Duchenne Muscular DystrophyPhase 1NCT02081625
Exploratory Study of NS-065/NCNP-01 in DMD
A Phase 1 study testing NS-065/NCNP-01 in people with Duchenne Muscular Dystrophy, run by National Center of Neurology and Psychiatry, Japan at 1 site in Japan.
Trial summary
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-065/NCNP-01 in subjects diagnosed with Duchenne muscular dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 5 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- NS-065/NCNP-01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteJapan
- Study dates
- Jun 2013to Aug 2015
- 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 5 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subject with Duchenne muscular dystrophy eligible for enrolment in the study must meet all of the following criteria:
- Has an out of frame deletion(s) that could be corrected by skipping exon 53 as confirmed by any of methodology at the time of visit 1. If not confirmed by any of methodology that evaluates the relative copy number of all exons (i.e. MLPA, CGH etc), must be confirmed through these techniques by the time of visit 4.
- DNA sequencing of exon 53 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-065/NCNP-01 and pre-mRNA.
- There is confirmation of detection of dystrophin mRNA with skipping of exon 53 and dystrophin production after in vitro exposure of NS-065/NCNP-01 to subject-derived cells.
- Male and >= 5 years and < 18 years of age at the time of obtaining informed consent and/or assent.
- Able to give informed consent in writing signed by parent(s) or legal guardian who is able to understand all of the study procedure requirements. If applicable, able to give informed assent in writing signed by the subject.
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- Life expectancy of at least 1 year
- Unable to ambulate. Ambulant subject can be enrolled according to the circumstances.
- Have intact muscles, which have adequate quality for biopsy. (No lacks or severe atrophy of tibialis anterior muscle)
- QTc <450 msec (based on 12-lead ECGs), or <480 msec for subject with Bundle Branch Block.
- If taking glucocorticosteroids, no significant change in total daily dosage or dosing regimen after the time of visit 1.
Who cannot join
- Subject with Duchenne muscular dystrophy meeting any of the following criteria must not be enrolled in the study:
- Has participated in other pharmacological clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
- A forced vital capacity (FVC) < 50% of predicted.
- A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
- Surgery within the last 3 months prior to the first anticipated administration of study medication or planned for anytime during the duration of the study.
- Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening.
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- Current diagnosis of any immune deficiency or autoimmune disease.
- Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease.
- Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication.
- History of any severe drug allergy.
- Unable to give informed consent about using adequate contraception from the first administration until at least 6 months after the last dose of study medication, by parent(s) or legal guardian.
- Subject considered by the investigator (or sub-investigator), for any reason, to be an unsuitable candidate for the study.
- -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Exploratory Study of NS-065/NCNP-01 in Duchenne Muscular Dystrophy
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-065/NCNP-01 in subjects diagnosed with Duchenne muscular dystrophy (DMD).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- NS-065/NCNP-01
- Keywords
- Duchenne muscular dystrophy, NS-065/NCNP-01, exon 53 skipping, morpholino
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NS-065/NCNP-01Drug
NS-065/NCNP-01 for Infusion is packaged as 25 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows:
Cohort 1: 1.25mg/kg once weekly for 12 weeks; Cohort 2: 5.0mg/kg once weekly for 12 weeks; Cohort 3: 20.0mg/kg once weekly for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| NS-065/NCNP-01 | Experimental | Not described Receives: Drug: NS-065/NCNP-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability (adverse event and adverse drug reaction)
Measured: Up to 15-17 weeks (12 weeks treatment period and 3-5 weeks follow up period)
Secondary outcomes (5)
Expression of dystrophin protein
Measured: At 14-15 weeks (2-3 week after from 12 weeks treatment period)
Detection of exon53 skipped mRNA of dystrophin
Measured: At 14-15 weeks (2-3 week after from 12 weeks treatment period)
NS-065/NCNP-01 concentration of the blood plasma
Measured: 12 weeks
NS-065/NCNP-01 concentration of the urine
Measured: 12 weeks
Serum Creatine kinase concentration
Measured: 14 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2014 (estimated)
- Study startJun 2013
- Primary completionNov 2014
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Center of Neurology and Psychiatry, Japan
- Sponsor type
- University, hospital or other
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Shin'ichi Takeda, MD, PhD, Study director, National center of Neurology and PsychiatryHirofumi Komaki, MD, PhD, Principal investigator, National center of Neurology and Psychiatry
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02081625
- Sponsor's protocol ID
- NCNP/DMT01
- Other IDs
- UMIN000010964
- Status last verified
- Feb 2020
- First submitted
- 5 Mar 2014
- Last updated
- 26 Feb 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploratory Study of NS-065/NCNP-01 in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02081625
- Acronym / sponsor ID
- NCNP/DMT01
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, NS-065/NCNP-01, exon 53 skipping, morpholino
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- National Center of Neurology and Psychiatry, JapanUniversity, hospital or other
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Study start
- Jun 2013
- Primary completion
- Nov 2014 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 7 Mar 2014 (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.
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-065/NCNP-01 in subjects diagnosed with Duchenne muscular dystrophy (DMD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NS-065/NCNP-01 | Experimental | Drug: NS-065/NCNP-01 |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Subject with Duchenne muscular dystrophy eligible for enrolment in the study must meet all of the following criteria:
- Has an out of frame deletion(s) that could be corrected by skipping exon 53 as confirmed by any of methodology at the time of visit 1. If not confirmed by any of methodology that evaluates the relative copy number of all exons (i.e. MLPA, CGH etc), must be confirmed through these techniques by the time of visit 4.
- DNA sequencing of exon 53 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-065/NCNP-01 and pre-mRNA.
- There is confirmation of detection of dystrophin mRNA with skipping of exon 53 and dystrophin production after in vitro exposure of NS-065/NCNP-01 to subject-derived cells.
- Male and >= 5 years and < 18 years of age at the time of obtaining informed consent and/or assent.
Key exclusion criteria (13 listed)
- Subject with Duchenne muscular dystrophy meeting any of the following criteria must not be enrolled in the study:
- Has participated in other pharmacological clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
- A forced vital capacity (FVC) < 50% of predicted.
- A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
- Surgery within the last 3 months prior to the first anticipated administration of study medication or planned for anytime during the duration of the study.
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 |
|---|---|---|
| NS-065/NCNP-01NS-065/NCNP-01 for Infusion is packaged as 25 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows: Cohort 1: 1.25mg/kg once weekly for 12 weeks; Cohort 2: 5.0mg/kg once weekly for 12 weeks; Cohort 3: 20.0mg/kg once weekly for 12 weeks | Drug | NS-065/NCNP-01 |
Primary outcome
Safety and tolerability (adverse event and adverse drug reaction)
Time frame: Up to 15-17 weeks (12 weeks treatment period and 3-5 weeks follow up period)
5 secondary outcomes
Expression of dystrophin protein
Time frame: At 14-15 weeks (2-3 week after from 12 weeks treatment period)
Detection of exon53 skipped mRNA of dystrophin
Time frame: At 14-15 weeks (2-3 week after from 12 weeks treatment period)
NS-065/NCNP-01 concentration of the blood plasma
Time frame: 12 weeks
NS-065/NCNP-01 concentration of the urine
Time frame: 12 weeks
Serum Creatine kinase concentration
Time frame: 14 weeks
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 26 Feb 2020.
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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.
Shin'ichi Takeda, MD, PhD study director
National center of Neurology and Psychiatry
Hirofumi Komaki, MD, PhD principal investigator
National center of Neurology and Psychiatry
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 NS-065/NCNP-01 in people with Duchenne Muscular Dystrophy, run by National Center of Neurology and Psychiatry, Japan 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 11 inclusion and 13 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 26 Feb 2020. 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?
Safety and tolerability (adverse event and adverse drug reaction) (time frame: Up to 15-17 weeks (12 weeks treatment period and 3-5 weeks follow up period)). 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 26 Feb 2020.