Pharmacokinetic study on N-acetylneuraminic acid in patients with Distal myopathy with rimmed vacuoles (DMRV) - hereditary inclusion body myopathy (hIBM)

Phase 1

• Conditions: Distal myopathy with rimmed vacuoles (DMRV) or hereditary inclusion body myopathy (hIBM)

• Registration Number: JPRN-UMIN000011532
• Lead Sponsor: Tohoku University Hospital, Department of Neurology

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-08-20
• Study Completion: 2015-01-31
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete: follow-up complete
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

Not provided

Exclusion Criteria

-Hepatic laboratory parameters (AST, ALT, gamma-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 -History of hypersensitivity to medicine or food -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

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Pharmacokinetics and safety of NPC-09 -Change in serum concentration, pharmacokinetic parameters, amount of urinary excretion of N-acetylneuraminic acid (24 hours before dosing and dosing days) -Safety (up to 5-7 days after dosing)