UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000001996 |
|---|---|
| Receipt number | 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 20:37:44 |
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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
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
kimusige@kumamoto-u.ac.jp
Sponsor or person
Institute
Kumamoto University
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000002433
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| Registered date | File name |
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| Registered date | File name |
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