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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000032431
Receipt No. 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

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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
Email 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
Email saitot@toneyama.go.jp

Sponsor
Institute National Hospital Organization Toneyama National Hospital
Institute
Department

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
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
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
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036974

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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