UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000032431 |
|---|---|
| Receipt number | R000036974 |
| Scientific Title | Evaluation of safety and efficacy of inhibitor of phosphodiesterase 5 to patients with dystrophinopathy |
| Date of disclosure of the study information | 2018/04/30 |
| Last modified on | 2018/04/30 15:30:53 |
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Basic information
Public title
Evaluation of safety and efficacy of inhibitor of phosphodiesterase 5 to patients with dystrophinopathy
Acronym
Safety and efficacy of PDE5 inhibitor to dystrophinopathy
Scientific Title
Evaluation of safety and efficacy of inhibitor of phosphodiesterase 5 to patients with dystrophinopathy
Scientific Title:Acronym
Safety and efficacy of PDE5 inhibitor to dystrophinopathy
Region
| Japan |
Condition
Condition
patients with dystrophinopathy
Classification by specialty
| Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
To Evaluate safety and efficacy of inhibitor of phosphodiesterase 5 to patients with dystrophinopathy
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
manual muscle test
Key secondary outcomes
6 minutes walk test(ambulant case)
VC, MIC, CPF
serum CK, CD34, BNP
EF (UCG)
evaluation time:
before administration of PDE5 inhibitor,
14 days after administration,
28 days after finishing administration
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
We use shildenafil as PDE5 inhibitor.
Observational period is assigned from the day before shildenafil administration to 28 days after finishing administration.
Shildenafil administration is planned as follows; 0.25mg/kg (start~day 3),0.5mg/kg (day 4 ~ day 7), and 1.0mg/kg (day 8~ day 14).
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
| 7 | years-old | <= |
Age-upper limit
| 50 | years-old | >= |
Gender
Male
Key inclusion criteria
patients with dystrophinopathy based on gene analysis or muscle biopsy findings
Key exclusion criteria
patients with 24-hour mechanical ventilation (not include night NPPV)
hypertension(Sys>135mmHg or Diast>90mmHg)
heart failure with BNP>100pg/ml or LVEF<50%
arrhythmia (Af,AF, VT,PSVT)
diabetus mellitus
Target sample size
10
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Toshio Saito |
Organization
National Hospital Organization Toneyama National Hospital
Division name
Division of Child Neurology, Department of Neurology
Zip code
Address
5-1-1 Toneyama, Toyonaka, Osaka
TEL
+81-6-6853-2001
saitot@toneyama.go.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Toshio Saito |
Organization
National Hospital Organization Toneyama National Hospital
Division name
Division of clinical research
Zip code
Address
5-1-1 Toneyama, Toyonaka, Osaka
TEL
+81-6-6853-2001
Homepage URL
saitot@toneyama.go.jp
Sponsor or person
Institute
National Hospital Organization Toneyama National Hospital
Institute
Department
Personal name
Funding Source
Organization
Grants-in-Aid for Scientific Research
Organization
Division
Category of Funding Organization
Japanese Governmental office
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
| 2018 | Year | 04 | Month | 30 | 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
Completed
Date of protocol fixation
| 2012 | Year | 08 | Month | 03 | Day |
Date of IRB
Anticipated trial start date
| 2012 | Year | 08 | Month | 03 | 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
| 2018 | Year | 04 | Month | 30 | Day |
Last modified on
| 2018 | Year | 04 | Month | 30 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036974
