UMIN-CTR Clinical Trial

Basic information
Public title	Construction of database and analysis of clinical condition of spinal muscular atrophy in NHO Osaka Toneyama Medical Center
Acronym	Construction of database and analysis of clinical condition of spinal muscular atrophy in NHO Osaka Toneyama Medical Center
Scientific Title	Construction of database and analysis of clinical condition of spinal muscular atrophy in NHO Osaka Toneyama Medical Center
Scientific Title:Acronym	Construction of database and analysis of clinical condition of spinal muscular atrophy in NHO Osaka Toneyama Medical Center
Region	Japan

Condition
Condition	spinal muscular atrophy
Classification by specialty	Neurology Pediatrics Rehabilitation medicine
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To construct database of clinical condition of spinal muscular atrophy in NHO Osaka Toneyama Medical Center
Basic objectives2	Others
Basic objectives -Others	To analyze the information of database of clinical condition of spinal muscular atrophy in NHO Osaka Toneyama Medical Center
Trial characteristics_1	Exploratory
Trial characteristics_2	Pragmatic
Developmental phase	Not applicable

Assessment
Primary outcomes	General condition, respiratory status, nutritional status, changes in motor function
Key secondary outcomes	changes in motor function by drug administration

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	patient diagnosed as spinal muscular atrophy
Key exclusion criteria	patient who is not diagnosed as spinal muscular atrophy
Target sample size	50

Research contact person
Name of lead principal investigator	1st name Toshio Middle name Last name Saito
Organization	National Hospital Organization Osaka Toneyama Medical Center
Division name	Neurology Department
Zip code	560-8552
Address	5-1-1 Toneyama, Toyonaka, Osaka
TEL	06-6853-2001
Email	saito.toshio.cq@mail.hosp.go.jp

Public contact
Name of contact person	1st name Toshio Middle name Last name Saito
Organization	National Hospital Organization Osaka Toneyama Medical Center
Division name	Neurology Department
Zip code	560-8552
Address	5-1-1 Toneyama, Toyonaka, Osaka
TEL	06-6853-2001
Homepage URL
Email	saito.toshio.cq@mail.hosp.go.jp

Sponsor
Institute	National Hospital Organization Osaka Toneyama Medical Center
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
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	Department of Clinical Research, National Hospital Organization Osaka Toneyama Medical Center
Address	5-1-1 Toneyama, Toyonaka, Osaka
Tel	06-6853-2001
Email	410-chiken@mail.hosp.go.jp

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	2020 Year 09 Month 07 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	Open public recruiting
Date of protocol fixation	2020 Year 06 Month 20 Day
Date of IRB	2020 Year 07 Month 27 Day
Anticipated trial start date	2020 Year 09 Month 07 Day
Last follow-up date	2025 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other

Other related information

Research object code, birth year and month, gender,Diagnostic basis, Motor milestone, etc. Clinical information: Height, body weight, motor function (ambulant, wheel chair, bed ridden,etc) Respiratory function (voluntary, non-invasive ventilation, invasive ventilation, time of introducing, etc) Nutritional method (per oral, tube feeding, gastrostomy tube, time of introducing,etc) Other complication (heart failure, etc) Medication Evaluation of rehabilitation: Manual muscle test:MMT, Hammersmith Functional Motor Scale-Expanded:HFMSE for SMA, revised upper limb mudule: RULM, grip power, pinch power, 2-minute walk distance, 10-meter walking evaluation, VC,CPF,MIC, etc Clinical laboratory test: Blood analysis, blood chemistry, atrial blood gas analysis Chest x-ray, spine x-ray, MRI(brain, muscle, etc), CT (brain, muscle, chest, etc), echocardiography, electrocardiography, respiratory function (FVC,%FVC, etc)

Management information
Registered date	2020 Year 09 Month 05 Day
Last modified on	2021 Year 03 Month 07 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000047595