- Trials with a EudraCT protocol (21)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
21 result(s) found for: Cerebroside.
Displaying page 1 of 2.
EudraCT Number: 2007-005516-61 | Sponsor Protocol Number: FARM6JBLZ2 | Start Date*: 2009-02-06 | |||||||||||
Sponsor Name:ISTITUTO GIANNINA GASLINI | |||||||||||||
Full Title: Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher di... | |||||||||||||
Medical condition: GAUCHER disease | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2007-007164-20 | Sponsor Protocol Number: EC07-90737 | Start Date*: 2008-05-20 | |||||||||||
Sponsor Name:INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD | |||||||||||||
Full Title: Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1 | |||||||||||||
Medical condition: Tratamiento de la enfermedad de Gaucher | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2008-007158-36 | Sponsor Protocol Number: GAU-CL-202X | Start Date*: 2009-02-25 | |||||||||||
Sponsor Name:Amicus Therapeutics, Inc. | |||||||||||||
Full Title: An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. | |||||||||||||
Medical condition: Type I Gaucher Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2007-000498-42 | Sponsor Protocol Number: PB-06-001 | Start Date*: 2008-05-08 | |||||||||||
Sponsor Name:Protalix Biotherapeutics | |||||||||||||
Full Title: A Phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patient... | |||||||||||||
Medical condition: Gaucher disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) ES (Completed) IT (Completed) Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-003676-37 | Sponsor Protocol Number: PB-06-006 | Start Date*: 2011-11-15 | |||||||||||
Sponsor Name:Protalix Biotherapeutics | |||||||||||||
Full Title: A Multicenter Extension Study of Taliglucerase alfa in Pediatric Subjects with Gaucher Disease | |||||||||||||
Medical condition: Gaucher disease | |||||||||||||
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Population Age: Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-003685-32 | Sponsor Protocol Number: PB-06-007 | Start Date*: 2012-01-27 | |||||||||||
Sponsor Name:Protalix Biotherapeutics | |||||||||||||
Full Title: A Multicenter Extension Study of Taliglucerase alfa in Adult Subjects with Gaucher Disease | |||||||||||||
Medical condition: Gaucher disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2006-006304-11 | Sponsor Protocol Number: TKT034 | Start Date*: 2007-07-03 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies Inc | |||||||||||||
Full Title: A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase | |||||||||||||
Medical condition: Type I Gaucher Disease | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) IT (Completed) ES (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2024-000041-27 | Sponsor Protocol Number: LPS16031 | Start Date*: 2024-02-04 | |||||||||||
Sponsor Name:SANOFI (CHINA) INVESTMENT CO., LTD | |||||||||||||
Full Title: A single arm, prospective, open label, multicenter study to evaluate efficacy and safety of one-year maximum dosage in Chinese label of imiglucerase treatment in Chinese patients who are diagnosed ... | |||||||||||||
Medical condition: | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2008-001965-27 | Sponsor Protocol Number: HGT-GCB-044 | Start Date*: 2008-10-29 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies, Inc. | |||||||||||||
Full Title: An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | |||||||||||||
Medical condition: Type I Gaucher disease | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) ES (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2009-015811-42 | Sponsor Protocol Number: GZGD03109 | Start Date*: 2010-12-20 | |||||||||||
Sponsor Name:Genzyme Europe B.V. | |||||||||||||
Full Title: A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients wit... | |||||||||||||
Medical condition: Gaucher Disease Type I | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: SE (Completed) PT (Completed) AT (Completed) GR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2005-001651-37 | Sponsor Protocol Number: OGT 918-011 | Start Date*: 2005-10-27 |
Sponsor Name:Actelion Pharmaceuticals Ltd | ||
Full Title: A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement ... | ||
Medical condition: Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1 BA... | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: GB (Completed) ES (Completed) IT (Completed) DE (Completed) CZ (Completed) HU (Completed) | ||
Trial results: View results |
EudraCT Number: 2008-005825-12 | Sponsor Protocol Number: PB-06-002 | Start Date*: 2009-06-04 | |||||||||||
Sponsor Name:Protalix Biotherapeutics | |||||||||||||
Full Title: Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD)... | |||||||||||||
Medical condition: Enfermedad de Gaucher Gaucher Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Completed) GB (Completed) IT (Completed) DE (Completed) Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2008-005222-37 | Sponsor Protocol Number: GZGD02507 | Start Date*: 2009-11-04 | |||||||||||
Sponsor Name:Genzyme Europe B.V. | |||||||||||||
Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 | |||||||||||||
Medical condition: Gaucher Disease type I | |||||||||||||
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Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Completed) GB (Completed) BG (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2008-005826-35 | Sponsor Protocol Number: PB-06-003 | Start Date*: 2009-06-04 | |||||||||||
Sponsor Name:Protalix Biotherapeutics | |||||||||||||
Full Title: Ensayo clínico de extensión, multicéntrico, doble ciego con dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermeda... | |||||||||||||
Medical condition: Enfermedad de Gaucher Gaucher Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Completed) GB (Completed) IT (Completed) Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2007-002840-21 | Sponsor Protocol Number: HGT-GCB-039 | Start Date*: 2007-10-30 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies Inc | |||||||||||||
Full Title: A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gauc... | |||||||||||||
Medical condition: Type I Gaucher disease Enfermedad de Gaucher tipo 1 | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Completed) GB (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2008-005223-28 | Sponsor Protocol Number: GZGD02607 | Start Date*: 2009-11-04 | |||||||||||
Sponsor Name:Genzyme Europe B.V. | |||||||||||||
Full Title: A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached ... | |||||||||||||
Medical condition: Gaucher Disease type I | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Completed) GB (Completed) FR (Completed) DE (Completed) ES (Completed) CZ (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-002044-28 | Sponsor Protocol Number: HGT-MLD-070 | Start Date*: 2012-06-14 | |||||||||||
Sponsor Name:Shire Human Genetics Therapies Inc | |||||||||||||
Full Title: A Phase I/II, Multicenter, Open-label, Dose Escalation Study of HGT-1110 Administered Intrathecally in Children with Metachromatic Leukodystrophy | |||||||||||||
Medical condition: Treatment of Metachromatic Leukodystrophy | |||||||||||||
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Population Age: Infants and toddlers, Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) DK (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-001730-26 | Sponsor Protocol Number: 205756 | Start Date*: 2017-10-30 | |||||||||||
Sponsor Name:Orchard Therapeutics (Europe) Ltd | |||||||||||||
Full Title: A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA OTL-200, for the treatment of early onset Metachromatic... | |||||||||||||
Medical condition: Metachromatic Leukodystrophy | |||||||||||||
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Population Age: Infants and toddlers, Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2012-003775-20 | Sponsor Protocol Number: HGT-MLD-071 | Start Date*: 2013-09-11 | |||||||||||
Sponsor Name:Shire Human Genetics Therapies Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited | |||||||||||||
Full Title: An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy | |||||||||||||
Medical condition: Treatment of Metachromatic Leukodystrophy | |||||||||||||
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Population Age: Infants and toddlers, Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Trial now transitioned) DK (Trial now transitioned) FR (Trial now transitioned) CZ (Trial now transitioned) GB (GB - no longer in EU/EEA) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2011-004410-42 | Sponsor Protocol Number: C11-09 | Start Date*: 2012-11-26 | |||||||||||||||||||||
Sponsor Name:Inserm | |||||||||||||||||||||||
Full Title: A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication deficient adeno-associated virus gene transfer vector serotype rh.10 expressing the human ARSA c... | |||||||||||||||||||||||
Medical condition: Early onset forms of MLD | |||||||||||||||||||||||
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Population Age: Infants and toddlers, Children, Under 18 | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: FR (Completed) | |||||||||||||||||||||||
Trial results: (No results available) |
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