Pharmacokinetic Study on N-acetylneuraminic Acid

NCT01236898 | Completed Phase 1 DMC

• 1 other identifier: Sialic acid-1
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Nonaka Myopathy; Hereditary Inclusion Body Myopathy

Keywords

N-acetylneuraminic acid,; pharmacokinetics; hIBM

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (3)

• 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

Study Arms (1)

NPC-09

EXPERIMENTAL

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

Interventions

NPC-09 DRUG

NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride)

NPC-09

Eligibility Criteria

• Age: 20 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Confirmed mutations in GNE gene
• No severe complications when informed consent is obtained
• More than 40 kg in weight before administration

You may not qualify if:

• 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

Sponsors & Collaborators

• Tohoku University: lead

Study Sites (1)

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Location

MeSH Terms

Conditions

Distal myopathy, Nonaka type

Study Officials

• Masashi Aoki

  Tohoku University Hospital Internal Neurology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: November 8, 2010
• First Posted: November 9, 2010
• Study Start: November 1, 2010
• Primary Completion: June 1, 2011
• Study Completion: June 1, 2011
• Last Updated: September 8, 2011

Record last verified: 2011-08

Locations

JP (1)