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Recruitment status Completed
Unique ID issued by UMIN UMIN000011532
Receipt No. R000013489
Scientific Title Pharmacokinetic study on N-acetylneuraminic acid in patients with Distal myopathy with rimmed vacuoles (DMRV) - hereditary inclusion body myopathy (hIBM)
Date of disclosure of the study information 2013/08/20
Last modified on 2015/02/04

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Basic information
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
Condition Distal myopathy with rimmed vacuoles (DMRV) or hereditary inclusion body myopathy (hIBM)
Classification by specialty
Neurology
Classification by malignancy Others
Genomic information NO

Objectives
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

Assessment
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

In outcomes field, the entry of just a few words such as "safety" or "efficiency" will not be accepted. Specify the name of outcome measures, including the time when you plan to measure. Usually, only one primary outcome is accepted. Write the other outcomes in "secondary outcomes" field.

Base
Study type Interventional

Study design
Basic design Factorial
Randomization Non-randomized
Randomization unit
Blinding Open -no one is blinded
Control Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
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

In interventions field, include the details of interventions, such as duration, amount, and frequency. If the intervention includes prescription or use of medical devices, duration is required.

Eligibility
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

Research contact person
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

Public contact
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

Sponsor
Institute Tohoku University Hospital, Department of Neurology
Institute
Department

Sponsor means an organization that is responsible for plan, deployment and
report of the research including funding management. It doesn't mean
funding agency". Therefore, all clinical trial should have the one.

Funding Source
Organization Ministry of Health, Labour and Welfare of Japan
Organization
Division
Category of Funding Organization
Nationality of Funding Organization Japan

Other related organizations
Co-sponsor Nobelpharma Co., Ltd.
Name of secondary funder(s) Ministry of Education, Culture, Sports, Science and Technology (MEXT) of Japan

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
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
Institutions 東北大学病院(宮城県)

Other administrative information
Date of disclosure of the study information
2013 Year 08 Month 20 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
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

Progress
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
Other related information

Management information
Registered date
2013 Year 08 Month 20 Day
Last modified on
2015 Year 02 Month 04 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000013489


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