UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000011532 |
|---|---|
| Receipt number | 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 18:34:32 |
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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
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
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
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
aksuzuki@hosp.tohoku.ac.jp
Sponsor or person
Institute
Tohoku University Hospital, Department of Neurology
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000013489
