Found 32 matching trials
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease PHASE3 Although miglustat has been approved as a treatment for mild to moderate ty...
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Multiple Locations
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Phase II Study of the Safety and Surrogate Marker Efficacy of Butyldeoxynojirimycin (SC-48334) and AZT in Symptomatic HIV-1 Infected Patients With 200 - 500 CD4+ Cells/mm3. (NOTE: Asymptomatic HIV-1 Infected Patients Also Eligible) PHASE2 To assess the dose-related antiviral effects of SC-48334 and zidovudine (AZ...
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A Global Prospective Observational Registry of Patients With Pompe Disease N/A This is a global, multicenter, prospective, observational registry of patie...
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University of Arkansas Medical Science
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Glycosphingolipid Inhibition and Spermatogenesis in Man: A Pilot Study (MIG 2) PHASE4 The purpose of this research study is to help in the development of safe, e...
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University of Washington
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A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 PHASE3 This is a Phase 3, open-label, multicenter study to evaluate the safety, ef...
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University of Florida Clinical Research Center
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Application of Miglustat in Patients With Niemann-Pick Type C PHASE3 To evaluate the changes in cognitive function after miglustat treatment in ...
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National Taiwan University Hospital
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OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease PHASE2 Gaucher disease is an inherited functional deficiency of glucocerebrosidase...
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National Institutes of Health Clinical Center, 9000 Rockville Pike
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A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease PHASE2 This open-label study aims to gather long-term safety, tolerability, PK, bi...
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Associação Hospitalar de Prot à Infância Dr. Raul Carneiro
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An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease PHASE1 This is an open label study in approximately 6 subjects in 2 centers to ass...
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Texas Children Hospital
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Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation PHASE2 The purpose of this study is to demonstrate that Miglustat restores the fun...
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Assistance publique-Hôpitaux de Paris, Hôpital Cochin
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