Open-label pilot clinical trial of citicoline for fragile X-associated tremor/ataxia syndrome (FXTAS)
Fragile X-associated tremor/ataxia syndrome (FXTAS) is a late onset neurodegenerative disorder that is characterized by tremor, cerebellar ataxia, frequent falls, cognitive decline, and progressive loss of motor function. There are currently no approved treatments for this disorder. The purpose of t...
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| creator | Hall, Deborah A Robertson, Erin E Leehey, Maureen McAsey, Andrew Ouyang, Bichun Berry-Kravis, Elizabeth O'Keefe, Joan A |
| description | Fragile X-associated tremor/ataxia syndrome (FXTAS) is a late onset neurodegenerative disorder that is characterized by tremor, cerebellar ataxia, frequent falls, cognitive decline, and progressive loss of motor function. There are currently no approved treatments for this disorder. The purpose of this study was to determine if citicoline was safe for the treatment of tremor and balance abnormalities and to stabilize cognitive decline in patients with FXTAS. Ten participants with diagnosed FXTAS were administered 1000 mg of citicoline once daily for 12 months. Outcome measures and neurological examination were performed at baseline, 3 months, 6 months, and 12 months. The primary outcome was the FXTAS Rating Scale score. Secondary outcomes included change in a battery of neuropsychological tests, an instrumented Timed up and go test, computerized dynamic posturography, 9-hole pegboard test, and balance confidence and psychiatric symptom questionnaires. Safety was also evaluated. Citicoline treatment resulted in minimal adverse events in all but one subject over the course of the study. There was a significant improvement in the Beck Anxiety Inventory (p = 0.03) and the Stroop Color-Word test (p = 0.03), with all other measures remaining stable over the course of 12 months. This open-label pilot trial of citicoline for individuals with FXTAS showed that it is safe and well tolerated in this population. Registration: This trial was registered at ClinicalTrials.gov. Identifier: NCT0219710. |
| doi_str_mv | 10.1371/journal.pone.0225191 |
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There are currently no approved treatments for this disorder. The purpose of this study was to determine if citicoline was safe for the treatment of tremor and balance abnormalities and to stabilize cognitive decline in patients with FXTAS. Ten participants with diagnosed FXTAS were administered 1000 mg of citicoline once daily for 12 months. Outcome measures and neurological examination were performed at baseline, 3 months, 6 months, and 12 months. The primary outcome was the FXTAS Rating Scale score. Secondary outcomes included change in a battery of neuropsychological tests, an instrumented Timed up and go test, computerized dynamic posturography, 9-hole pegboard test, and balance confidence and psychiatric symptom questionnaires. Safety was also evaluated. Citicoline treatment resulted in minimal adverse events in all but one subject over the course of the study. There was a significant improvement in the Beck Anxiety Inventory (p = 0.03) and the Stroop Color-Word test (p = 0.03), with all other measures remaining stable over the course of 12 months. This open-label pilot trial of citicoline for individuals with FXTAS showed that it is safe and well tolerated in this population. Registration: This trial was registered at ClinicalTrials.gov. Identifier: NCT0219710.</description><identifier>ISSN: 1932-6203</identifier><identifier>EISSN: 1932-6203</identifier><identifier>DOI: 10.1371/journal.pone.0225191</identifier><identifier>PMID: 32053612</identifier><language>eng</language><publisher>United States: Public Library of Science</publisher><subject>Abnormalities ; Aged ; Alzheimer's disease ; Anxiety ; Ataxia ; Ataxia - diagnosis ; Ataxia - drug therapy ; Balance ; Biology and Life Sciences ; Cerebellar ataxia ; Cerebellum ; Citicoline ; Clinical trials ; Cognition - drug effects ; Cognitive ability ; Cytidine Diphosphate Choline - administration & dosage ; Cytidine Diphosphate Choline - adverse effects ; Female ; Fragile X syndrome ; Fragile X Syndrome - diagnosis ; Fragile X Syndrome - drug therapy ; Humans ; Intellectual disabilities ; Intelligence ; Laboratories ; Male ; Medicine and Health Sciences ; Middle Aged ; Motor ability ; Motor Activity - drug effects ; Neurodegenerative diseases ; Neuropsychological Tests ; Nootropic Agents - administration & dosage ; Nootropic Agents - adverse effects ; Pilot Projects ; Postural Balance - drug effects ; Research and Analysis Methods ; Severity of Illness Index ; Social Sciences ; Time and Motion Studies ; Time Factors ; Treatment Outcome ; Tremor ; Tremor - diagnosis ; Tremor - drug therapy ; Tremors ; Womens health</subject><ispartof>PloS one, 2020-02, Vol.15 (2), p.e0225191-e0225191</ispartof><rights>2020 Hall et al. This is an open access article distributed under the terms of the Creative Commons Attribution License: http://creativecommons.org/licenses/by/4.0/ (the “License”), which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><rights>2020 Hall et al 2020 Hall et al</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c526t-868c5e1ef4eca4588f06a4e4f00a618c3b69d677415569002e6c51956a08168c3</citedby><cites>FETCH-LOGICAL-c526t-868c5e1ef4eca4588f06a4e4f00a618c3b69d677415569002e6c51956a08168c3</cites><orcidid>0000-0003-2772-7851</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC7018079/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC7018079/$$EHTML$$P50$$Gpubmedcentral$$Hfree_for_read</linktohtml><link.rule.ids>230,314,727,780,784,864,885,2102,2928,23866,27924,27925,53791,53793,79600,79601</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32053612$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><contributor>Bayer, Antony</contributor><creatorcontrib>Hall, Deborah A</creatorcontrib><creatorcontrib>Robertson, Erin E</creatorcontrib><creatorcontrib>Leehey, Maureen</creatorcontrib><creatorcontrib>McAsey, Andrew</creatorcontrib><creatorcontrib>Ouyang, Bichun</creatorcontrib><creatorcontrib>Berry-Kravis, Elizabeth</creatorcontrib><creatorcontrib>O'Keefe, Joan A</creatorcontrib><title>Open-label pilot clinical trial of citicoline for fragile X-associated tremor/ataxia syndrome (FXTAS)</title><title>PloS one</title><addtitle>PLoS One</addtitle><description>Fragile X-associated tremor/ataxia syndrome (FXTAS) is a late onset neurodegenerative disorder that is characterized by tremor, cerebellar ataxia, frequent falls, cognitive decline, and progressive loss of motor function. There are currently no approved treatments for this disorder. The purpose of this study was to determine if citicoline was safe for the treatment of tremor and balance abnormalities and to stabilize cognitive decline in patients with FXTAS. Ten participants with diagnosed FXTAS were administered 1000 mg of citicoline once daily for 12 months. Outcome measures and neurological examination were performed at baseline, 3 months, 6 months, and 12 months. The primary outcome was the FXTAS Rating Scale score. Secondary outcomes included change in a battery of neuropsychological tests, an instrumented Timed up and go test, computerized dynamic posturography, 9-hole pegboard test, and balance confidence and psychiatric symptom questionnaires. Safety was also evaluated. Citicoline treatment resulted in minimal adverse events in all but one subject over the course of the study. There was a significant improvement in the Beck Anxiety Inventory (p = 0.03) and the Stroop Color-Word test (p = 0.03), with all other measures remaining stable over the course of 12 months. This open-label pilot trial of citicoline for individuals with FXTAS showed that it is safe and well tolerated in this population. Registration: This trial was registered at ClinicalTrials.gov. Identifier: NCT0219710.</description><subject>Abnormalities</subject><subject>Aged</subject><subject>Alzheimer's disease</subject><subject>Anxiety</subject><subject>Ataxia</subject><subject>Ataxia - diagnosis</subject><subject>Ataxia - drug therapy</subject><subject>Balance</subject><subject>Biology and Life Sciences</subject><subject>Cerebellar ataxia</subject><subject>Cerebellum</subject><subject>Citicoline</subject><subject>Clinical trials</subject><subject>Cognition - drug effects</subject><subject>Cognitive ability</subject><subject>Cytidine Diphosphate Choline - administration & dosage</subject><subject>Cytidine Diphosphate Choline - adverse effects</subject><subject>Female</subject><subject>Fragile X syndrome</subject><subject>Fragile X Syndrome - diagnosis</subject><subject>Fragile X Syndrome - drug therapy</subject><subject>Humans</subject><subject>Intellectual disabilities</subject><subject>Intelligence</subject><subject>Laboratories</subject><subject>Male</subject><subject>Medicine and Health Sciences</subject><subject>Middle Aged</subject><subject>Motor ability</subject><subject>Motor Activity - 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diagnosis</topic><topic>Ataxia - drug therapy</topic><topic>Balance</topic><topic>Biology and Life Sciences</topic><topic>Cerebellar ataxia</topic><topic>Cerebellum</topic><topic>Citicoline</topic><topic>Clinical trials</topic><topic>Cognition - drug effects</topic><topic>Cognitive ability</topic><topic>Cytidine Diphosphate Choline - administration & dosage</topic><topic>Cytidine Diphosphate Choline - adverse effects</topic><topic>Female</topic><topic>Fragile X syndrome</topic><topic>Fragile X Syndrome - diagnosis</topic><topic>Fragile X Syndrome - drug therapy</topic><topic>Humans</topic><topic>Intellectual disabilities</topic><topic>Intelligence</topic><topic>Laboratories</topic><topic>Male</topic><topic>Medicine and Health Sciences</topic><topic>Middle Aged</topic><topic>Motor ability</topic><topic>Motor Activity - drug effects</topic><topic>Neurodegenerative diseases</topic><topic>Neuropsychological Tests</topic><topic>Nootropic Agents - administration & dosage</topic><topic>Nootropic Agents - 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There are currently no approved treatments for this disorder. The purpose of this study was to determine if citicoline was safe for the treatment of tremor and balance abnormalities and to stabilize cognitive decline in patients with FXTAS. Ten participants with diagnosed FXTAS were administered 1000 mg of citicoline once daily for 12 months. Outcome measures and neurological examination were performed at baseline, 3 months, 6 months, and 12 months. The primary outcome was the FXTAS Rating Scale score. Secondary outcomes included change in a battery of neuropsychological tests, an instrumented Timed up and go test, computerized dynamic posturography, 9-hole pegboard test, and balance confidence and psychiatric symptom questionnaires. Safety was also evaluated. Citicoline treatment resulted in minimal adverse events in all but one subject over the course of the study. There was a significant improvement in the Beck Anxiety Inventory (p = 0.03) and the Stroop Color-Word test (p = 0.03), with all other measures remaining stable over the course of 12 months. This open-label pilot trial of citicoline for individuals with FXTAS showed that it is safe and well tolerated in this population. Registration: This trial was registered at ClinicalTrials.gov. Identifier: NCT0219710.</abstract><cop>United States</cop><pub>Public Library of Science</pub><pmid>32053612</pmid><doi>10.1371/journal.pone.0225191</doi><orcidid>https://orcid.org/0000-0003-2772-7851</orcidid><oa>free_for_read</oa></addata></record> |
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| ispartof | PloS one, 2020-02, Vol.15 (2), p.e0225191-e0225191 |
| issn | 1932-6203 1932-6203 |
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| source | MEDLINE; DOAJ Directory of Open Access Journals; Public Library of Science (PLoS) Journals Open Access; EZB-FREE-00999 freely available EZB journals; PubMed Central; Free Full-Text Journals in Chemistry |
| subjects | Abnormalities Aged Alzheimer's disease Anxiety Ataxia Ataxia - diagnosis Ataxia - drug therapy Balance Biology and Life Sciences Cerebellar ataxia Cerebellum Citicoline Clinical trials Cognition - drug effects Cognitive ability Cytidine Diphosphate Choline - administration & dosage Cytidine Diphosphate Choline - adverse effects Female Fragile X syndrome Fragile X Syndrome - diagnosis Fragile X Syndrome - drug therapy Humans Intellectual disabilities Intelligence Laboratories Male Medicine and Health Sciences Middle Aged Motor ability Motor Activity - drug effects Neurodegenerative diseases Neuropsychological Tests Nootropic Agents - administration & dosage Nootropic Agents - adverse effects Pilot Projects Postural Balance - drug effects Research and Analysis Methods Severity of Illness Index Social Sciences Time and Motion Studies Time Factors Treatment Outcome Tremor Tremor - diagnosis Tremor - drug therapy Tremors Womens health |
| title | Open-label pilot clinical trial of citicoline for fragile X-associated tremor/ataxia syndrome (FXTAS) |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-25T03%3A43%3A52IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_plos_&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Open-label%20pilot%20clinical%20trial%20of%20citicoline%20for%20fragile%20X-associated%20tremor/ataxia%20syndrome%20(FXTAS)&rft.jtitle=PloS%20one&rft.au=Hall,%20Deborah%20A&rft.date=2020-02-13&rft.volume=15&rft.issue=2&rft.spage=e0225191&rft.epage=e0225191&rft.pages=e0225191-e0225191&rft.issn=1932-6203&rft.eissn=1932-6203&rft_id=info:doi/10.1371/journal.pone.0225191&rft_dat=%3Cproquest_plos_%3E2354742673%3C/proquest_plos_%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=2354742673&rft_id=info:pmid/32053612&rft_doaj_id=oai_doaj_org_article_41d8f9fa71a9492391d4154b42ce5a9e&rfr_iscdi=true |