- Trials with a EudraCT protocol (8)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
8 result(s) found for: Scheie syndrome.
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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... | |||||||||||||
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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) | |||||||||||||||||||||||
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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 | |||||||||||||
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Population Age: Infants and toddlers, Children, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
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 | |||||||||||||
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Population Age: Infants and toddlers, Children, Under 18 | 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. | |||||||||||||||||||||||
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Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: NL (Ongoing) | |||||||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2018-000206-28 | 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) | |||||||||||||
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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: 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 | |||||||||||||
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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 | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Not Authorised) | |||||||||||||
Trial results: (No results available) |
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