UMIN-CTR Clinical Trial

Basic information
Public title	Quantification of myocardial fibrosis with cardiovascular magnetic resonance imaging in patients with muscular dystrophy
Acronym	Quantification of myocardial fibrosis with CMR in muscular dystrophy
Scientific Title	Quantification of myocardial fibrosis with cardiovascular magnetic resonance imaging in patients with muscular dystrophy
Scientific Title:Acronym	Quantification of myocardial fibrosis with CMR in muscular dystrophy
Region	Japan

Condition
Condition	Muscular dystrophy
Classification by specialty	Cardiology Neurology Pediatrics Adult
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Our objective is to evaluate and predict the progression of myocardial fibrosis in patients with muscular dystrophy or suspected muscular dystrophy carriers using cardiovascular magnetic resonance imaging including T1 mapping.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	The prespecified primary outcome of the study is to assess improvement of left ventricular ejection fraction 24 months after the first cardiovascular magnetic resonance imaging in patients with muscular dystrophy, suspected muscular dystrophy carriers, and volunteers.
Key secondary outcomes	The prespecified secondary outcomes of the study are as follows: 1. To assess predictors of the occurrence of left ventricular dysfunction (left ventricular ejection fraction <50% or decrease by 5% or more per year) in patients with muscular dystrophy or suspected muscular dystrophy carriers. 2. To assess the association between absolute change and percent change in left ventricular ejection fraction per year and baseline values of the extent of late gadolinium enhancement, T1 mapping measurements, circumferential strain analysis by the tagging method, coronary flow reserve, echocardiography measurements, and biomarkers. 3. To assess the cross-sectional and longitudinal association between left ventricular ejection fraction, the extent of late gadolinium enhancement, T1 mapping measurements, circumferential strain analysis by the tagging method, coronary flow reserve, echocardiography measurements, and biomarkers. 4. To assess predictors of all-cause death, cardiovascular death, left ventricular assist device implantation, cardiac transplantation, heart failure admission, life-threatening arrhythmia (ventricular tachycardia, ventricular fibrillation).

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	6 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	Patient group: 1. Patients 6 years of age or older are eligible. 2. Patients diagnosed as Duchenne muscular dystrophy, Becker muscular dystrophy, Emery-Dreifuss muscular dystrophy, limb-girdle muscular dystrophy, or myotonic dystrophy are eligible. 3. Patients who are scheduled for cardiac magnetic resonance imaging are eligible. 4. Patients who can be scheduled for cardiac magnetic resonance imaging 24 months after the first scan are eligible. 5. Patients who provided informed consent, parental permission, or assent are eligible. Muscular dystrophy carrier group: 1. Proven or suspected muscular dystrophy carriers 20 years of age or older are eligible. 2. Proven or suspected carriers of Duchenne or Becker muscular dystrophy who have at least one first-degree male relatives with a previously established Duchenne or Becker muscular dystrophy diagnosis. 3. Proven or suspected carriers who are scheduled for cardiac magnetic resonance imaging are eligible. 4. Proven or suspected carriers who can be scheduled for cardiac magnetic resonance imaging 24 months after the first scan are eligible. 5. Proven or suspected carriers who provided informed consent are eligible. Normal volunteer group: 1. Persons 20 years of age or older are eligible. 2. Persons without cardiac or kidney disease are eligible. 3. Persons who provided informed consent are eligible.
Key exclusion criteria	1. Patients with impaired renal function (estimated glomerular filtration rate <30 mL/min/1.73m2) 2. Patients with claustrophobia 3. Patients expected to live a year or less 4. Patients who contact persons judge as ineligible for this study
Target sample size	420

Research contact person
Name of lead principal investigator	1st name Tadao Middle name Last name Aikawa
Organization	Faculty of Medicine and Graduate School of Medicine, Hokkaido University
Division name	Department of Cardiovascular Medicine
Zip code	060-8638
Address	Kita-15, Nishi-7, Kita-ku, Sapporo
TEL	011-706-6973
Email	aikawatadao@med.hokudai.ac.jp

Public contact
Name of contact person	1st name Tadao Middle name Last name Aikawa
Organization	Faculty of Medicine and Graduate School of Medicine, Hokkaido University
Division name	Department of Cardiovascular Medicine
Zip code	060-8638
Address	Kita-15, Nishi-7, Kita-ku, Sapporo
TEL	011-706-6973
Homepage URL
Email	aikawatadao@med.hokudai.ac.jp

Sponsor
Institute	Hokkaido University Hospital
Institute
Department

Funding Source
Organization	Japan Society for the Promotion of Science Japan Heart Foundation Watanabe Foundation Japan Intractable Diseases Research Foundation
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

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

IRB Contact (For public release)
Organization	Division of Clinical Research Administration, Hokkaido University Hospital
Address	Kita-14, Nishi-5, Kita-ku, Sapporo, 060-8648, Japan
Tel	011-706-7636
Email	crjimu@huhp.hokudai.ac.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	北海道大学病院（北海道）

Other administrative information
Date of disclosure of the study information	2019 Year 08 Month 06 Day

Related information
URL releasing protocol	https://www.jstage.jst.go.jp/article/mrms/advpub/0/advpub_bc.2020-0069/_article/-char/en
Publication of results	Published

Result
URL related to results and publications	https://www.jstage.jst.go.jp/article/mrms/advpub/0/advpub_bc.2020-0069/_article/-char/en
Number of participants that the trial has enrolled	11
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results	2020 Year 09 Month 07 Day
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Main results already published
Date of protocol fixation	2019 Year 08 Month 01 Day
Date of IRB	2019 Year 08 Month 01 Day
Anticipated trial start date	2019 Year 08 Month 06 Day
Last follow-up date	2020 Year 04 Month 27 Day
Date of closure to data entry	2020 Year 04 Month 27 Day
Date trial data considered complete	2020 Year 04 Month 27 Day
Date analysis concluded	2020 Year 04 Month 27 Day

Other

Other related information

In this prospective observational study, we evaluated left ventricular ejection fraction, the extent of late gadolinium enhancement, T1 mapping measurements (native T1 and extracellular volume fraction mapping), coronary flow reserve, circumferential strain analysis using cardiac magnetic resonance imaging, echocardiography measurements, and biomarkers to assess the primary and secondary outcomes.

Management information
Registered date	2019 Year 08 Month 06 Day
Last modified on	2020 Year 09 Month 07 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000042869