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 @ 12:43 PM
Ignite Modification Date:
2025-12-24 @ 12:43 PM
NCT ID:
NCT02346461
Eligibility Criteria:
* INCLUSION CRITERIA:
* Subject is age 18-60 years, inclusive, and of either gender.
* Subject has a diagnosis of GNE myopathy based upon a consistent clinical course and identification of two GNE gene mutations.
* Subject must be willing to stop any treatment with ManNAc, sialic acid, intravenous immunoglobulin (IVIG), and/or other supplements containing sialic acid (e.g. St. John s wort, sialyllactose) 90 days prior to dosing and remain off such treatment for the duration of the trial.
* Subjects must have a body mass index (BMI) between 18 and 30 kg/m2, with a bodyweight of \>50 kg.
* Subjects must have 20-75% of predicted strength measured by QMA at baseline on at least one of the following: 1) ankle dorsiflexion, 2) knee flexion, 3) hip extension, 4) grip, 5) elbow flexion, shoulder abduction
* 20-75% of predicted strength measures by OMA at baseline, or
* If predicted muscle strength above 75%, a documented change of at least 10% per year.
* Subject has the ability to travel to the NIH Clinical Center for admissions.
* Subject has an INR less than or equal to 1.5 and must have stopped warfarin and other anticoagulants 2 weeks prior and after muscle biopsy procedures. Aspirin and clopidogrel should be stopped 3 days and 5 days before the procedure, respectively.
* Subject must be able to comply with requirements of the protocol, including blood collection, drug administration, muscle MRI/MRS, muscle biopsy and muscle strength assessments.
* If a woman of reproductive age, subject must be willing to use an effective method of contraception for the duration of the trial.
* Subject must be able to provide informed consent.
EXCLUSION CRITERIA:
* Subject had a clinical significant infection or medical illness 30 days prior to the first protocol visit.
* 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, panic disorder, or behavioral problems, which interfere with effective communication.
* 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 known adverse reactions to anesthetic or sedatives utilized for muscle biopsy.
* Subject is anemic (defined as Hematocrit \<30%) or has platelets \<100,000 or white blood cell count less than 3,000.
* 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 less than 90 days prior to the first protocol visit.
* Subject has hypersensitivity to ManNAc or in the judgment of the investigator, has a condition that places the subject at increased risk for adverse effects.
* Subject has received ManNAc, sialic acid, intravenous immunoglobulin (IVIG), and/or other supplements containing sialic acid (e.g. St. John s wort, sialyllactose) less than 90 days prior to the first protocol visit.
* The presence of persistent diarrhea or malabsorption that could interfere with the subject s ability to absorb drugs or to tolerate ManNAc therapy.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
18 Years
Maximum Age:
60 Years
Study:
NCT02346461
Study Brief:
Protocol Section:
NCT02346461