Tranilast for advanced heart failure in patients with muscular dystrophy: a single-arm, open-label, multicenter study. was written by Matsumura, Tsuyoshi;Hashimoto, Hiroya;Sekimizu, Masahiro;Saito, Akiko M;Motoyoshi, Yasufumi;Nakamura, Akinori;Kuru, Satoshi;Fukudome, Takayasu;Segawa, Kazuhiko;Takahashi, Toshiaki;Tamura, Takuhisa;Komori, Tetsuo;Watanabe, Chigusa;Asakura, Masanori;Kimura, Koichi;Iwata, Yuko. And the article was included in Orphanet journal of rare diseases in 2022.SDS of cas: 53902-12-8 This article mentions the following:
BACKGROUND: The transient receptor potential cation channel subfamily V member 2 (TRPV2) is a stretch-sensitive calcium channel. TRPV2 overexpression in the sarcolemma of skeletal and cardiac myocytes causes calcium influx into the cytoplasm, which triggers myocyte degeneration. In animal models of cardiomyopathy and muscular dystrophy (MD), TRPV2 inhibition was effective against heart failure and motor function. Our previous pilot study showed that tranilast, a TRPV2 inhibitor, reduced brain natriuretic peptide (BNP) levels in two MD patients with advanced heart failure. Thus, this single-arm, open-label, multicenter study aimed to evaluate the safety and efficacy of tranilast for heart failure. METHODS: The study enrolled MD patients with advanced heart failure whose serum BNP levels wereâ?gt;â?00Â pg/mL despite receiving standard cardioprotective therapy. Tranilast was administered orally at 100Â mg, thrice daily. The primary endpoint was the change in log (BNP) (Îlog [BNP]) at 6Â months from baseline. The null hypothesis was determined based on a previous multicenter study of carvedilol results in a mean population Îlog (BNP) of 0.18. TRPV2 expression on peripheral blood mononuclear cell surface, cardiac events, total mortality, left ventricular fractional shortening, human atrial natriuretic peptide, cardiac troponin T, and creatine kinase, and pinch strength were also assessed. RESULTS: Because of the poor general condition of many patients, only 18 of 34 patients were included and 13 patients could be treated according to the protocol throughout the 6-month period. However, there were no serious adverse events related to tranilast except diarrhea, a known adverse effect, and the drug was administered safely. TRPV2 expression on the mononuclear cell surface was elevated at baseline and reduced after treatment. Cardiac biomarkers such as BNP, human atrial natriuretic peptide, and fractional shortening remained stable, suggesting a protective effect against the progression of heart failure. In the per protocol set group, Îlog [BNP] was -â?.2 and significantly lower than that in the null hypothesis. CONCLUSIONS: Tranilast is safe and effective in inhibiting TRPV2 expression, even in MD patients with advanced heart failure. Further trials are needed to evaluate the efficacy of tranilast in preventing myocardial damage, heart failure, motor impairment, and respiratory failure. Clinical trial registration The study was registered in the UMIN Clinical Trials Registry (UMIN-CTR: UMIN000031965, URL: http://www.umin.ac.jp/ctr/ ) [March 30, 2018] and the Japan Registry of Clinical Trials (jRCT, registration number: jRCTs031180038, URL: https://jrct.niph.go.jp/ ) [November 12, 2021]. Patient registration was started in December 19, 2018. In the experiment, the researchers used many compounds, for example, 2-(3-(3,4-Dimethoxyphenyl)acrylamido)benzoic acid (cas: 53902-12-8SDS of cas: 53902-12-8).
2-(3-(3,4-Dimethoxyphenyl)acrylamido)benzoic acid (cas: 53902-12-8) belongs to amides. Amides can be viewed as a derivative of a carboxylic acid RC(=O)OH with the hydroxyl group âOH replaced by an amine group âNRâ²Râ? or, equivalently, an acyl (alkanoyl) group RC(=O)â?joined to an amine group. Amides can be recrystallised from large quantities of water, ethanol, ethanol/ether, aqueous ethanol, chloroform/toluene, chloroform or acetic acid. The likely impurities are the parent acids or the alkyl esters from which they have been made. The former can be removed by thorough washing with aqueous ammonia followed by recrystallisation, whereas elimination of the latter is by trituration or recrystallisation from an organic solvent.SDS of cas: 53902-12-8
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