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http://hdl.handle.net/2445/179931
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DC Field | Value | Language |
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dc.contributor.author | Bremova‐Ertl, Tatiana | - |
dc.contributor.author | Claassen, Jens | - |
dc.contributor.author | Foltan, Tomas | - |
dc.contributor.author | Gascon Bayarri, Jordi | - |
dc.contributor.author | Gissen, Paul | - |
dc.contributor.author | Hahn, Andreas | - |
dc.contributor.author | Hassan, Anhar | - |
dc.contributor.author | Hennig, Anita | - |
dc.contributor.author | Jones, Simon A. | - |
dc.contributor.author | Kolnikova, Miriam | - |
dc.contributor.author | Martakis, Kyriakos | - |
dc.contributor.author | Raethjen, Jan | - |
dc.contributor.author | Ramaswami, Uma | - |
dc.contributor.author | Sharma, Reena | - |
dc.contributor.author | Schneider, Susanne A. | - |
dc.date.accessioned | 2021-09-10T10:05:23Z | - |
dc.date.available | 2021-09-10T10:05:23Z | - |
dc.date.issued | 2021-08-13 | - |
dc.identifier.issn | 1432-1459 | - |
dc.identifier.uri | http://hdl.handle.net/2445/179931 | - |
dc.description.abstract | Objective: To investigate the safety and efficacy of N-acetyl-L-leucine (NALL) on symptoms, functioning, and quality of life in pediatric (≥ 6 years) and adult Niemann-Pick disease type C (NPC) patients. Methods: In this multi-national, open-label, rater-blinded Phase II study, patients were assessed during a baseline period, a 6-week treatment period (orally administered NALL 4 g/day in patients ≥ 13 years, weight-tiered doses for patients 6-12 years), and a 6-week post-treatment washout period. The primary Clinical Impression of Change in Severity (CI-CS) endpoint (based on a 7-point Likert scale) was assessed by blinded, centralized raters who compared randomized video pairs of each patient performing a pre-defined primary anchor test (8-Meter Walk Test or 9-Hole Peg Test) during each study periods. Secondary outcomes included cerebellar functional rating scales, clinical global impression, and quality of life assessments. Results: 33 subjects aged 7-64 years with a confirmed diagnosis of NPC were enrolled. 32 patients were included in the primary modified intention-to-treat analysis. NALL met the CI-CS primary endpoint (mean difference 0.86, SD = 2.52, 90% CI 0.25, 1.75, p = 0.029), as well as secondary endpoints. No treatment-related serious adverse events occurred. Conclusions: NALL demonstrated a statistically significant and clinical meaningfully improvement in symptoms, functioning, and quality of life in 6 weeks, the clinical effect of which was lost after the 6-week washout period. NALL was safe and well-tolerated, informing a favorable benefit-risk profile for the treatment of NPC. CLINICALTRIALS. Gov identifier: NCT03759639. | - |
dc.format.extent | 12 p. | - |
dc.format.mimetype | application/pdf | - |
dc.language.iso | eng | - |
dc.publisher | Springer Science and Business Media LLC | - |
dc.relation.isformatof | Reproducció del document publicat a: https://doi.org/10.1007/s00415-021-10717-0 | - |
dc.relation.ispartof | Journal of Neurology, 2021 | - |
dc.relation.uri | https://doi.org/10.1007/s00415-021-10717-0 | - |
dc.rights | cc by (c) Bremova‐Ertl, Tatiana et al, 2021 | - |
dc.rights.uri | http://creativecommons.org/licenses/by/3.0/es/ | * |
dc.source | Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL)) | - |
dc.subject.classification | Malaltia d'Alzheimer | - |
dc.subject.classification | Infants | - |
dc.subject.classification | Terapèutica | - |
dc.subject.other | Alzheimer's disease | - |
dc.subject.other | Children | - |
dc.subject.other | Therapeutics | - |
dc.title | Efficacy and safety of N-acetyl-l-leucine in Niemann–Pick disease type C | - |
dc.type | info:eu-repo/semantics/article | - |
dc.type | info:eu-repo/semantics/publishedVersion | - |
dc.date.updated | 2021-09-10T08:24:23Z | - |
dc.rights.accessRights | info:eu-repo/semantics/openAccess | - |
dc.identifier.pmid | 34387740 | - |
Appears in Collections: | Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL)) |
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File | Description | Size | Format | |
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Bremova-Ertl2021_Article_EfficacyAndSafetyOfN-acetyL-L-.pdf | 1.36 MB | Adobe PDF | View/Open |
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