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