Distal myopathy with rimmed vacuoles (DMRV) or hereditary inclusion body myopathy (hIBM)
Conditions
Interventions
NPC-09 2000mg single oral dosing
NPC-09 2000mg 3 times oral dosing a day
NPC-09 2000mg 3 times oral dosing a day for 7 consecutive days
Sponsors
Tohoku University Hospital, Department of Neurology
Nobelpharma Co., Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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) | — |
Countries
Japan
Contacts
Public ContactAkifumi Suzuki
Clinical Research, Innovation, and Education Center, Tohoku University Hospital (CRIETO) Department of Development Promotion
Outcome results
None listed