Evidence map›Paper›PMID 40814256›Full record

ArticleNeuropsychopharmacology reports2025

Nonrandomized Allocation of Steroid Therapy in Patients With Fukuyama Congenital Muscular Dystrophy: Study Protocol for a Phase II Clinical Trial.

Terumi Murakami, Takatoshi Sato, Takami Ishizuka, Harumasa Nakamura, Hisateru Tachimori, Hiroko Harada, Hideki Oi, Kenji Hatano, Mari S Oba, Kumiko Ishiguro and 10 more

Abstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in Neuropsychopharmacology reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

20 authors.

Terumi MurakamiDepartment of Pediatrics, Tokyo Women's Medical University School of Medicine, Shinjuku-ku, Tokyo, Japan.
Takatoshi SatoDepartment of Pediatrics, Tokyo Women's Medical University School of Medicine, Shinjuku-ku, Tokyo, Japan.
Takami IshizukaDepartment of Clinical Research Support, Clinical Research and Education Promotion Division, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.ORCID https://orcid.org/0000-0003-3641-3328
Harumasa NakamuraDepartment of Clinical Research Support, Clinical Research and Education Promotion Division, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.
Hisateru TachimoriDepartment of Health Policy and Management, Keio University School of Medicine, Shinjuku-ku, Tokyo, Japan.
Hiroko HaradaDepartment of Clinical Research Support, Clinical Research and Education Promotion Division, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.
Hideki OiDepartment of Clinical Data Science, Clinical Research and Education Promotion Division, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.
Kenji HatanoDepartment of Clinical Data Science, Clinical Research and Education Promotion Division, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.
Mari S ObaDepartment of Clinical Data Science, Clinical Research and Education Promotion Division, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.
Kumiko IshiguroDepartment of Pediatrics, Tokyo Women's Medical University School of Medicine, Shinjuku-ku, Tokyo, Japan.
Minobu ShichijiDepartment of Pediatrics, Tokyo Women's Medical University School of Medicine, Shinjuku-ku, Tokyo, Japan.
Yuki KiharaDepartment of Pediatrics, Tokyo Women's Medical University School of Medicine, Shinjuku-ku, Tokyo, Japan.
Yasuhiro TakeshimaDepartment of Pediatrics, Hyogo Medical University, Nishinomiya, Hyogo, Japan.
Mariko Taniguchi-IkedaDepartment of Clinical Genetics, Fujita Health University Hospital, Toyoake, Aichi, Japan.
Ayako HattoriDepartment of Pediatrics, Nagoya City University East Medical Ceter, Chikusa-ku, Nagoya, Japan.
Yuko Shimizu-MotohashiDepartment of Child Neurology, National Center Hospital National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.
Hiroyuki AwanoDepartment of Clinical Genetics, Tottori University Hospital, Yonago, Tottori, Japan.
Ryosuke BoDepartment of Pediatrics, Kobe University Graduate School of Medicine, Chuo-ku, Kobe, Japan.
Satoru NagataDepartment of Pediatrics, Tokyo Women's Medical University School of Medicine, Shinjuku-ku, Tokyo, Japan.
Keiko IshigakiDepartment of Pediatrics, Tokyo Women's Medical University School of Medicine, Shinjuku-ku, Tokyo, Japan.ORCID https://orcid.org/0000-0001-8435-7418

Funding

Japan Agency for Medical Research and Development 23ek0109611h0002Japan Agency for Medical Research and Development 25ek0109611h0004
6 · The paper itself

Abstract

aimThis study aimed to evaluate the efficacy and safety of steroid therapy in patients with Fukuyama congenital muscular dystrophy (FCMD).

methodsThis is a noncontrolled, nonblinded, multicenter collaborative phase 2 trial in patients with a definite diagnosis of 3-kb insertion mutation homozygous or compound heterozygous FCMD based on genetic testing. The first part of the study evaluates efficacy; patients with homozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 24 weeks. The second part of the study evaluates safety; patients with homozygous and heterozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 48 weeks. Homozygous patients will be evaluated in the first part of the study for up to 24 weeks after prednisolone administration, after which they will continue receiving prednisolone in the second part for an additional 24 weeks. The primary endpoints were the changes in motor function evaluated using the gross motor function measure after treatment with prednisolone in the first part and the safety profiles based on the results of physical examination, vital signs, 12-lead electrocardiography (ECG), echocardiography, ophthalmic testing, SpO DISCUSSION: Based on previous clinical research, prednisolone shows great potential as a therapeutic drug in patients with FCMD. To achieve this goal, we planned an investigator-initiated trial to confirm the effectiveness and safety of prednisolone.

Indexed as

GlucocorticoidsPrednisoloneWalker-Warburg SyndromeAdolescentAdultChildChild, PreschoolClinical Trials, Phase II as TopicFemaleHumansMaleMulticenter Studies as TopicTreatment OutcomeYoung AdultGlucocorticoidsPrednisolonedrug repositioningFukuyama congenital muscular dystrophyinvestigator‐initiated clinical trialprednisolonesteroid therapy

Identifiers

PMID40814256
PMCPMC12355001

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.