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Name:
UMIN ID:

Recruitment status No longer recruiting
Unique ID issued by UMIN UMIN000010964
Receipt No. R000012799
Scientific Title Exploratory Study of NS-065/NCNP-01 in Duchenne muscular dystrophy
Date of disclosure of the study information 2013/06/17
Last modified on 2014/11/07

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Basic information
Public title Exploratory Study of NS-065/NCNP-01 in Duchenne muscular dystrophy
Acronym Exploratory Study of NS-065/NCNP-01
Scientific Title Exploratory Study of NS-065/NCNP-01 in Duchenne muscular dystrophy
Scientific Title:Acronym Exploratory Study of NS-065/NCNP-01
Region
Japan

Condition
Condition Duchenne muscular dystrophy
Classification by specialty
Neurology Pediatrics
Classification by malignancy Others
Genomic information YES

Objectives
Narrative objectives1 The purpose of this study is to evaluate safety of NS-065/NCNP-01, to elucidate its pharmacokinetics and to explore biomarkers related to its efficacy against Duchenne muscular dystrophy.
Basic objectives2 Safety
Basic objectives -Others
Trial characteristics_1 Exploratory
Trial characteristics_2 Explanatory
Developmental phase Phase I

Assessment
Primary outcomes Safety (adverse event and adverse drug reaction)
Key secondary outcomes 1. Expression of dystrophin protein
2. Detection of exon53 skipped mRNA of dystrophin
3. NS-065/NCNP-01 concentration of the blood plasma
4. NS-065/NCNP-01 concentration of the urine
5. Serum Creatine kinase concentration

Base
Study type Interventional

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

Intervention
No. of arms 1
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 Cohort 1: NS-065/NCNP-01 1.25 mg/kg once weekly for 12 weeks (n=3)
Cohort 2: NS-065/NCNP-01 5.0 mg/kg once weekly for 12 weeks (n=3)
Cohort 3: NS-065/NCNP-01 20.0 mg/kg once weekly for 12 weeks (n=3 or 4)
Interventions/Control_2
Interventions/Control_3
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
5 years-old <=
Age-upper limit
18 years-old >
Gender Male
Key inclusion criteria Subject with Duchenne muscular dystrophy eligible for enrolment in the study must meet all of the following criteria:

1) 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.
2) DNA sequencing of exon 53 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-065/NCNP-01 and pre-mRNA.
3) 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.
4) Male and >= 5 years and < 18 years of age at the time of obtaining informed consent and/or assent.
5) 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.
6) Life expectancy of at least 1 year
7) Unable to ambulate. Ambulant subject can be enrolled according to the circumstances.
8) Have intact muscles, which have adequate quality for biopsy.
(No lacks or severe atrophy of tibialis anterior muscle)
9) QTc <450 msec (based on 12-lead ECGs), or <480 msec for subject with Bundle Branch Block.
10) If taking glucocorticosteroids, no significant change in total daily dosage or dosing regimen after the time of visit 1.
Key exclusion criteria Subject with Duchenne muscular dystrophy meeting any of the following criteria must not be enrolled in the study:

1)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.
2) A forced vital capacity (FVC) < 50% of predicted.
3) A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
4) 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.
5) Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening.
6) Current diagnosis of any immune deficiency or autoimmune disease.
7) Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease.
8) Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication.
9) History of any severe drug allergy.
10) 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.
11) Subject considered by the investigator (or sub-investigator), for any reason, to be an unsuitable candidate for the study.
Target sample size 10

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Hirofumi Komaki
Organization National Center of Neurology and Psychiatry
Division name Department of Child Neurology, National Center Hospital
Zip code
Address 4-1-1, Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
TEL 042-341-2711
Email komakih@ncnp.go.jp

Public contact
Name of contact person
1st name
Middle name
Last name Maki Ohata
Organization National Center of Neurology and Psychiatry
Division name Clinical Research Unit, National Center Hospital
Zip code
Address 4-1-1, Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
TEL 042-341-2711
Homepage URL
Email chiken5@ncnp.go.jp

Sponsor
Institute National Center of Neurology and Psychiatry
Institute
Department

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

Other related organizations
Co-sponsor Nippon Shinyaku Co., Ltd.
Name of secondary funder(s) Nippon Shinyaku Co., Ltd.

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs YES
Study ID_1 NCT02081625
Org. issuing International ID_1 ClinicalTrials.gov
Study ID_2
Org. issuing International ID_2
IND to MHLW 2013年5月13日(初回届)

Institutions
Institutions 独立行政法人 国立精神・神経医療研究センター(東京都)/National Center of Neurology and Psychiatry(Tokyo)

Other administrative information
Date of disclosure of the study information
2013 Year 06 Month 17 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 No longer recruiting
Date of protocol fixation
2013 Year 06 Month 13 Day
Date of IRB
Anticipated trial start date
2013 Year 06 Month 17 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2013 Year 06 Month 14 Day
Last modified on
2014 Year 11 Month 07 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000012799

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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