Pharmacokinetic Study on N-acetylneuraminic Acid

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

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.

Study Overview

• Status: Completed
• Conditions: Hereditary Inclusion Body Myopathy; Nonaka Myopathy
• Intervention / Treatment: Drug: NPC-09

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• Japan
  • Miyagi
    • Sendai, Miyagi, Japan, 980-8574
      • Tohoku University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 38 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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	Drug: NPC-09; NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in serum concentration of N-acetylneuraminic acid	Patients with DMRV(hIBM) show low serum N-acetylneuraminic acid level.	24 hours before dosing and dosing days
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

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Tohoku University
• Investigators: Principal Investigator: Masashi Aoki, Tohoku University Hospital Internal Neurology

Study record dates

Study Major Dates

• Study Start: November 1, 2010
• Primary Completion (Actual): June 1, 2011
• Study Completion (Actual): June 1, 2011

Study Registration Dates

• First Submitted: November 8, 2010
• First Submitted That Met QC Criteria: November 8, 2010
• First Posted (Estimate): November 9, 2010

Study Record Updates

• Last Update Posted (Estimate): September 8, 2011
• Last Update Submitted That Met QC Criteria: September 7, 2011
• Last Verified: August 1, 2011

More Information

Terms related to this study

• Keywords: pharmacokinetics; N-acetylneuraminic acid,; hIBM
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Diseases; Distal Myopathies
• Other Study ID Numbers: Sialic acid-1