Pharmacokinetic Study on N-acetylneuraminic Acid

NCT ID: NCT01236898

Last Updated: 2011-09-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-11-30

Study Completion Date

2011-06-30

Brief Summary

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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.

Detailed Description

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Conditions

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Nonaka Myopathy Hereditary Inclusion Body Myopathy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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NPC-09

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

Group Type EXPERIMENTAL

NPC-09

Intervention Type DRUG

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

Interventions

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NPC-09

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

20 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tohoku University

OTHER

Sponsor Role lead

Responsible Party

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Tohoku University Hospital Internal Neurology

Principal Investigators

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Masashi Aoki

Role: PRINCIPAL_INVESTIGATOR

Tohoku University Hospital Internal Neurology

Locations

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Tohoku University Hospital

Sendai, Miyagi, Japan

Site Status

Countries

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Japan

Other Identifiers

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Sialic acid-1

Identifier Type: -

Identifier Source: org_study_id

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