Evidence map›Paper›PMID 31957953›Full record

Trial reportAnnals of clinical and translational neurology2020

Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy.

Hirofumi Komaki, Yoshihiro Maegaki, Tsuyoshi Matsumura, Kazuhiro Shiraishi, Hiroyuki Awano, Akinori Nakamura, Satoru Kinoshita, Katsuhisa Ogata, Keiko Ishigaki, Shinji Saitoh and 6 more

Open access · goldAbstract readClinical Trial, Phase IIRandomized Controlled Trial
In one paragraph

Trial report in Annals of clinical and translational neurology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
2.2field-weighted citation impact, top 12% of its field
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

14 citing papers in PubMed, 33 citations in OpenAlex.

  1. Review
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  4. Urinary prostaglandin DScientific reports · 2025
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  14. Current Pharmacological Strategies for Duchenne Muscular Dystrophy.Frontiers in cell and developmental biology · 2021
    Review
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

16 authors at 10 institutions in 1 country.

Hirofumi KomakiDepartment of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.ORCID 0000-0002-0659-1417
Yoshihiro MaegakiDivision of Child Neurology, Department of Brain and Neurosciences, Faculty of Medicine, Tottori University, Tottori, Japan.
Tsuyoshi MatsumuraDepartment of Neurology, National Hospital Organization Osaka Toneyama Medical Center, Osaka, Japan.
Kazuhiro ShiraishiDepartment of Pediatrics, National Hospital Organization Utano National Hospital, Kyoto, Japan.
Hiroyuki AwanoDepartment of Pediatrics, Kobe University Graduate School of Medicine, Hyogo, Japan.
Akinori NakamuraThird Department of Medicine, Shinshu University School of Medicine, Nagano, Japan.
Satoru KinoshitaDepartment of Pediatrics, National Hospital Organization Niigata National Hospital, Niigata, Japan.
Katsuhisa OgataDepartment of Neurology, National Hospital Organization Higashisaitama National Hospital, Saitama, Japan.
Keiko IshigakiDepartment of Pediatrics, Tokyo Women's Medical University, Tokyo, Japan.
Shinji SaitohDepartment of Pediatrics and Neonatology, Nagoya City University Graduate School of Medical Sciences, Aichi, Japan.
Michinori FunatoDepartment of Pediatrics, National Hospital Organization Nagara Medical Center, Gifu, Japan.
Satoshi KuruDepartment of Neurology, National Hospital Organization Suzuka National Hospital, Mie, Japan.
Takahiro NakayamaDepartment of Neurology, Division of Neuromuscular diseases, Yokohama Rosai Hospital, Kanagawa, Japan.
Yasuyuki IwataDepartment of Rehabilitation, National Center of Neurology and Psychiatry, Tokyo, Japan.
Hiroyuki YajimaDepartment of Rehabilitation, National Center of Neurology and Psychiatry, Tokyo, Japan.
Shin'ichi TakedaNational Center of Neurology and Psychiatry, Tokyo, Japan.
National Center of Neurology and Psychiatry · JPNational Hospital Organization · JPKobe University · JPNagara Medical Center · JPNagoya City University · JPShinshu University · JPTokyo Women's Medical University · JPToneyama National Hospital · JPTottori University · JPYokohama Rosai Hospital · JP

Funding

Taiho Pharmaceutical
6 · The paper itself

Abstract

objectiveDuchenne muscular dystrophy (DMD) is a progressive muscular disease characterized by chronic cycles of inflammatory and necrotic processes. Prostaglandin D

methodsPatients were randomized 1:1:1 to receive low-dose TAS-205 (6.67-13.33 mg/kg/dose), high-dose TAS-205 (13.33-26.67 mg/kg/dose), or placebo. The primary endpoint was the change from baseline in a 6-minute walk distance (6MWD) at Week 24.

resultsThirty-six patients were enrolled, of whom 35 patients were analysed for safety. The mean (standard error) changes from baseline to Week 24 in 6MWD were -17.0 (17.6) m in the placebo group (n = 10), -3.5 (20.3) m in the TAS-205 low-dose group (n = 11), and -7.5 (11.2) m in the TAS-205 high-dose group (n = 11). The mean (95% confidence interval) difference from the placebo group was 13.5 (-43.3 to 70.2) m in the TAS-205 low-dose group and 9.5 (-33.3 to 52.4) m in the TAS-205 high-dose group. No obvious differences were observed in the incidences of adverse events between treatment groups. No adverse drug reactions specific to TAS-205 treatment were observed.

interpretationThe HPGDS inhibitor TAS-205 showed a favorable safety profile in DMD patients. Further research is required to examine the effectiveness of TAS-205 in a larger trial.

Indexed as

ChildChild, PreschoolEnzyme InhibitorsHumansIntramolecular OxidoreductasesMaleMorpholinesMuscular Dystrophy, DuchenneOutcome Assessment, Health CarePiperidinesPyrrolesEnzyme InhibitorsHPGDS protein, humanIntramolecular OxidoreductasesMorpholinesPiperidinesPyrrolesTAS-205

Identifiers

PMID31957953
PMCPMC7034509
OpenAlexW3003031855

What OpenQuestion holds

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LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.