Pharmacokinetic Study on N-acetylneuraminic Acid

Tohoku University

Status and phase: Completed; Phase 1

Conditions: Hereditary Inclusion Body Myopathy; Nonaka Myopathy

Treatments: Drug: NPC-09

Study type: Interventional

Funder types: Other

Identifiers

NCT01236898

Sialic acid-1

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: All

Ages: 20 to 40 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

NPC-09

Experimental group

Description:

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

Treatment:

Drug: NPC-09