Pharmacokinetic Study on N-acetylneuraminic Acid

Tohoku University6 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Nonaka Myopathy Hereditary Inclusion Body Myopathy

Interventions

NPC-09DRUG

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

Eligibility

Sex: ALLMin age: 20 YearsMax age: 40 Years

Medical Language ↔ Plain English

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

Locations (1)

Tohoku University Hospital

Sendai, Miyagi, Japan