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)

Stopped early · Phase 3

Conditions studied: Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy

In brief

The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.

Key facts

Study ID: NCT02736188
Run by: Ultragenyx Pharmaceutical Inc
People needed: 143
Starts: 2016-05-02
Expected to finish: 2018-01-10
Last updated by the study team: 2023-03-24

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Have completed UX001-CL202, UX001-CL301 or UX001-CL203 study
Willing and able to provide written, signed informed consent after the nature of the study has been explained, and before any research-related procedures are conducted
Willing to comply with all study procedures
Female participants of child-bearing potential or male participants with female partners of child-bearing potential who have not undergone a bilateral salpingo-oophorectomy and are sexually active must consent to use a highly effective method of contraception as determined by the site investigator (i.e., oral hormonal contraceptives, patch hormonal contraceptives, vaginal ring, intrauterine device, physical double-barrier methods, surgical hysterectomy, vasectomy, tubal ligation or true abstinence [when this is in line with the preferred and usual lifestyle of the subject], which means not having sex because the subject chooses not to), from the period following the signing of the informed consent through 30 days after last dose of study drug
Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause for at least two years, have had tubal ligation at least one year prior to Screening, or who have had a total hysterectomy or bilateral salpingo oophorectomy

You may not qualify if…

Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
Ingestion of N-acetyl-D-mannosamine (ManNAc) or related metabolites; intravenous immunoglobulin (IVIG); or anything that can be metabolized to produce SA in the body within 60 days prior to the Screening Visit
Has had any hypersensitivity to sialic acid (SA) or its excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects
Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study
Use of any investigational product or investigational medical device within 30 days prior to Screening, or anticipated requirement for any investigational agent prior to completion of all scheduled study assessments
Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment or may not allow safe participation in the study
Has a concurrent disease, active suicidal ideation, or other condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or would affect safety

Where it is running

UCLA — Los Angeles, California, United States
University of California, Irvine — Orange, California, United States
Washington University School of Medicine, St. Louis — St Louis, Missouri, United States
New York University School of Medicine — New York, New York, United States
Icahn School of Medicine at Mount Sinai — New York, New York, United States
UMHAT Alexandrovska, Bulgaria — Sofia, Bulgaria
McMaster University — Hamilton, Ontario, Canada
CHU La Réunion - site GHSR — Saint-Pierre, Reunion, France
Institut de Myologie GH Pitié-Salpêtrière — Paris, France
Hadassah-Hebrew University Medical Center — Jerusalem, Israel
University of Messina — Messina, Italy
University of Milan — Milan, Italy
Università Cattolica — Rome, Italy
The Newcastle upon Tyne Hospitals — Newcastle upon Tyne, Tyne and Wear, United Kingdom

Full record on ClinicalTrials.gov

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