Pharmacokinetic Study on N-acetylneuraminic Acid

Phase 1

Completed

• Conditions: Nonaka Myopathy; Hereditary Inclusion Body Myopathy
• Interventions: Drug: NPC-09

• Registration Number: NCT01236898
• Lead Sponsor: Tohoku University

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 Description

Not available

Study Timeline

• Study Start: 2010-11
• Study First Submitted: 2010-11-08
• Study First Submitted QC: 2010-11-08
• Study First Posted: 2010-11-09
• Primary Completion: 2011-06
• Study Completion: 2011-06
• Status Verified: 2011-08
• Last Update Submitted: 2011-09-07
• Last Update Posted: 2011-09-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
NPC-09	NPC-09	Period 1: NPC-09 800mg single oral dosing NPC-09 800mg three times oral dosing a day Period 2: NPC-09 800mg three times oral dosing a day for 5 consecutive days

Primary Outcome Measures

Name	Time	Method
Change in serum concentration of N-acetylneuraminic acid	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	24 hours before dosing and dosing days
Change in amount of urinary excretion of N-acetylneuraminic acid	24 hours before dosing and dosing days
Safety(adverse events)	Up to 5-7 days after dosing

Secondary Outcome Measures

Name	Time	Method
Change in serum concentration of N-glycolylneuraminic acid	24 hours before dosing and dosing days
Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt	24 hours before dosing and dosing days
Change in amount of urinary excretion of N-glycolylneuraminic acid	24 hours before dosing and dosing days

Trial Locations

Locations (1)

Tohoku University Hospital; 🇯🇵 Sendai, Miyagi, Japan