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Pract."],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Corticobasal syndrome (CBS) is a rare progressive neurodegenerative disorder, with no disease-modifying treatments currently available. The most common underlying pathology is a 4-repeat tauopathy. Neurofilament light chain (NfL) is a biomarker of neuronal damage and has shown potential as a measure of disease progression. Glycerol phenylbutyrate (GPB), a prodrug of phenylbutyric acid, has demonstrated potential neuroprotective properties in preclinical studies on tauopathies. This phase II clinical trial will investigate the effects of GPB on NfL levels in CBS patients. The primary objective is to assess the efficacy of GPB in reducing NfL levels over 26\u00a0weeks compared to placebo as well as safety and tolerability of GPB. Secondary objectives include evaluating changes in clinical scales.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods and analysis<\/jats:title>\n                    <jats:p>This is an investigator-initiated double-blind, randomized, placebo-controlled, parallel-group phase II clinical trial, performed in two German university hospitals. A total of 32 patients with CBS will be enrolled and randomized to receive either GPB or placebo. The primary outcome is the change in NfL levels between baseline and 26\u00a0weeks as well as safety and tolerability of GPB. Secondary outcomes are changes in clinical scores. Exploratory analyses involve pharmacokinetics, changes in the metabolomic, proteomic and lipidomic profiles and imaging outcomes, such as MRI and microglia-PET.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Ethics and dissemination<\/jats:title>\n                    <jats:p>The study protocol has been approved by the lead ethics committee at LMU Munich and conforms to the ethical principles outlined in the Declaration of Helsinki and Good Clinical Practice (GCP) guidelines.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Perspectives<\/jats:title>\n                    <jats:p>If successful, this clinical trial could identify a novel therapeutic approach for slowing disease progression in CBS, contributing to a broader understanding of GPB\u2019s therapeutic potential.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Clinical trial registration<\/jats:title>\n                    <jats:p>The clinical trial has been registered in ClinicalTrials.gov (NCT05983588) and due to Transition in the Clinical Trials Information System (CTIS; EUCT No. 2024-516897-31-00, date of transition: 2024-09-26).<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s42466-026-00504-5","type":"journal-article","created":{"date-parts":[[2026,7,3]],"date-time":"2026-07-03T14:44:16Z","timestamp":1783089856000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Study protocol: double-blind, randomized, prospective, placebo controlled parallel group phase II study to investigate the effect of glycerol phenylbutyrate (GPB) on neurofilament light chain (NfL) levels in patients with corticobasal syndrome (CBS)"],"prefix":"10.1186","volume":"8","author":[{"given":"Carla","family":"Palleis","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Endy","family":"Weidinger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"Maa\u00df","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie","family":"Linke","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthias","family":"Brendel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophia","family":"St\u00f6cklein","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amani","family":"Al Tawil","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrich","family":"Mansmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Teupser","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tobias","family":"Borst","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ralph","family":"Heimke-Brinck","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernhard","family":"Hemmer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"G\u00fcnter U.","family":"H\u00f6glinger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helen","family":"Bidner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mikael","family":"Simons","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannes","family":"Levin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,7,3]]},"reference":[{"key":"504_CR1","doi-asserted-by":"publisher","first-page":"496","DOI":"10.1212\/WNL.0b013e31827f0fd1","volume":"80","author":"MJ Armstrong","year":"2013","unstructured":"Armstrong, M. 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A patient can only be included in the clinical trial, if she\/he provides written consent after being informed by a designated investigator (orally and in writing) about the nature, significance and scope of the clinical trial in an appropriate and understandable way. The investigator must fully explain the purpose of the clinical trial to the patient prior to entering the patient into the clinical trial. The investigator is responsible for obtaining written informed consent from each subject to be included. Patients have the right to withdraw study participation at any time without any reason given. The findings of this clinical trial will be disseminated at several levels including the publication of scientific papers in international peer-reviewed journals and presentation in relevant national and international conferences. The main conclusions of the project will be disseminated through press releases to the media, fostering a connection between science and society.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent of publication"}},{"value":"CP, MS and JL are inventors in a patent \u201cOral Phenylbutyrate for Treatment of Human 4-Repeat Tauopathies\u201d (PCT\/EP2024\/053388) filed by LMU Munich. CP reports author fees from Thieme medical publishers and W. Kohlhammer GmbH medical publishers. MB has received speaker honoraria from Roche, GE Healthcare, and Life Molecular Imaging; has advised Life Molecular Imaging; and is currently on the advisory board of MIAC. JL reports speaker fees from Bayer Vital, Biogen, EISAI, TEVA, Zambon, Esteve, Merck and Roche, consulting fees from Axon Neuroscience, EISAI and Biogen, author fees from Thieme medical publishers and W. Kohlhammer GmbH medical publishers. In addition, he reports compensation for serving as chief medical officer for MODAG GmbH, is beneficiary of the phantom share program of MODAG GmbH and is inventor in a patent \u201cPharmaceutical Composition and Methods of Use\u201d (EP 22159408.8) filed by MODAG GmbH, all activities outside the, submitted work.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"54"}}