Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 4:59 PM
Ignite Modification Date:
2025-12-24 @ 4:59 PM
NCT ID:
NCT01634750
Eligibility Criteria:
* INCLUSIONI CRITERIA:
* Subject is 18-70 years, either gender, inclusive.
* Subject has a diagnosis of HIBM (or IBM2, GNE myopathy, DMRV or Nonaka myopathy) based upon a consistent clinical course and identification of 2 GNE mutations. Molecular confirmation of the diagnosis will be obtained for all subjects in the study.
* Subject must be willing to stop any treatment with ManNAc, sialic acid, IVIG, and/or other supplements containing sialic acid (eg, St John s wort, sialyllactose) 30 days prior to randomization and remain off such treatment for the duration of the trial.
* Subject has the ability to travel to the NIH Clinical Center (CC) for admissions.
* Subject (if a woman of reproductive age) must be willing to use an effective method of contraception for the duration of the trial.
* Subject provides written informed consent.
EXCLUSION CRITERIA:
* Subject has a severe disease manifestation that would interfere with the ability to comply with the requirements of this protocol.
* Subject has a psychiatric illness or neurological disease that would interfere with the ability to comply with the requirements of this protocol. This includes, but is not limited to, uncontrolled/untreated psychotic depression, bipolar disorder, schizophrenia, substance abuse or dependence, antisocial personality disorder, or panic disorder.
* Subject has hepatic laboratory parameters (AST, ALT, GGTP), or renal laboratory parameters (creatinine, BUN) greater than 3 times the upper limit of normal.
* Subject has a QTcB \>450 msec (males) or QTcB \>470 msec (females).
* Subject is anemic (defined as two standard deviations below normal for age and gender).
* Subject shows evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, or gastrointestinal disease, or has a condition that requires immediate surgical intervention.
* Subject is pregnant or breastfeeding at any time during the study.
* Subject has received treatment with another investigational drug, investigational device, or approved therapy for investigational use within 4 weeks of initial screening.
* Subject has a hypersensitivity to ManNAc or in the judgment of the investigator, has a condition that places the subject at increased risk for adverse effects.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
18 Years
Maximum Age:
70 Years
Study:
NCT01634750
Study Brief:
Protocol Section:
NCT01634750