Pharmacokinetic Study on N-acetylneuraminic Acid

Hereditary Inclusion Body Myopathy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01236898
• Other study ID #: Sialic acid-1
• Status: Completed
• Phase: Phase 1
• First received: November 8, 2010
• Last updated: September 7, 2011
• Start date: November 2010
• Est. completion date: June 2011

Study information

• Verified date: August 2011
• Source: Tohoku University
• Contact: n/a
• Is FDA regulated: No
• Health authority: Japan: Pharmaceuticals and Medical Devices Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: June 2011
• Est. primary completion date: June 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 20 Years to 40 Years

Eligibility

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, ?-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

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hereditary Inclusion Body Myopathy
• Muscular Diseases
• Nonaka Myopathy

Intervention

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

Locations

Country	Name	City	State
Japan	Tohoku University Hospital	Sendai	Miyagi

Sponsors (1)

Lead Sponsor	Collaborator
Tohoku University

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Primary	Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt		24 hours before dosing and dosing days	No
Primary	Change in amount of urinary excretion of N-acetylneuraminic acid		24 hours before dosing and dosing days	No
Primary	Safety(adverse events)		Up to 5-7 days after dosing	Yes
Secondary	Change in serum concentration of N-glycolylneuraminic acid		24 hours before dosing and dosing days	No
Secondary	Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt		24 hours before dosing and dosing days	No
Secondary	Change in amount of urinary excretion of N-glycolylneuraminic acid		24 hours before dosing and dosing days	No