2012-06-19
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2011-005682-20 Sponsor Protocol Number: MOR-008 Start Date*: 2012-06-19
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    16.0 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2010-021048-16 Sponsor Protocol Number: MOR-100 Start Date*: 2010-10-07
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (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: 2018-000192-33 Sponsor Protocol Number: SB-913-1602 Start Date*: 2018-08-17
    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-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II)
    Medical condition: Mucopolysaccharidosis type II (MPS II)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-002158-35 Sponsor Protocol Number: IVA_01_ODI_HMPS_17_002 Start Date*: 2017-11-16
    Sponsor Name:Inventiva S.A.
    Full Title: A phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI.
    Medical condition: Mucopolysaccharidosis (MPS) type VI.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) FR (Completed) PT (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)
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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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