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Recruitment status
Unique ID issued by UMIN UMIN000001996
Receipt No. R000002433
Scientific Title Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients
Date of disclosure of the study information 2009/06/01
Last modified on 2009/05/21

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Basic information
Public title Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients
Acronym Checking of exon skipping using human cell
Scientific Title Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients
Scientific Title:Acronym Checking of exon skipping using human cell
Region
Japan

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

Objectives
Narrative objectives1 Promising results of the antisense morpholino therapy have recently been reported on a genetic therapy aimed at restoring the reading frame of the dystrophin pre-mRNA in cells from mdx mouse model. Prior to the initiation of clinical treatment, it is very important to confirm the correct exon skipping events with antisense, because the sequences are different between the human and animal models and a few Duchenne type has in frame deletion of dystrophin. Therefore, we need a system that can easily screen sequences of the antisense and identify patients who are eligible for the therapy.
Basic objectives2 Efficacy
Basic objectives -Others
Trial characteristics_1 Confirmatory
Trial characteristics_2 Explanatory
Developmental phase Not applicable

Assessment
Primary outcomes Fibroblasts from DMD patients were isolated, induced to differentiate to the myogenic lineage by AdMyoD (adenoviral vectors encoding MyoD regulated by CAG Promoter) and transfected with antisense morpholinos, which were designed to induce exon 51 skipping. The exon-skipping event was analyzed by RT-PCR.
Key secondary outcomes

Base
Study type Interventional

Study design
Basic design Single arm
Randomization Randomized
Randomization unit
Blinding Open -but assessor(s) are blinded
Control No treatment
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms 1
Purpose of intervention Treatment
Type of intervention
Medicine Gene
Interventions/Control_1 antisense morpholino
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
0 years-old <=
Age-upper limit
30 years-old >=
Gender Male
Key inclusion criteria Duchenne muscular dystrophy, who is predicted of the restoration of the dystrophin open reading frame when the exon 51 was excluded.
Key exclusion criteria The patients who can not consent the protocol.
Target sample size 10

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Shigemi Kimura
Organization Kumamoto University Graduate School
Division name Department of Child Development,
Zip code
Address 1-1-1 Honjou Kumamoto 860-0811, Japan
TEL
Email

Public contact
Name of contact person
1st name
Middle name
Last name
Organization Kumamoto University Graduate School
Division name Department of Child Development
Zip code
Address
TEL
Homepage URL
Email kimusige@kumamoto-u.ac.jp

Sponsor
Institute Kumamoto University
Institute
Department

Funding Source
Organization This study was supported by the research grant (19-7) for Nervous and Mental Disorders from the Ministry of Health, Labour
Organization
Division
Category of Funding Organization
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

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

Institutions
Institutions

Other administrative information
Date of disclosure of the study information
2009 Year 06 Month 01 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
Date of protocol fixation
2009 Year 05 Month 12 Day
Date of IRB
Anticipated trial start date
2009 Year 06 Month 01 Day
Last follow-up date
2013 Year 03 Month 01 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2009 Year 05 Month 21 Day
Last modified on
2009 Year 05 Month 21 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000002433

Research Plan
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Research case data specifications
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Research case data
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