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Terminated

Terminated Clinical Trials

Study Identifier Study Name Disorders / CDG Type(s) Therapeutic approach Eligibility Study Type Industry / Institution(s) Region Location Contact Persons Direct Link
NCT02511041 Immunologic Effects of Supplemental Monosaccharide and Nucleoside Derivatives in Patients With Inherited Disorders of Glycosylation PGM3-CDG Drugs: N-Acetylglucosamine (GlcNAc) / Uridine
Administration: Oral
PGM3-CDG patients older than 2 years with immunologic abnormalities and altered glycosylation detected using clinical tests evaluating N- and O-linked glycan by mass spectroscopy Interventional Phase 1 National Institutes of Health Clinical Center

MD
United States

Jonathan J Lyons, M.D.
NCT02731690 2015-004553-41 A study to evaluate the safety of Aceneuramic Acid Extended Release (Ace-ER) tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) patients with severe ambulatory impairment GNE Myopathy Drug: Aceneuramic Acid Extended-Release tablets
(Ace-ER)
Administration: Oral
Older than 18 years with documented diagnosis Interventional Phase 2 Ultragenyx Pharmaceutical Inc

United States

,

Canada

,

Bulgaria

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NCT02736188 2016-000360-42 Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) GNE Myopathy Drug: Aceneuramic Acid Extended-Release Tablets
(Ace-ER)
Administration: Oral
Have completed UX001-CL202, UX001-CL301 or UX001-CL203 studies Interventional Phase 3 Ultragenyx Pharmaceutical Inc Europe

United States

,

Canada

,

Israel

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