Evidence map›Paper›PMID 36410013›Full record

ArticlePloS one2022

A natural history study to track brain and spinal cord changes in individuals with Friedreich's ataxia: TRACK-FA study protocol.

Nellie Georgiou-Karistianis, Louise A Corben, Kathrin Reetz, Isaac M Adanyeguh, Manuela Corti, Dinesh K Deelchand, Martin B Delatycki, Imis Dogan, Rebecca Evans, Jennifer Farmer and 27 more

Erratum issued Registry-linked trialOpen access · goldAbstract readClinical Trial Protocol
In one paragraph

Article in PloS one, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT04349514 (A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia), which is not on this map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
1.7field-weighted citation impact, top 16% 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.

NCT04349514 active not recruitingnot on this map

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

TypeobservationalSponsorMonash UniversityRan2021 to 2025Enrolled300ConditionsFriedreich AtaxiaArmsNatural history
3 · Its place in the literature

Who cites it

15 citing papers in PubMed, 18 citations in OpenAlex.

  1. Review
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  4. Observational
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  13. Reproducible Spinal Cord Quantitative MRI Analysis with the Spinal Cord Toolbox.Magnetic resonance in medical sciences : MRMS : an official journal of Japan Society of Magnetic Resonance in Medicine · 2024
    Review
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

37 authors at 14 institutions in 6 countries.

Nellie Georgiou-KaristianisSchool of Psychological Sciences, The Turner Institute for Brain and Mental Health, Monash University, Clayton, Victoria, Australia.ORCID 0000-0003-0718-6760
Louise A CorbenBruce Lefroy Centre for Genetic Health Research, Murdoch Children's Research Institute, Parkville, Victoria, Australia.
Kathrin ReetzDepartment of Neurology, RWTH Aachen University, Aachen, Germany.
Isaac M AdanyeguhCenter for Magnetic Resonance Research and Department of Radiology, University of Minnesota, Minneapolis, Minnesota, United States of America.
Manuela CortiPowell Gene Therapy Centre, University of Florida, Gainesville, Florida, United States of America.
Dinesh K DeelchandCenter for Magnetic Resonance Research and Department of Radiology, University of Minnesota, Minneapolis, Minnesota, United States of America.
Martin B DelatyckiSchool of Psychological Sciences, The Turner Institute for Brain and Mental Health, Monash University, Clayton, Victoria, Australia.
Imis DoganDepartment of Neurology, RWTH Aachen University, Aachen, Germany.
Rebecca EvansTakeda Pharmaceutical Company Ltd, Cambridge, Massachusetts, United States of America.
Jennifer FarmerFriedreich's Ataxia Research Alliance (FARA), Downingtown, Pennsylvania, United States of America.
Marcondes C FrançaDepartment of Neurology, University of Campinas, Campinas, Sao Paulo, Brazil.
William GaetzDepartment of Radiology, Lurie Family Foundations MEG Imaging Center, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America.
Ian H HardingDepartment of Neuroscience, Central Clinical School, Monash University, Melbourne, Australia.
Karen S HarrisSchool of Psychological Sciences, The Turner Institute for Brain and Mental Health, Monash University, Clayton, Victoria, Australia.
Steven HerschNeurology Business Group, Eisai Inc., Nutley, New Jersey, United States of America.
Richard JoulesIXICO plc, London, England.
James J JoersCenter for Magnetic Resonance Research and Department of Radiology, University of Minnesota, Minneapolis, Minnesota, United States of America.ORCID 0000-0002-8444-2185
Michelle L KrishnanTranslational Medicine, Novartis Institutes for Biomedical Research, Cambridge, MA, United States of America.
Michelle LaxIXICO plc, London, England.
Eric F LockDivision of Biostatistics, School of Public Health, University of Minnesota, Minneapolis, MN, United States of America.
David LynchDepartment of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America.
Thomas MareciDepartment of Biochemistry and Molecular Biology, University of Florida, Gainesville, FL, United States of America.
Sahan Muthuhetti GamageSchool of Psychological Sciences, The Turner Institute for Brain and Mental Health, Monash University, Clayton, Victoria, Australia.
Massimo PandolfoDepartment of Neurology and Neurosurgery, McGill University, Montreal, Canada.
Marina PapoutsiIXICO plc, London, England.
Thiago J R RezendeDepartment of Neurology, University of Campinas, Campinas, Sao Paulo, Brazil.
Timothy P L RobertsDepartment of Radiology, Lurie Family Foundations MEG Imaging Center, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America.
Jens T RosenbergMcKnight Brain Institute, Department of Neurology, University of Florida, Gainesville, Florida, United States of America.ORCID 0000-0001-5957-0313
Sandro RomanzettiDepartment of Neurology, RWTH Aachen University, Aachen, Germany.
Jörg B SchulzDepartment of Neurology, RWTH Aachen University, Aachen, Germany.
Traci SchillingPTC Therapeutics, Inc, South Plainfield, New Jersey, United States of America.
Adam J SchwarzTakeda Pharmaceutical Company Ltd, Cambridge, Massachusetts, United States of America.
Sub SubramonyMcKnight Brain Institute, Department of Neurology, University of Florida, Gainesville, Florida, United States of America.
Bert YaoPTC Therapeutics, Inc, South Plainfield, New Jersey, United States of America.
Stephen ZichaTakeda Pharmaceutical Company Ltd, Cambridge, Massachusetts, United States of America.
Christophe LengletCenter for Magnetic Resonance Research and Department of Radiology, University of Minnesota, Minneapolis, Minnesota, United States of America.
Pierre-Gilles HenryCenter for Magnetic Resonance Research and Department of Radiology, University of Minnesota, Minneapolis, Minnesota, United States of America.
University of Minnesota · USForschungszentrum Jülich · DEMonash University · AUUniversity of Florida · USChildren's Hospital of Philadelphia · USIxico (United Kingdom) · GBTakeda (United States) · USPTC Therapeutics (United States) · USThe University of Melbourne · AUUniversidade Estadual de Campinas (UNICAMP) · BREisai (United States) · USFriedreich's Ataxia Research Alliance · USMcGill University · CANovartis (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionDrug development for neurodegenerative diseases such as Friedreich's ataxia (FRDA) is limited by a lack of validated, sensitive biomarkers of pharmacodynamic response in affected tissue and disease progression. Studies employing neuroimaging measures to track FRDA have thus far been limited by their small sample sizes and limited follow up. TRACK-FA, a longitudinal, multi-site, and multi-modal neuroimaging natural history study, aims to address these shortcomings by enabling better understanding of underlying pathology and identifying sensitive, clinical trial ready, neuroimaging biomarkers for FRDA.

methods200 individuals with FRDA and 104 control participants will be recruited across seven international study sites. Inclusion criteria for participants with genetically confirmed FRDA involves, age of disease onset ≤ 25 years, Friedreich's Ataxia Rating Scale (FARS) functional staging score of ≤ 5, and a total modified FARS (mFARS) score of ≤ 65 upon enrolment. The control cohort is matched to the FRDA cohort for age, sex, handedness, and years of education. Participants will be evaluated at three study visits over two years. Each visit comprises of a harmonized multimodal Magnetic Resonance Imaging (MRI) and Spectroscopy (MRS) scan of the brain and spinal cord; clinical, cognitive, mood and speech assessments and collection of a blood sample. Primary outcome measures, informed by previous neuroimaging studies, include measures of: spinal cord and brain morphometry, spinal cord and brain microstructure (measured using diffusion MRI), brain iron accumulation (using Quantitative Susceptibility Mapping) and spinal cord biochemistry (using MRS). Secondary and exploratory outcome measures include clinical, cognitive assessments and blood biomarkers. DISCUSSION: Prioritising immediate areas of need, TRACK-FA aims to deliver a set of sensitive, clinical trial-ready neuroimaging biomarkers to accelerate drug discovery efforts and better understand disease trajectory. Once validated, these potential pharmacodynamic biomarkers can be used to measure the efficacy of new therapeutics in forestalling disease progression. CLINICAL

trial registrationClinicalTrails.gov Identifier: NCT04349514.

Indexed as

Friedreich AtaxiaAdultBiomarkersBrainClinical Trials as TopicDisease ProgressionHumansMagnetic Resonance SpectroscopyBiomarkers

Identifiers

PMID36410013
PMCPMC9678384
OpenAlexW4309525110

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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.