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Pharmacokinetic Study on N-acetylneuraminic Acid

Primary Purpose

Nonaka Myopathy, Hereditary Inclusion Body Myopathy

Status

Completed

Phase

Phase 1

Locations

Japan

Study Type

Interventional

Intervention

NPC-09

About this trial

This is an interventional treatment trial for Nonaka Myopathy focused on measuring N-acetylneuraminic acid,, pharmacokinetics, hIBM

Eligibility Criteria

20 Years - 40 Years (Adult)All SexesDoes not accept 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

Sites / Locations

Tohoku University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

NPC-09

Arm 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

Outcomes

Primary Outcome Measures

Change in serum concentration of N-acetylneuraminic acid

Patients with DMRV(hIBM) show low serum N-acetylneuraminic acid level.

Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt

Change in amount of urinary excretion of N-acetylneuraminic acid

Safety(adverse events)

Secondary Outcome Measures

Change in serum concentration of N-glycolylneuraminic acid

Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt

Change in amount of urinary excretion of N-glycolylneuraminic acid

Full Information

NCT ID

NCT01236898

First Posted

November 8, 2010

Last Updated

September 7, 2011

Sponsor

Tohoku University

1. Study Identification

Unique Protocol Identification Number

NCT01236898

Brief Title

Pharmacokinetic Study on N-acetylneuraminic Acid

Official Title

Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)

Study Type

Interventional

2. Study Status

Record Verification Date

August 2011

Overall Recruitment Status

Completed

Study Start Date

November 2010 (undefined)

Primary Completion Date

June 2011 (Actual)

Study Completion Date

June 2011 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Tohoku University

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Nonaka Myopathy, Hereditary Inclusion Body Myopathy

Keywords

N-acetylneuraminic acid,, pharmacokinetics, hIBM

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

NPC-09

Arm Type

Experimental

Arm 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

Intervention Type

Drug

Intervention Name(s)

NPC-09

Intervention Description

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

Primary Outcome Measure Information:

Title

Change in serum concentration of N-acetylneuraminic acid

Description

Patients with DMRV(hIBM) show low serum N-acetylneuraminic acid level.

Time Frame

24 hours before dosing and dosing days

Title

Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt

Time Frame

24 hours before dosing and dosing days

Title

Change in amount of urinary excretion of N-acetylneuraminic acid

Time Frame

24 hours before dosing and dosing days

Title

Safety(adverse events)

Time Frame

Up to 5-7 days after dosing

Secondary Outcome Measure Information:

Title

Change in serum concentration of N-glycolylneuraminic acid

Time Frame

24 hours before dosing and dosing days

Title

Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt

Time Frame

24 hours before dosing and dosing days

Title

Change in amount of urinary excretion of N-glycolylneuraminic acid

Time Frame

24 hours before dosing and dosing days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

20 Years

Maximum Age & Unit of Time

40 Years

Accepts Healthy Volunteers

Eligibility 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: 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Masashi Aoki

Organizational Affiliation

Tohoku University Hospital Internal Neurology

Official's Role

Principal Investigator

Facility Information:

Facility Name

Tohoku University Hospital

City

Sendai

State/Province

Miyagi

ZIP/Postal Code

980-8574

Country

Japan

12. IPD Sharing Statement

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Pharmacokinetic Study on N-acetylneuraminic Acid

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