The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.

开始日期2011-04-01

申办/合作机构

HemaQuest Pharmaceuticals, Inc.

NCT00790127 / Completed临床1/2期

A Multi-National, Blinded, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HQK-1001 in Subjects With Beta Thalassemia Intermedia, Including Hemoglobin E Beta Thalassemia

The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia

开始日期2009-03-01

申办/合作机构

HemaQuest Pharmaceuticals, Inc.

100 项与 Dimebutic acid 相关的临床结果

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100 项与 Dimebutic acid 相关的转化医学

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100 项与 Dimebutic acid 相关的专利（医药）

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项与 Dimebutic acid 相关的文献（医药）

2023-06-01·Science of The Total Environment

Appraising co-composting efficiency of biodegradable plastic bags and food wastes: Assessment microplastics morphology, greenhouse gas emissions, and changes in microbial community

Article

作者: Qiu, Yizhan ; Zhang, Luxi ; Ren, Lianhai ; Wang, Pan ; Muhmood, Atif ; Lu, Jiaxin

Biodegradable plastic bags (BPBs) to collect food waste and microplastics (MPs) produced from their biodegradation have received considerable scientific attention recently. Therefore, the current study was carried out to assess the co-composting efficiency of biodegradable plastic bags (polylactic acid (PLA) + polybutylene terephthalate (PBAT) + ST20 and PLA + PBAT+MD25) and food waste. The variations in greenhouse gas (GHG) emissions, microbial community and compost fertility were likewise assessed. Compared with the control, PLA + PBAT+ST20 and PLA + PBAT+MD25 both accelerated organic matter degradation and increased temperature. Moreover, PLA + PBAT+ST20 aggravated CH₄ and CO₂ emissions by 12.10 % and 11.01 %, respectively. PLA + PBAT+MD25 decreased CH₄ and CO₂ emissions by 5.50 % and 9.12 %, respectively. Meanwhile, compared with PLA + PBAT+ST20, the combined effect of plasticizer and inorganic additive in PLA + PBAT+MD25, reduced the NO₃^--N contents, seed germination index (GI) and compost maturity. Furthermore, adding BPBs changed the richness and diversity of the bacterial community (Firmicutes, Proteobacteria and Bacteroidetes). Likewise, redundancy analysis (RDA) showed that the co-compost system of BPBs and food waste accelerated significantly bacterial community succession from Firmicutes and Bacteroidetes at the initial stage to Proteobacteria and Actinobacteria at the mature stage, increased co-compost temperature to over 64 °C and extended thermophilic composting phase, and promoted the degradation of MPs. Additionally, according to structural equation model quantification results, the inorganic additive of PLA + PBAT+MD25 had more serious toxicity to microorganisms and had significantly adverse effects on GI through CO₂-C (λ = -0.415, p < 0.05) and NO₃^--N (λ = -0.558, p < 0.001), thus reduced compost fertility and quality. The results also indicated that the BPBs with ST20 as an additive could be more suitable for industrial composting than the BPBs with MD25 as an additive. This study provided a vital basis for understanding the potential environmental and human health risks of the MPs' generated by the degradation of BPBs in compost.

2023-01-13·The Cochrane database of systematic reviews

Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias.

Review

作者: Lau, Doris Sc ; Ho, Jacqueline J ; Loh, C Khai ; Foong, Wai Cheng

BACKGROUNDNon-transfusion-dependent β-thalassaemia (NTDβT) is a subset of inherited haemoglobin disorders characterised by reduced production of the β-globin chain of haemoglobin leading to anaemia of varying severity. Although blood transfusion is not a necessity for survival, it may be required to prevent complications of chronic anaemia, such as impaired growth and hypercoagulability. People with NTDβT also experience iron overload due to increased iron absorption from food sources which becomes more pronounced in those requiring blood transfusion. People with a higher foetal haemoglobin (HbF) level have been found to require fewer blood transfusions, thus leading to the emergence of treatments that could increase its level. HbF inducers stimulate HbF production without altering any gene structures. Evidence for the possible benefits and harms of these inducers is important for making an informed decision on their use.OBJECTIVESTo compare the effectiveness and safety of the following for reducing blood transfusion for people with NTDβT: 1. HbF inducers versus usual care or placebo; 2. single HbF inducer with another HbF inducer, and single dose with another dose; and 3. combination of HbF inducers versus usual care or placebo, or single HbF inducer.SEARCH METHODSWe used standard, extensive Cochrane search methods. The latest search date was 21 August 2022.SELECTION CRITERIAWe included randomised controlled trials (RCTs) or quasi-RCTs comparing single HbF inducer with placebo or usual care, with another single HbF inducer or with a combination of HbF inducers; or comparing different doses of the same HbF inducer.DATA COLLECTION AND ANALYSISWe used standard Cochrane methods. Our primary outcomes were blood transfusion and haemoglobin levels. Our secondary outcomes were HbF levels, the long-term sequelae of NTDβT, quality of life and adverse events.MAIN RESULTSWe included seven RCTs involving 291 people with NTDβT, aged two to 49 years, from five countries. We reported 10 comparisons using eight different HbF inducers (four pharmacological and four natural): three RCTs compared a single HbF inducer to placebo and seven to another HbF inducer. The duration of the intervention lasted from 56 days to six months. Most studies did not adequately report the randomisation procedures or whether and how blinding was achieved. HbF inducer against placebo or usual care Three HbF inducers, HQK-1001, Radix Astragali or a 3-in-1 combined natural preparation (CNP), were compared with a placebo. None of the comparisons reported the frequency of blood transfusion. We are uncertain whether Radix Astragali and CNP increase haemoglobin at three months (mean difference (MD) 1.33 g/dL, 95% confidence interval (CI) 0.54 to 2.11; 1 study, 2 interventions, 35 participants; very low-certainty evidence). We are uncertain whether Radix Astragali and CNP have any effect on HbF (MD 12%, 95% CI -0.74% to 24.75%; 1 study, 2 interventions, 35 participants; very low-certainty evidence). Only medians on haemoglobin and HbF levels were reported for HQK-1001. Adverse effects reported for HQK-1001 were nausea, vomiting, dizziness and suprapubic pain. There were no prespecified adverse effects for Radix Astragali and CNP. HbF inducer versus another HbF inducer Four studies compared a single inducer with another over three to six months. Comparisons included hydroxyurea versus resveratrol, hydroxyurea versus thalidomide, hydroxyurea versus decitabine and Radix Astragali versus CNP. No study reported our prespecified outcomes on blood transfusion. Haemoglobin and HbF were reported for the comparison Radix Astragali versus CNP, but we are uncertain whether there were any differences (1 study, 24 participants; low-certainty evidence). Different doses of the same HbF inducer Two studies compared two different types of HbF inducers at different doses over two to six months. Comparisons included hydroxyurea 20 mg/kg/day versus 10 mg/kg/day and HQK-1001 10 mg/kg/day, 20 mg/kg/day, 30 mg/kg/day and 40 mg/kg/day. Blood transfusion, as prespecified, was not reported. In one study (61 participants) we are uncertain whether the lower levels of both haemoglobin and HbF at 24 weeks were due to the higher dose of hydroxyurea (haemoglobin: MD -2.39 g/dL, 95% CI -2.80 to -1.98; very low-certainty evidence; HbF: MD -10.20%, 95% CI -16.28% to -4.12%; very low-certainty evidence). The study of the four different doses of HQK-1001 did not report results for either haemoglobin or HbF. We are not certain if major adverse effects may be more common with higher hydroxyurea doses (neutropenia: risk ratio (RR) 9.93, 95% CI 1.34 to 73.97; thrombocytopenia: RR 3.68, 95% CI 1.12 to 12.07; very low-certainty evidence). Taking HQK-1001 20 mg/kg/day may result in the fewest adverse effects. A combination of HbF inducers versus a single HbF inducer Two studies compared three combinations of two inducers with a single inducer over six months: hydroxyurea plus resveratrol versus resveratrol or hydroxyurea alone, and hydroxyurea plus l-carnitine versus hydroxyurea alone. Blood transfusion was not reported. Hydroxyurea plus resveratrol may reduce haemoglobin compared with either resveratrol or hydroxyurea alone (MD -0.74 g/dL, 95% CI -1.45 to -0.03; 1 study, 54 participants; low-certainty evidence). We are not certain whether the gastrointestinal disturbances, headache and malaise more commonly reported with hydroxyurea plus resveratrol than resveratrol alone were due to the interventions. We are uncertain whether hydroxyurea plus l-carnitine compared with hydroxyurea alone may increase mean haemoglobin, and reduce pulmonary hypertension (1 study, 60 participants; very low-certainty evidence). Adverse events were reported but not in the intervention group. None of the comparisons reported the outcome of HbF.AUTHORS' CONCLUSIONSWe are uncertain whether any of the eight HbF inducers in this review have a beneficial effect on people with NTDβT. For each of these HbF inducers, we found only one or at the most two small studies. There is no information on whether any of these HbF inducers have an effect on our primary outcome, blood transfusion. For the second primary outcome, haemoglobin, there may be small differences between intervention groups, but these may not be clinically meaningful and are of low- to very low-certainty evidence. Data on adverse effects and optimal doses are limited. Five studies are awaiting classification, but none are ongoing.

2017-03-01·Toxicon

St20, a new venomous animal derived natural peptide with immunosuppressive and anti-inflammatory activities

Article

作者: Cao, Zhijian ; Li, Dandan ; Chai, Lin ; Xiao, Min ; Sun, Yuwen ; Chen, Zongyun ; Li, Wenxin ; Wu, Yingliang ; Li, Jian ; Li, Shan ; Yang, Weishan ; Zhang, Hua ; Ding, Li ; Yang, Xianyi

Peptide toxins from venomous animals are natural resources with diverse biological functions and therapeutic potential towards human diseases. For venomous scorpions, many valuable peptide toxins have been discovered from Buthidae scorpions, but few works were done about non-buthidae scorpions. Here, we cloned and characterized the first disulfide-bridged toxin peptide St20 from the non-buthidae scorpion Scorpiops tibetanus. St20 has a putative 23-residue signal peptide, followed by a presumed 34-residue mature peptide including 8 cysteines. Sequence alignments and structural analysis suggested that St20 is a new member of α-KTx23 scorpion toxin subfamily with a conserved CSα/β structural fold. Functional studies showed that St20 inhibited human T lymphocyte surface marker CD69 expression and cytokine IL-2 secretion. Beside this, St20 inhibited two important pro-inflammatory factors TNF-α and IFN-γ secretion in the activated human T lymphocyte. Animal experiments showed that the delayed-type hypersensitivity response in rat autoimmune disease model was ameliorated in the present of peptide toxin St20. Together, our results showed that St20 is the first disulfide-bridged toxin peptide from the non-buthidae scorpion Scorpiops tibetanus with immunosuppressive and anti-inflammatory activities, suggesting that toxins from non-buthidae scorpions might be a new source of peptide drug discovery towards human diseases.

项与 Dimebutic acid 相关的新闻（医药）

2008-12-09

·BioSpace

HemaQuest Announces Clinical Progress on New Sickle Cell Anemia Drug

SAN FRANCISCO, CA--(Marketwire - December 09, 2008) - HemaQuest Pharmaceuticals announced today the successful completion of Phase 1 clinical trials of HQK-1001, an orally-administered therapeutic which the company is developing to treat hemoglobin disorders, including sickle cell anemia and beta thalassemia. The company presented the results at the annual meeting of the American Society of Hematology in San Francisco.

100 项与 Dimebutic acid 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
镰状细胞血症	临床2期	美国	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	牙买加	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	加拿大	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	埃及	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	黎巴嫩	HemaQuest Pharmaceuticals, Inc.	2011-04-01
地中海贫血	临床2期	-	Boston University	-
β地中海贫血	临床前	黎巴嫩	HemaQuest Pharmaceuticals, Inc.	2012-05-01

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


Pubmed \| Am J Hematol	临床2期	镰状细胞血症	76	HQK-1001	(膚醖繭艱窪獵艱齋繭廠) = The most common adverse events in the HQK-1001 group, usually graded as mild or moderate, consisted of abdominal pain 願積艱膚鏇顧膚廠壓積 (廠壓憲壓鹽範憲夢築夢 ) 更多	不佳	2014-07-01
				Placebo
NCT00842088 (Pubmed \| Am J Hematol)	临床1/2期	镰状细胞血症	24	HQK-1001 10 mg/kg	(鹹鏇製醖蓋膚鑰窪顧膚) = 獵積憲獵網窪網願鬱遞製餘餘艱憲衊糧淵簾顧 (簾積觸憲網餘鹹淵艱夢 ) 更多	-	2012-11-01
				HQK-1001 20 mg/kg	(鹹鏇製醖蓋膚鑰窪顧膚) = 繭廠壓窪壓廠遞鹽鹹淵製餘餘艱憲衊糧淵簾顧 (簾積觸憲網餘鹹淵艱夢 ) 更多