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Pharmacokinetic Study on N-acetylneuraminic Acid

This study has been completed.
Sponsor:
Information provided by:
Tohoku University
ClinicalTrials.gov Identifier:
NCT01236898
First received: November 8, 2010
Last updated: September 7, 2011
Last verified: August 2011
History of Changes
  Purpose

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.


Condition Intervention Phase
Nonaka Myopathy
Hereditary Inclusion Body Myopathy
 Drug: NPC-09
 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)

Resource links provided by NLM:

MedlinePlus related topics: Muscle Disorders
U.S. FDA Resources

Further study details as provided by Tohoku University:

Primary Outcome Measures:
  • Change in serum concentration of N-acetylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
  • Change in amount of urinary excretion of N-acetylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ] [ Designated as safety issue: No ]
  • Safety(adverse events) [ Time Frame: Up to 5-7 days after dosing ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change in serum concentration of N-glycolylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ] [ Designated as safety issue: No ]
  • Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt [ Time Frame: 24 hours before dosing and dosing days ] [ Designated as safety issue: No ]
  • Change in amount of urinary excretion of N-glycolylneuraminic acid [ Time Frame: 24 hours before dosing and dosing days ] [ Designated as safety issue: No ]

Enrollment: 6
Study Start Date: November 2010
Study Completion Date: June 2011
Primary Completion Date: June 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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)

  Eligibility

Ages Eligible for Study:   20 Years to 40 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01236898

Locations
Japan
Tohoku University Hospital
Sendai, Miyagi, Japan, 980-8574
Sponsors and Collaborators
Tohoku University
Investigators
Principal Investigator: Masashi Aoki Tohoku University Hospital Internal Neurology
  More Information

No publications provided

Responsible Party: Tohoku University Hospital Internal Neurology
ClinicalTrials.gov Identifier: NCT01236898     History of Changes
Other Study ID Numbers: Sialic acid-1
Study First Received: November 8, 2010
Last Updated: September 7, 2011
Health Authority: Japan: Pharmaceuticals and Medical Devices Agency

Keywords provided by Tohoku University:
N-acetylneuraminic acid,
pharmacokinetics
hIBM

Additional relevant MeSH terms:
Muscular Diseases
Distal Myopathies
Musculoskeletal Diseases
Neuromuscular Diseases
 Nervous System Diseases
Muscular Dystrophies
Muscular Disorders, Atrophic
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on June 18, 2013