This is a randomized, placebo-controlled, 3-way cross-over phase IIa trial comparing two dose levels of THN102 to placebo in patients suffering from Parkinson's disease associated with excessive daytime sleepiness.

开始日期2018-07-12

申办/合作机构

Theranexus SA

EUCTR2017-004475-31-HU / Not yet recruiting临床2期

Randomised, double-blind, placebo-controlled, complete 3-way cross-over phase IIa trial to investigate safety and efficacy of two THN102 doses in subjects with excessive daytime sleepiness associated with Parkinson’s disease

开始日期2018-04-17

申办/合作机构-

NCT02821715 / Completed临床2期

Safety and Efficacy of THN102 on Sleepiness in Narcoleptic Patients

This Phase 2 study is a 8-site, double-blind, randomised, placebo-controlled, 3-way cross-over trial, involving 3 treatments with Modafinil 300 mg or the combination drug THN102 (Modafinil/Flecainide 300 /3 mg, Modafinil/Flecainide 300 /27 mg).

开始日期2016-09-01

申办/合作机构

Theranexus SA

100 项与 SCNA x DAT 相关的临床结果

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100 项与 SCNA x DAT 相关的转化医学

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0 项与 SCNA x DAT 相关的专利（医药）

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项与 SCNA x DAT 相关的文献（医药）

2022-03-30·Disease Markers4区 · 医学

Screening and Validation of a Carvacrol-Targeting Viability-Regulating Protein, SLC6A3, in Liver Hepatocellular Carcinoma

4区 · 医学

ArticleOA

作者: Yin, Xieling ; Chen, Shi ; Chen, Hongjian ; Zhang, Suqing

Background. Liver hepatocellular carcinoma (LIHC) is the second leading cause of tumor-related death in the world. Carvacrol was also found to inhibit multiple cancer types. Here, we proposed that Carvacrol inhibited LIHC. Methods. We used MTT assay to determine the inhibition of Carvacrol on LIHC cells. BATMAN-TCM was used to predict targets of Carvacrol. These targets were further screened by their survival association and expression in cancer using TCGA data. The bioinformatic screened candidates were further validated in in vitro experiments and clinical samples. Finally, docking models of the interaction of Carvacrol and target protein were conducted. Results. Carvacrol inhibited the viability of LIHC cell lines. 40 target genes of Carvacrol were predicted, 8 of them associated with survival. 4 genes were found differentially expressed in LIHC vs. normal liver. Among these genes, the expression of SLC6A3 and SCN4A was found affected by Carvacrol in LIHC cells, but only SLC6A3 correlated with the viability inhibition of Carvacrol on LIHC cell lines. A docking model of the interaction of Carvacrol and SLC6A3 was established with a good binding affinity. SLC6A3 knockdown and expression revealed that SLC6A3 promoted the viability of LIHC cells. Conclusion. Carvacrol inhibited the viability of LIHC cells by downregulating SLC6A3.

2021-03-01·Brain and Development

Dravet syndrome with parkinsonian symptoms and intact dopaminergic neurons: A case report

作者: Watanabe, Yasuhiro ; Sakata, Ryoichi ; Hanajima, Ritsuko ; Saito, Yoshiaki ; Nishimura, Yoko ; Maegaki, Yoshihiro ; Adachi, Tadashi ; Kanatani, Masahiro

INTRODUCTIONDravet syndrome (DS) is severe myoclonic epilepsy in infancy and associated with a heterozygous mutation of the gene for the sodium channel alpha 1 subunit (SCN1A). Recently, adult patients with DS have been reported to show parkinsonism, but no corresponding neuroimaging data are available. Here, we present neuroimaging data in 2 adult patients with DS showing parkinsonian symptoms.CASE REPORTCase 1: A man who had intractable seizures from the age of 1 year and 2 months was diagnosed with DS at 7 with a mutation in the SCN1A gene. At 18, he had parkinsonian symptoms such as masked face and bradykinesia. At 20, he was admitted to our department. Dopamine transporter single-photon emission computed tomography (DAT SPECT) showed no decrease in striatal binding of ¹²³I-N-ω-fluoropropyl-2β-carbomethoxy-3β-(4-iodophenyl) nortropane (¹²³I-FP-CIT), and myocardial scintigraphy showed no decrease in cardiac uptake of ¹²³I-metaiodobenzylguanidine (¹²³I-MIBG). Levodopa showed no significant improvement in his symptoms. Case 2: A woman who had febrile seizures at 4 months of age and myoclonic seizures at 1 year and 5 months was diagnosed with DS at 31. She had myoclonus, resting tremor, hypertonia, antecollis, crouch gait, and bradykinesia. DAT SPECT imaging showed no decrease in striatal FP-CIT binding, and levodopa did not improve her symptoms.DISCUSSIONThe normal DAT SPECT and ¹²³I-MIBG results suggest that dopaminergic neurons projecting onto striatal neurons were not impaired in our patients, explaining the lack of response to levodopa. Thus, dopamine imaging can help to guide treatment decisions in patients with DS and parkinsonism.

2021-01-01·Parkinsonism & Related Disorders

Parkinsonism in children: Clinical classification and etiological spectrum

Review

作者: Leuzzi, Vincenzo ; Pons, Roser ; Nardecchia, Francesca ; Galosi, Serena

Infantile- and childhood-onset parkinsonism is mainly due to genetic alterations and is an exceedingly rare condition, unlike Parkinson's disease (PD), which is one of the most common neurologic disorders in adulthood. The clinical characterization of parkinsonism during early stages of neuromotor development is controversial due to the lack of consensus regarding the clinical criteria of PD or parkinsonism in the immature brain. The classification here proposed is based on a review of conditions that emerge during infancy and childhood, with key symptoms evocative of adult parkinsonism. The proposed nosography is based on age at presentation, clinical features, outcome, and etiological background. It includes developmental parkinsonism, infantile degenerative parkinsonism, parkinsonism in the setting of neurodevelopmental disorders, parkinsonism in the setting of multisystem brain diseases, juvenile parkinsonism and dystonia-parkinsonism, and acquired parkinsonism. The subgroups denoting peculiar clinical presentations as a consequence of disease impact on the immature brain are developmental parkinsonism due to monoamine metabolic disorders and infantile degenerative parkinsonism caused by DAT and WASR2 defects. More tardive parkinsonisms occur in genetic conditions that cause a generalized derangement of neurodevelopmental processes, such as those due to MECP2, NR4A2, SCN1A, and RAB39B. Some conditions presenting with neurodevelopmental disorder can progress later, disclosing their neurodegenerative nature (i.e. WDR45 and KCND3). Finally, new emerging conditions with childhood-onset parkinsonism arise from the cumulative effect of multiple genetic lesions.