EUCTR2014-005432-33-ITActive, not recruitingPhase 1
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) - not applicable
TRAGENYX PHARMACEUTICAL INC.0 sites89 target enrollmentStarted: January 19, 2021Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 89
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1) Male or female, aged 18 - 50 years, inclusive
- •2) 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
- •3) Have a documented diagnosis of GNEM, HIBM, DMRV, or Nonaka
- •disease due to previously demonstrated mutations in the gene encoding
- •the GNE/MNK enzyme (genotyping will not be conducted in this study)
- •4) Able to provide reproducible force in elbow flexors (i.e. two
- •dynamometry force values with no more than 15% variability in the
- •dominant arm) at Screening
- •5) Able to walk a minimum of 200 meters during the 6MWT at Screening
- •without the use of assistive devices, including a cane, crutch(es),
- •walker, wheelchair or scooter (AFOs are permitted)
- •6) Willing and able to comply with all study procedures
- •7) Participants of child-bearing potential or with partners of childbearing
- •potential who have not undergone a bilateral salpingooophorectomy
- •and are sexually active must consent to use an 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) from the period following the signing of the
- •informed consent through 3 months after last dose of study drug
- •8) 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 salpingooophorectomy
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 89
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
Exclusion Criteria
- •1) Ingestion of N-acetyl-D-mannosamine (ManNAc), SA, 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
- •2) History of more than 30 days treatment with SA-ER and/or SA-IR in
- •prior clinical trials in the past year
- •3) Has had any hypersensitivity to SA or its excipients that, in the
- •judgment of the investigator, places the subject at increased risk for
- •adverse effects
- •4) Has serum transaminase (i.e. aspartate aminotransferase [AST] or
- •gamma-glutamyl transpeptidase [GGT]) levels greater than 3X the upper
- •limit of normal (ULN) for age/gender, or serum creatinine of greater
- •than 2X ULN at Screening
- •5) Pregnant or breastfeeding at Screening or planning to become
- •pregnant (self or partner) at any time during the study
- •6) 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
- •7) 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
- •8) 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
Investigators
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