UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000062551 |
|---|---|
| Receipt number | R000071504 |
| Scientific Title | Side effects and their management associated with DMD gene therapy: A study based on real-world clinical data in Japan. |
| Date of disclosure of the study information | 2026/08/18 |
| Last modified on | 2026/08/11 10:30:22 |
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Basic information
Public title
Side effects and their management associated with DMD gene therapy: A study based on real-world clinical data in Japan.
Acronym
Real-world clinical data study of DMD gene therapy
Scientific Title
Side effects and their management associated with DMD gene therapy: A study based on real-world clinical data in Japan.
Scientific Title:Acronym
Real-world clinical data study of DMD gene therapy
Region
| Japan |
Condition
Condition
Duchenne Muscular Dystrophy (DMD)
Classification by specialty
| Neurology | Pediatrics | Child |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
This study aims to clarify the real-world profile of side effects following the administration of a novel gene therapy (delandistrogene moxeparvovec) for Duchenne Muscular Dystrophy (DMD). By retrospectively analyzing anonymized real-world clinical data collected through the online consultation platform "Medii E-Consult" as part of the safety management project by the Japanese Society of Child Neurology, we will systematically evaluate the incidence, severity, and clinical course of side effects. This study aims to generate objective evidence, such as establishing side effect management guidelines, to contribute to the safety and health protection of future patients receiving this treatment.
Basic objectives2
Others
Basic objectives -Others
The primary objective is the evaluation of Safety, specifically:To clarify the incidence (frequency and severity classification) of side effects associated with DMD gene therapy. To evaluate the clinical features of severe complications, such as hepatotoxicity and myocarditis caused by the AAV vector, and myositis induced by the micro-dystrophin protein. To evaluate therapeutic interventions for side effects and their outcomes (e.g., recovery, sequelae, necessity of continuous treatment).
Trial characteristics_1
Exploratory
Trial characteristics_2
Explanatory
Developmental phase
Not applicable
Assessment
Primary outcomes
Incidence rate and severity of side effects associated with DMD gene therapy (Evaluation period: 3 months post-administration).
Key secondary outcomes
Incidence and clinical features of hepatotoxicity and myocarditis caused by AAV vector (Evaluation period: 3 months post-administration).
Expression patterns of myositis induced by micro-dystrophin protein (Evaluation period: 3 months post-administration).
Therapeutic interventions for side effects and their outcomes (e.g., recovery, sequelae, necessity of continuous treatment) (Evaluation period: 3 months post-administration).
Changes in blood test parameters (AST, ALT, CK, GGT, albumin, PT%, PT-INR, total bilirubin, direct bilirubin, ammonia, D-dimer, ALP, cardiac troponin I, anti-AAVrh74 antibody titer, etc.) (Evaluation time points: before administration, and every week up to 3 months (12 weeks) post-administration).
Changes in cardiac function test parameters (electrocardiogram, echocardiography) (Evaluation time points: before administration, week 1, week 4, and 3 months (12 weeks) post-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
| 3 | years-old | <= |
Age-upper limit
| 8 | years-old | > |
Gender
Male and Female
Key inclusion criteria
Patients diagnosed with DMD.
Patients who received gene therapy with delandistrogene moxeparvovec.
Cases for which expert consultation was conducted via Medii E-Consult after the treatment.
Patients aged 3 years to less than 8 years (within the indicated age range).
Key exclusion criteria
Cases with incomplete data judged unsuitable for analysis.
Cases without data after the initiation of gene therapy.
Cases where written informed consent for participation in this study could not be obtained.
Target sample size
200
Research contact person
Name of lead principal investigator
| 1st name | Hirofumi |
| Middle name | |
| Last name | Komaki |
Organization
National Center of Neurology and Psychiatry
Division name
Translational Medical Center
Zip code
187-8551
Address
4-1-1, Ogawahigashi, Kodaira, Tokyo, Japan
TEL
0423412711
komakihiro@ncnp.go.jp
Public contact
Name of contact person
| 1st name | HIROFUMI |
| Middle name | |
| Last name | KOMAKI |
Organization
National Center of Neurology and Psychiatry
Division name
Translational Medical Center
Zip code
187-8551
Address
4-1-1, Ogawahigashi, Kodaira, Tokyo, Japan
TEL
0423412711
Homepage URL
komakihiro@ncnp.go.jp
Sponsor or person
Institute
National Center of Neurology and Psychiatry
Institute
Department
Personal name
Hirofumi Komaki
Funding Source
Organization
National Center of Neurology and Psychiatry
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
National Center of Neurology and Psychiatry
Address
4-1-1, Ogawahigashi, Kodaira, Tokyo, Japan
Tel
0423412711
rinri-jimu@ncnp.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
北海道大学（北海道）、宮城県立こども病院（宮城県）、国立成育医療研究センター（東京都）、東京女子医科大学（東京都）、国立精神・神経医療研究センター（東京都）、長野県立こども病院（長野県）、名古屋市立大学（愛知県）、名古屋大学（愛知県）、大阪母子医療センター（大阪府）、神戸大学（兵庫県）、兵庫医科大学（兵庫県）、鳥取大学（鳥取県）、九州大学（福岡県）
Other administrative information
Date of disclosure of the study information
| 2026 | Year | 08 | Month | 18 | 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
Enrolling by invitation
Date of protocol fixation
| 2026 | Year | 07 | Month | 01 | Day |
Date of IRB
| 2026 | Year | 07 | Month | 03 | Day |
Anticipated trial start date
| 2026 | Year | 07 | Month | 04 | Day |
Last follow-up date
| 2030 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
None
Management information
Registered date
| 2026 | Year | 08 | Month | 11 | Day |
Last modified on
| 2026 | Year | 08 | Month | 11 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000071504
