CompletedPhase 1

Pharmacokinetic Study on N-acetylneuraminic Acid

Japan6 participantsStarted 2010-11

Plain-language summary

The aim of this study is to investigate pharmacokinetics and safety of N-acetylneuraminic acid in patients with Distal myopathy with rimmed vacuoles (DMRV) - hereditary inclusion body myopathy (hIBM). Dosages are 800mg single and three times a day and 800mg 3 times a day for 5 consecutive days. N-acetylneuraminic acid and N-glycolylneuraminic acid in serum and urine are measured before and after oral administration of N-acetylneuraminic acid.

Who can participate

Age range

20 Years – 40 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Confirmed mutations in GNE gene * No severe complications when informed consent is obtained * More than 40 kg in weight before administration Exclusion Criteria: * Hepatic laboratory parameters (AST, ALT, γ-GTP) or Renal laboratory parameters (Cr, BUN) are greater than three times of upper limit of reference value * Presence or history of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, gastrointestinal, brain, psychiatric, neurologic disease * Intake of supplement contains sialic acid, St. John's wort or grapefruit within 7 days * Enrollment in another investigational study within 3 months * More than 400 mL blood donation within 3 months * Presence of alcohol or drug dependency * Women who are pregnant , breast feeding or possible to be pregnant. * Patients whom the investigator judges not to be appropriate for the subject

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in serum concentration of N-acetylneuraminic acid

Timeframe: 24 hours before dosing and dosing days

Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt

Timeframe: 24 hours before dosing and dosing days

Change in amount of urinary excretion of N-acetylneuraminic acid

Timeframe: 24 hours before dosing and dosing days

Safety(adverse events)

Timeframe: Up to 5-7 days after dosing

Trial details

NCT IDNCT01236898

SponsorTohoku University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2011-06

View on ClinicalTrials.gov More Nonaka Myopathy trials

Plain-language summary

Who can participate

Age range

20 Years – 40 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Change in serum concentration of N-acetylneuraminic acid

Timeframe: 24 hours before dosing and dosing days

Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt

Timeframe: 24 hours before dosing and dosing days

Change in amount of urinary excretion of N-acetylneuraminic acid

Timeframe: 24 hours before dosing and dosing days

Safety(adverse events)

Timeframe: Up to 5-7 days after dosing