Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses
NCT ID: NCT04808297
Last Updated: 2022-04-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
8 participants
OBSERVATIONAL
2020-08-01
2021-10-01
Brief Summary
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This study aims to collect clinical and laboratory data of patients with NCL taking Trehalose.
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* Signed informed consent
Exclusion Criteria
* Therapeutic and eating changes in the last four months prior to the study
* Unstable clinical conditions (myoclonus worsening, instability in sleep, parenteral nutrition)
* Refusal to sign the informed consent
7 Years
45 Years
ALL
No
Sponsors
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IRCCS Fondazione Stella Maris
OTHER
Responsible Party
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Filippo Maria Santorelli
Director Molecular Medicine, Neurogenetics and Neuromuscular Disorders
Locations
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IRCCS Fondazione Stella Maris
Pisa, PI, Italy
Countries
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References
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Bajaj L, Lotfi P, Pal R, Ronza AD, Sharma J, Sardiello M. Lysosome biogenesis in health and disease. J Neurochem. 2019 Mar;148(5):573-589. doi: 10.1111/jnc.14564. Epub 2018 Oct 18.
Lee HJ, Yoon YS, Lee SJ. Mechanism of neuroprotection by trehalose: controversy surrounding autophagy induction. Cell Death Dis. 2018 Jun 15;9(7):712. doi: 10.1038/s41419-018-0749-9.
Palmieri M, Pal R, Nelvagal HR, Lotfi P, Stinnett GR, Seymour ML, Chaudhury A, Bajaj L, Bondar VV, Bremner L, Saleem U, Tse DY, Sanagasetti D, Wu SM, Neilson JR, Pereira FA, Pautler RG, Rodney GG, Cooper JD, Sardiello M. mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases. Nat Commun. 2017 Feb 6;8:14338. doi: 10.1038/ncomms14338.
Sardiello M. Transcription factor EB: from master coordinator of lysosomal pathways to candidate therapeutic target in degenerative storage diseases. Ann N Y Acad Sci. 2016 May;1371(1):3-14. doi: 10.1111/nyas.13131.
Schulz A, Ajayi T, Specchio N, de Los Reyes E, Gissen P, Ballon D, Dyke JP, Cahan H, Slasor P, Jacoby D, Kohlschutter A; CLN2 Study Group. Study of Intraventricular Cerliponase Alfa for CLN2 Disease. N Engl J Med. 2018 May 17;378(20):1898-1907. doi: 10.1056/NEJMoa1712649. Epub 2018 Apr 24.
Other Identifiers
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3AL-CLN36
Identifier Type: -
Identifier Source: org_study_id
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