UMIN-CTR Clinical Trial

Public title

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

Acronym

Pharmacokinetic study on N-acetylneuraminic acid

Scientific Title

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

Scientific Title:Acronym

Pharmacokinetic study on N-acetylneuraminic acid

Region

Japan

Condition

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

Classification by specialty

Neurology

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

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 2000mg single and three times a day and 2000mg 3 times a day for 7 consecutive days. N-acetylneuraminic acid in serum and urine are measured before and after oral administration of N-acetylneuraminic acid.

Basic objectives2

Pharmacokinetics

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Developmental phase

Phase I

Primary outcomes

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)

Key secondary outcomes

Study type

Interventional

Basic design

Factorial

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

3

Purpose of intervention

Treatment

Type of intervention

Medicine

Interventions/Control_1

NPC-09 2000mg single oral dosing

Interventions/Control_2

NPC-09 2000mg 3 times oral dosing a day

Interventions/Control_3

NPC-09 2000mg 3 times oral dosing a day for 7 consecutive days

Interventions/Control_4

Interventions/Control_5

Interventions/Control_6

Interventions/Control_7

Interventions/Control_8

Interventions/Control_9

Interventions/Control_10

Age-lower limit

20

years-old

<=

Age-upper limit

40

years-old

>

Gender

Male and Female

Key inclusion criteria

Confirmed mutations in GNE gene
-No severe complications when informed consent is obtained
-More than 40 kg in weight before administration

Key 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

Target sample size

9

Name of lead principal investigator

1st name
Middle name
Last name	Masashi Aoki, MD, PhD

Organization

Tohoku University School of Medicine

Division name

Neurology

Zip code

Address

1-1 Seiryo-machi, Aoba-ku, Sendai, Miyagi 980-8574 Japan

TEL

022-717-7189

Email

aokim@med.tohoku.ac.jp

Name of contact person

1st name
Middle name
Last name	Akifumi Suzuki

Organization

Clinical Research, Innovation, and Education Center, Tohoku University Hospital (CRIETO)

Division name

Department of Development Promotion

Zip code

Address

1-1 Seiryo-machi, Aoba-ku, Sendai, Miyagi, 980-8574, Japan

TEL

022-717-7136

Homepage URL

Email

aksuzuki@hosp.tohoku.ac.jp

Institute

Tohoku University Hospital, Department of Neurology

Institute

Department

Personal name

Organization

Ministry of Health, Labour and Welfare of Japan

Organization

Division

Category of Funding Organization

Nationality of Funding Organization

Japan

Co-sponsor

Nobelpharma Co., Ltd.

Name of secondary funder(s)

Ministry of Education, Culture, Sports, Science and Technology (MEXT) of Japan

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

2010.10.12., 2回

Institutions

東北大学病院（宮城県）

Date of disclosure of the study information

2013

Year

08

Month

20

Day

URL releasing protocol

Publication of results

Unpublished

URL related to results and publications

Number of participants that the trial has enrolled

Results

Results date posted

Results Delayed

Results Delay Reason

Date of the first journal publication of results

Baseline Characteristics

Participant flow

Adverse events

Outcome measures

Plan to share IPD

IPD sharing Plan description

Recruitment status

Completed

Date of protocol fixation

2013

Year

06

Month

27

Day

Date of IRB

Anticipated trial start date

2013

Year

09

Month

01

Day

Last follow-up date

2015

Year

01

Month

31

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2013

Year

08

Month

20

Day

Last modified on

2015

Year

02

Month

04

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000013489