Phase 2a/b randomised placebo-controlled dose-escalation trial of triheptanoin for ataxia-telangiectasia: treating mitochondrial dysfunction with anaplerosis.
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| Title: | Phase 2a/b randomised placebo-controlled dose-escalation trial of triheptanoin for ataxia-telangiectasia: treating mitochondrial dysfunction with anaplerosis. |
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| Authors: | Lynch M; Department of Metabolic Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Australian Institute for Bioengineering and Nanotechnology, University of Queensland, Saint Lucia Brisbane, QLD, 4067, Australia., Manoy S; Department of Metabolic Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia., Sly PD; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia., Wainwright CE; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Respiratory and Sleep Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Wolvetang E; Australian Institute for Bioengineering and Nanotechnology, University of Queensland, Saint Lucia Brisbane, QLD, 4067, Australia., Feenstra JE; Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Adult Respiratory and Sleep Medicine, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia., Dowling J; Australian e-Health Research Centre, CSIRO, Herston, Brisbane, QLD, 4006, Australia., Ware RS; School of Medicine and Dentistry, Griffith University, Gold Coast, 4222, Australia; Griffith Biostatistics Unit, Griffith University, Nathan Campus, Brisbane, QLD, 4111, Australia., Patel MS; Griffith Biostatistics Unit, Griffith University, Nathan Campus, Brisbane, QLD, 4111, Australia., Hermith-Ramirez D; Griffith Biostatistics Unit, Griffith University, Nathan Campus, Brisbane, QLD, 4111, Australia., Vogel A; School of Health Sciences, The University of Melbourne, Parkville, Melbourne, 3010, Australia; Redenlab Inc., Melbourne 3000, Australia., Preece K; Department of Immunology and Allergy, John Hunter Children's Hospital, New Lambton Heights, Newcastle, NSW, 2305, Australia., Zappala T; Department of Paediatrics and Dermatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Dai S; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Ophthalmology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Webber A; Department of Ophthalmology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Yeo A; University of Queensland Centre for Clinical Research, Royal Brisbane and Women's Hospital, Herston Brisbane, QLD, 4029, Australia., Subramanian G; Department of Gastroenterology and Hepatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Rao G; Human Immune Disorders Laboratory, Garvan Institute of Medical Research, NSW, 2010, Australia., Ma CS; Human Immune Disorders Laboratory, Garvan Institute of Medical Research, NSW, 2010, Australia; School of Clinical Medicine, Faculty of Medicine and Health, UNSW Sydney, NSW, Australia., Jose S; University of Queensland Centre for Clinical Research, Royal Brisbane and Women's Hospital, Herston Brisbane, QLD, 4029, Australia., Gatei M; Department of Gastroenterology and Hepatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Xin B; Australian e-Health Research Centre, CSIRO, Herston, Brisbane, QLD, 4006, Australia., Sandona N; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia., Lewindon P; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Gastroenterology and Hepatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Sinclair KG; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Neurosciences Department, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia., Nayler S; QIMR-Berghofer Research Institute, QLD, 4006, Australia., Lavin MF; University of Queensland Centre for Clinical Research, Royal Brisbane and Women's Hospital, Herston Brisbane, QLD, 4029, Australia., Coman DJ; Department of Metabolic Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; School of Medicine and Dentistry, Griffith University, Gold Coast, 4222, Australia; Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia. Electronic address: david.coman@health.qld.gov.au. |
| Source: | EBioMedicine [EBioMedicine] 2025 Aug; Vol. 118, pp. 105840. Date of Electronic Publication: 2025 Jul 04. |
| Publication Type: | Journal Article; Clinical Trial, Phase II; Randomized Controlled Trial |
| Journal Info: | Publisher: Elsevier B.V Country of Publication: Netherlands NLM ID: 101647039 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2352-3964 (Electronic) Linking ISSN: 23523964 NLM ISO Abbreviation: EBioMedicine Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 40616902 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Phase 2a/b randomised placebo-controlled dose-escalation trial of triheptanoin for ataxia-telangiectasia: treating mitochondrial dysfunction with anaplerosis. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Lynch+M%22">Lynch M</searchLink>; Department of Metabolic Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Australian Institute for Bioengineering and Nanotechnology, University of Queensland, Saint Lucia Brisbane, QLD, 4067, Australia.<br /><searchLink fieldCode="AU" term="%22Manoy+S%22">Manoy S</searchLink>; Department of Metabolic Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Sly+PD%22">Sly PD</searchLink>; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Wainwright+CE%22">Wainwright CE</searchLink>; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Respiratory and Sleep Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Wolvetang+E%22">Wolvetang E</searchLink>; Australian Institute for Bioengineering and Nanotechnology, University of Queensland, Saint Lucia Brisbane, QLD, 4067, Australia.<br /><searchLink fieldCode="AU" term="%22Feenstra+JE%22">Feenstra JE</searchLink>; Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Adult Respiratory and Sleep Medicine, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia.<br /><searchLink fieldCode="AU" term="%22Dowling+J%22">Dowling J</searchLink>; Australian e-Health Research Centre, CSIRO, Herston, Brisbane, QLD, 4006, Australia.<br /><searchLink fieldCode="AU" term="%22Ware+RS%22">Ware RS</searchLink>; School of Medicine and Dentistry, Griffith University, Gold Coast, 4222, Australia; Griffith Biostatistics Unit, Griffith University, Nathan Campus, Brisbane, QLD, 4111, Australia.<br /><searchLink fieldCode="AU" term="%22Patel+MS%22">Patel MS</searchLink>; Griffith Biostatistics Unit, Griffith University, Nathan Campus, Brisbane, QLD, 4111, Australia.<br /><searchLink fieldCode="AU" term="%22Hermith-Ramirez+D%22">Hermith-Ramirez D</searchLink>; Griffith Biostatistics Unit, Griffith University, Nathan Campus, Brisbane, QLD, 4111, Australia.<br /><searchLink fieldCode="AU" term="%22Vogel+A%22">Vogel A</searchLink>; School of Health Sciences, The University of Melbourne, Parkville, Melbourne, 3010, Australia; Redenlab Inc., Melbourne 3000, Australia.<br /><searchLink fieldCode="AU" term="%22Preece+K%22">Preece K</searchLink>; Department of Immunology and Allergy, John Hunter Children's Hospital, New Lambton Heights, Newcastle, NSW, 2305, Australia.<br /><searchLink fieldCode="AU" term="%22Zappala+T%22">Zappala T</searchLink>; Department of Paediatrics and Dermatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Dai+S%22">Dai S</searchLink>; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Ophthalmology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Webber+A%22">Webber A</searchLink>; Department of Ophthalmology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Yeo+A%22">Yeo A</searchLink>; University of Queensland Centre for Clinical Research, Royal Brisbane and Women's Hospital, Herston Brisbane, QLD, 4029, Australia.<br /><searchLink fieldCode="AU" term="%22Subramanian+G%22">Subramanian G</searchLink>; Department of Gastroenterology and Hepatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Rao+G%22">Rao G</searchLink>; Human Immune Disorders Laboratory, Garvan Institute of Medical Research, NSW, 2010, Australia.<br /><searchLink fieldCode="AU" term="%22Ma+CS%22">Ma CS</searchLink>; Human Immune Disorders Laboratory, Garvan Institute of Medical Research, NSW, 2010, Australia; School of Clinical Medicine, Faculty of Medicine and Health, UNSW Sydney, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Jose+S%22">Jose S</searchLink>; University of Queensland Centre for Clinical Research, Royal Brisbane and Women's Hospital, Herston Brisbane, QLD, 4029, Australia.<br /><searchLink fieldCode="AU" term="%22Gatei+M%22">Gatei M</searchLink>; Department of Gastroenterology and Hepatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Xin+B%22">Xin B</searchLink>; Australian e-Health Research Centre, CSIRO, Herston, Brisbane, QLD, 4006, Australia.<br /><searchLink fieldCode="AU" term="%22Sandona+N%22">Sandona N</searchLink>; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia.<br /><searchLink fieldCode="AU" term="%22Lewindon+P%22">Lewindon P</searchLink>; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Department of Gastroenterology and Hepatology, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Sinclair+KG%22">Sinclair KG</searchLink>; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; Neurosciences Department, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia.<br /><searchLink fieldCode="AU" term="%22Nayler+S%22">Nayler S</searchLink>; QIMR-Berghofer Research Institute, QLD, 4006, Australia.<br /><searchLink fieldCode="AU" term="%22Lavin+MF%22">Lavin MF</searchLink>; University of Queensland Centre for Clinical Research, Royal Brisbane and Women's Hospital, Herston Brisbane, QLD, 4029, Australia.<br /><searchLink fieldCode="AU" term="%22Coman+DJ%22">Coman DJ</searchLink>; Department of Metabolic Medicine, Queensland Children's Hospital, South Brisbane, QLD, 4101, Australia; Department of Paediatrics, Wesley Medical Research, Auchenflower Brisbane, QLD, 4066, Australia; Child Health Research Centre, Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia; School of Medicine and Dentistry, Griffith University, Gold Coast, 4222, Australia; Faculty of Medicine, University of Queensland, South Brisbane, QLD, 4101, Australia. Electronic address: david.coman@health.qld.gov.au. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101647039%22">EBioMedicine</searchLink> [EBioMedicine] 2025 Aug; Vol. 118, pp. 105840. <i>Date of Electronic Publication: </i>2025 Jul 04. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article; Clinical Trial, Phase II; Randomized Controlled Trial – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier+B%2EV%22">Elsevier B.V </searchLink><i>Country of Publication: </i>Netherlands <i>NLM ID: </i>101647039 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2352-3964 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223523964%22">23523964 </searchLink><i>NLM ISO Abbreviation: </i>EBioMedicine <i>Subsets: </i>MEDLINE |
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| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1016/j.ebiom.2025.105840 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 105840 Titles: – TitleFull: Phase 2a/b randomised placebo-controlled dose-escalation trial of triheptanoin for ataxia-telangiectasia: treating mitochondrial dysfunction with anaplerosis. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Lynch M – PersonEntity: Name: NameFull: Manoy S – PersonEntity: Name: NameFull: Sly PD – PersonEntity: Name: NameFull: Wainwright CE – PersonEntity: Name: NameFull: Wolvetang E – PersonEntity: Name: NameFull: Feenstra JE – PersonEntity: Name: NameFull: Dowling J – PersonEntity: Name: NameFull: Ware RS – PersonEntity: Name: NameFull: Patel MS – PersonEntity: Name: NameFull: Hermith-Ramirez D – PersonEntity: Name: NameFull: Vogel A – PersonEntity: Name: NameFull: Preece K – PersonEntity: Name: NameFull: Zappala T – PersonEntity: Name: NameFull: Dai S – PersonEntity: Name: NameFull: Webber A – PersonEntity: Name: NameFull: Yeo A – PersonEntity: Name: NameFull: Subramanian G – PersonEntity: Name: NameFull: Rao G – PersonEntity: Name: NameFull: Ma CS – PersonEntity: Name: NameFull: Jose S – PersonEntity: Name: NameFull: Gatei M – PersonEntity: Name: NameFull: Xin B – PersonEntity: Name: NameFull: Sandona N – PersonEntity: Name: NameFull: Lewindon P – PersonEntity: Name: NameFull: Sinclair KG – PersonEntity: Name: NameFull: Nayler S – PersonEntity: Name: NameFull: Lavin MF – PersonEntity: Name: NameFull: Coman DJ IsPartOfRelationships: – BibEntity: Dates: – D: 01 M: 08 Text: 2025 Aug Type: published Y: 2025 Identifiers: – Type: issn-electronic Value: 2352-3964 Numbering: – Type: volume Value: 118 Titles: – TitleFull: EBioMedicine Type: main |
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