https://m2.mtmt.hu/ hun 2026-04-16 14:09 JournalArticle 34009554 VALIDATED true 0 false 2026-02-13T07:53:01.460+0000 2023-09-05T10:45:26.418+0000 2023-06-12T05:11:17.142+0000 true 10015544 Csajbók Edit /api/admin/10015544 Admin true 10015544 2023-11-10T22:47:41.422+0000 2023-09-05T10:45:26.428+0000 true 10080205 Szalóky-Siki Ágnes /api/admin/10080205 Admin true true false true 24 24 /api/publicationtype/24 PublicationType 1 true 24 JournalArticle true 10000359 Study Group true 24 24 /api/publicationtype/24 PublicationType 1 true 24 JournalArticle /api/subtype/10000359 Sokszerzős vagy csoportos szerzőségű szakcikk SubType 104 true 10000359 true 1 /api/category/1 Category true 1 Karaa, Amel Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy The MMPOWER-3 Randomized Clinical Trial. true true /api/journal/3355 REVIEWED true false 3355 false 3355 true 0028-3878 1526-632X Journal FOREIGN 101 3 e238 e252 e238 2023 Primary Mitochondrial Myopathies (PMMs) encompass a group of genetic disorders that impair mitochondrial oxidative phosphorylation, adversely impacting physical function, exercise capacity, and quality of life (QoL). Current PMM standards-of-care address symptoms, with limited clinical impact, constituting a significant therapeutic unmet need. We present data from MMPOWER-3, a pivotal, phase-3, randomized, double-blind, placebo-controlled clinical trial that evaluated the efficacy and safety of elamipretide in participants with genetically-confirmed PMM.Following screening, eligible participants were randomized 1:1 to receive either 24weeks of elamipretide 40mg/day or placebo subcutaneously. Primary efficacy endpoints included change from baseline to Week 24 on the distance walked on the 6-minute Walk Test (6MWT), and Total Fatigue on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA). Secondary endpoints included Most Bothersome Symptom Score on the PMMSA, NeuroQoL Fatigue Short Form scores, and the Patient- and Clinician-Global Impression of PMM Symptoms.Participants (N=218) were randomized (n=109 elamipretide; n=109 placebo). Mean age was 45.6 year (64% women; 94% white). The majority of participants (n=162 [74%]) had mitochondrial DNA (mtDNA) mutations, with the remainder having nuclear DNA (nDNA) defects. At screening, the most frequent bothersome PMM symptom on the PMMSA was tiredness during activities (28.9%). At baseline, mean distance walked on the 6MWT was 336.7±81.2 meters, mean score for Total Fatigue on the PMMSA was 10.6±2.5, and mean T-score for the Neuro-QoL Fatigue Short Form was 54.7±7.5. The study did not meet its primary endpoints assessing changes in the 6MWT and PMMSA Total Fatigue Score (TFS). Between the participants receiving elamipretide versus placebo, the difference in the Least Squares Mean (SE) from baseline to Week 24 on distance walked on the 6MWT was -3.2 (95% confidence interval,-18.7,12.3; p=0.69) meters and on the PMMSA Total Fatigue Score was -0.07 (95% confidence interval,-0.10,0.26; p=0.37). Elamipretide treatment was well-tolerated with most adverse events being mild to moderate in severity.Subcutaneous elamipretide treatment did not improve outcomes in the 6MWT and PMMSA TFS in patients with PMM. However, this phase-3 study demonstrated that subcutaneous elamipretide is well-tolerated.Trial registered with clinicaltrials.gov, Clinical Trials Identifier: NCT03323749; submitted on October 12, 2017;first patient enrolled October 9, 2017. https://clinicaltrials.gov/ct2/show/NCT03323749?term=elamipretide&draw=2&rank=9 CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that elamipretide does not improve the 6 minute walk test or fatigue at 24 weeks compared to placebo in patients with primary mitochondrial myopathy. true true 2023 true true true false true false GOLD 2026-02-13 true https://doi.org/10.1212/WNL.0000000000207402 51 0 51 42 0 51 51 47 49 51 51 42 42 42 9 0 51 42 51 42 0 1 1 Folyóiratcikk Journal Article 24 50 Könyvrészlet Chapter in Book 25 1 Könyv Book 23 0 Egyéb konferenciaközlemény Conference paper 31 0 Egyéb konferenciakötet Conference proceedings 32 0 Oltalmi formák Protection forms 26 0 Disszertáció Thesis 28 0 Egyéb Miscellaneous 29 0 Alkotás 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mtid="10002781"><a href="/gui2/?type=authors&mode=browse&sel=10002781" target="_blank">Molnar Maria Judit (<span class="authorship-author-name">Molnár Mária Judit</span> <span class="authorAux-mtmt"> Neurológia</span>) </a> </span> <span class="author-affil"><span title="Semmelweis Egyetem">SE</span>/<span title="Általános Orvostudományi Kar">AOK</span>/<span title="Intézet">I</span>/Genomikai Medicina és Ritka Betegségek Intézete</span> ;&nbsp;&nbsp;&nbsp; et al. (32) </div> <div class="autype autype-29"> Kollaborációs szervezet: <span class="author-affil">MMPOWER-3 Trial Investigators</span> </div> <div><a target="_blank" href="/api/publication/34009554?labelLang=hun">Összes szerző</a></div> </div> <div class="title"><a href="/gui2/?mode=browse&params=publication;34009554" target="_blank">Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy : The MMPOWER-3 Randomized Clinical Trial..</a></div> <div> <span class="journal-title">NEUROLOGY</span> <span class="journal-issn">(<a target="_blank" href="https://portal.issn.org/resource/ISSN/0028-3878">0028-3878</a> <a target="_blank" href="https://portal.issn.org/resource/ISSN/1526-632X">1526-632X</a>)</span>: <span class="journal-volume">101</span> <span class="journal-issue">3</span> <span class="page"> pp e238-e252 </span> <span class="year">(2023)</span> </div> <div class="pub-footer"> <span class="language" xmlns="http://www.w3.org/1999/html">Nyelv: Angol | </span> <span class="identifiers"> <span class="id identifier oa_GOLD" title=" Gold "> <a style="color:blue" title="10.1212/WNL.0000000000207402" target="_blank" href="https://doi.org/10.1212/WNL.0000000000207402"> DOI </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="001031737700012" target="_blank" href="https://www.webofscience.com/wos/woscc/full-record/001031737700012"> WoS </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="85165222625" target="_blank" href="http://www.scopus.com/record/display.url?origin=inward&eid=2-s2.0-85165222625"> Scopus </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="37268435" target="_blank" href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=37268435&dopt=Abstract"> PubMed </a> </span> </span> <OnlyViewableByAuthor><div class="ratings"> <div class="journal-subject">Folyóirat szakterülete: Scopus - Neurology (clinical)&nbsp;&nbsp;&nbsp;SJR indikátor:&nbsp;D1</div> </div></OnlyViewableByAuthor> <div class="publication-citation" style="margin-left: 0.5cm;"> <span title="Nyilvános idézőközlemények összesen, említések nélkül" class="citingPub-count">Nyilvános idéző összesen: 51</span> | Független: 42 | Függő: 9 | Nem jelölt: 0 | WoS jelölt: 47 | Scopus jelölt:&nbsp;49 | WoS/Scopus jelölt:&nbsp;51 | DOI jelölt:&nbsp;51 </div> <div class="publication-citation"> <a target="_blank" href="/api/publication?cond=citations.related;eq;34009554&sort=publishedYear,desc&sort=title"> Idézett közlemények száma: 1 </a> </div> <div class="mtid"><span class="long-pub-mtid">Közlemény: 34009554</span> | <span class="status-data status-VALIDATED"> Egyeztetett </span> Forrás Idéző | <span class="type-subtype">Folyóiratcikk ( Sokszerzős vagy csoportos szerzőségű szakcikk ) </span> | <span class="pub-category">Tudományos</span> | <span class="publication-sourceOfData">PubMed XML</span> </div> <div class="lastModified">Utolsó módosítás: 2023.09.05. 12:45 Szalóky-Siki Ágnes (SE_KK_Admin5_SZSA, admin) </div> </div></div>