Clinical Trial: Pharmacokinetic Study on N-acetylneuraminic Acid

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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.

Detailed Summary:
Sponsor: Tohoku University

Current Primary Outcome:

  • 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.
  • Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt [ Time Frame: 24 hours before dosing and dosing days ]
  • Change in amount of urinary excretion of N-acetylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]
  • Safety(adverse events) [ Time Frame: Up to 5-7 days after dosing ]


Original Primary Outcome:

  • 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.
  • Change in pharmacokinetic parameters of N-acetylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]
    Parameters are AUCt AUC∞ Cmax tmax λz MRTt t1/2 CL Vd
  • Change in amount of urinary excretion of N-acetylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]
  • Safety [ Time Frame: Up to 5-7 days after dosing ]


Current Secondary Outcome:

  • Change in serum concentration of N-glycolylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]
  • Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt [ Time Frame: 24 hours before dosing and dosing days ]
  • Change in amount of urinary excretion of N-glycolylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]


Original Secondary Outcome:

  • Change in serum concentration of N-glycolylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]
  • Change in pharmacokinetic parameters of N-glycolylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]
    Parameters are AUCt AUC∞ Cmax tmax λz MRTt t1/2 CL Vd
  • Change in amount of urinary excretion of N-glycolylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ]


Information By: Tohoku University

Dates:
Date Received: November 8, 2010
Date Started: November 2010
Date Completion:
Last Updated: September 7, 2011
Last Verified: August 2011