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Completed

NCT Number: NCT01236898

Pharmacokinetic Study on N-acetylneuraminic Acid

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.

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Key information

Conditions

Nonaka Myopathy Distal myopathy, Nonaka type Hereditary Inclusion Body Myopathy

Age range

20 year–40 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

NPC-09

Drug

NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride)

Primary outcomes

  1. Change in serum concentration of N-acetylneuraminic acid

    Time frame: 24 hours before dosing and dosing days

    Patients with DMRV(hIBM) show low serum N-acetylneuraminic acid level.

  2. Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt

    Time frame: 24 hours before dosing and dosing days

  3. Change in amount of urinary excretion of N-acetylneuraminic acid

    Time frame: 24 hours before dosing and dosing days

  4. Safety(adverse events)

    Time frame: Up to 5-7 days after dosing

Secondary outcomes

  1. Change in serum concentration of N-glycolylneuraminic acid

    Time frame: 24 hours before dosing and dosing days

  2. Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt

    Time frame: 24 hours before dosing and dosing days

  3. Change in amount of urinary excretion of N-glycolylneuraminic acid

    Time frame: 24 hours before dosing and dosing days

Sponsors and collaborators

Lead sponsor

Tohoku University

Other

Registry information

Official study title

Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)

Important dates

Study start
2010
Primary completion
2011
Study completion
2011
First posted
Nov 9, 2010
Registry last updated
Sep 8, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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