Nonaka MyopathyPhase 1NCT01236898
Pharmacokinetic Study on N-acetylneuraminic Acid
A Phase 1 study testing NPC-09 in people with Nonaka Myopathy and Hereditary Inclusion Body Myopathy, run by Tohoku University at 1 site in Japan.
Trial 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. Read more about this study
- Who is it for?
- Ages 20 to 40 years
- Conditions the study is for
- Nonaka Myopathy, Hereditary Inclusion Body Myopathy
- What is being tested?
- NPC-09
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteJapan
- Study dates
- Nov 2010to Jun 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Confirmed mutations in GNE gene
- No severe complications when informed consent is obtained
- More than 40 kg in weight before administration
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)
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.
- Being tested
- NPC-09
- Keywords
- N-acetylneuraminic acid,, pharmacokinetics, hIBM
- Medical subject headings
- Distal myopathy, Nonaka type
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NPC-09Drug
NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride)
| Study group | Role | What they receive |
|---|---|---|
| NPC-09 | Experimental | 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 Receives: Drug: NPC-09 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in serum concentration of N-acetylneuraminic acid
Patients with DMRV(hIBM) show low serum N-acetylneuraminic acid level.
Measured: 24 hours before dosing and dosing days
Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt
Measured: 24 hours before dosing and dosing days
Change in amount of urinary excretion of N-acetylneuraminic acid
Measured: 24 hours before dosing and dosing days
Safety(adverse events)
Measured: Up to 5-7 days after dosing
Secondary outcomes (3)
Change in serum concentration of N-glycolylneuraminic acid
Measured: 24 hours before dosing and dosing days
Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt
Measured: 24 hours before dosing and dosing days
Change in amount of urinary excretion of N-glycolylneuraminic acid
Measured: 24 hours before dosing and dosing days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2010 (estimated)
- Study startNov 2010
- Primary completionJun 2011
- Study completionJun 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nonaka MyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tohoku University
- Sponsor type
- University, hospital or other
- Investigators
- Masashi Aoki, Principal investigator, Tohoku University Hospital Internal Neurology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01236898
- Sponsor's protocol ID
- Sialic acid-1
- Status last verified
- Aug 2011
- First submitted
- 8 Nov 2010
- Last updated
- 8 Sep 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)
- ClinicalTrials.gov ID
- NCT01236898
- Acronym / sponsor ID
- Sialic acid-1
- Keywords
- N-acetylneuraminic acid,, pharmacokinetics, hIBM
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Tohoku UniversityUniversity, hospital or other
- Study start
- Nov 2010
- Primary completion
- Jun 2011 (actual)
- Study completion
- Jun 2011 (actual)
- First posted
- 9 Nov 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NPC-09 | Experimental | Drug: NPC-09Period 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 |
Who may qualify
- Ages
- 20 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Confirmed mutations in GNE gene
- No severe complications when informed consent is obtained
- More than 40 kg in weight before administration
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NPC-09NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride) | Drug | NPC-09 |
Primary outcome
Change in serum concentration of N-acetylneuraminic acid
Time frame: 24 hours before dosing and dosing days
Patients with DMRV(hIBM) show low serum N-acetylneuraminic acid level.
Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt
Time frame: 24 hours before dosing and dosing days
Change in amount of urinary excretion of N-acetylneuraminic acid
Time frame: 24 hours before dosing and dosing days
Safety(adverse events)
Time frame: Up to 5-7 days after dosing
3 secondary outcomes
Change in serum concentration of N-glycolylneuraminic acid
Time frame: 24 hours before dosing and dosing days
Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt
Time frame: 24 hours before dosing and dosing days
Change in amount of urinary excretion of N-glycolylneuraminic acid
Time frame: 24 hours before dosing and dosing days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2011.
Related trials for Nonaka Myopathy
RecruitingPhase 1/2
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GNE Myopathy
Active, not recruitingPhase 2
Multi-Center Study of ManNAc for GNE Myopathy
GNE Myopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Masashi Aoki principal investigator
Tohoku University Hospital Internal Neurology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NPC-09 in people with Nonaka Myopathy and Hereditary Inclusion Body Myopathy, run by Tohoku University at 1 site in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Sep 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in serum concentration of N-acetylneuraminic acid (time frame: 24 hours before dosing and dosing days). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2011.