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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
  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:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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:
Distal Myopathies
Muscular Diseases
Genetic Diseases, Inborn
Muscular Disorders, Atrophic
Muscular Dystrophies
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases

ClinicalTrials.gov processed this record on November 25, 2014