An Open-Label, Single-Dose, Phase 1/2 Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Human Protein C (TAK-662) for the Treatment of Congenital Protein C Deficiency in Japanese Subjects Followed by an Extension Part

Pharmacokinetic Part:
This study is for Japanese participants with congenital protein C deficiency. The main aim of this study is to check how much TAK-662 stays in their blood over time. This will help the study sponsor (Takeda) to work out the best dose to give patients in the future.
Participants will receive 1 single infusion of TAK-662.
They will stay at the clinic until 3 days after the infusion. Then, participants will return to their clinic 7 days after the infusion to check side effects from the study treatment.
Extension Part:
Participants who will complete the PK part will be given an opportunity to continue TAK-662 administration as 3 different treatment options (on-demand therapy, short-term prophylaxis, and long-term prophylaxis) in the Extension part, until the commercial protein C concentrate is available at each study site or study termination.

开始日期2021-09-07

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT01705808 / Terminated临床3期IIT

Administration of Protein C Concentrates in Adult Critically Ill Septic Patients

Severe sepsis and septic shock are life threatening medical emergencies and are among the most significant challenges in critical care. Case reports and case series suggest that plasma-derived protein C concentrate may improve the outcome of patients with acquired protein C deficiency. Evidence has accumulated on the clinical relevance of the PC pathway in modulating overwhelming inflammation and preventing coagulation derangements, two key mediators of organ damage, and thus of mortality and morbidity, in sepsis. The experience collected through these studies shows that PC is safe, in that it is not associated with bleeding or severe allergic complications,and possibly useful, at least to improve the coagulation abnormalities brought about by sepsis. Unfortunately, however, all we know comes from case series or case reports or an underpowered randomized controlled study. A randomized clinical trial, adequately powered for mortality or clinically relevant outcome, is necessary to confirm PC efficacy.The aim of this study is to demonstrate that Protein C zymogen has clinically relevant implications in terms of reduction of thromboembolic events, 30 days mortality, length of intensive care and hospital stay, time on mechanical ventilation, length of ICU and hospital stay. The study will also confirm that there is no bleeding concern with the use of Protein C concentrates.The study drug will be administered in the Intensive Care Unit for 72 hours and the patients observed till ICU discharge. Telephone followup will be performed at 30 days and at one year.

开始日期2012-09-01

申办/合作机构

Università Vita-Salute San Raffaele

EUCTR2011-004834-32-IT / Unknown statusN/A

Administration of concentrated Protein C in patients with severe sepsis and contraindications to activated protein C. A monocentric, randomised placebo-controlled study.

开始日期2012-06-08

申办/合作机构-

100 项与蛋白质C 相关的临床结果

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100 项与蛋白质C 相关的转化医学

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100 项与蛋白质C 相关的专利（医药）

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401

项与蛋白质C 相关的文献（医药）

2024-02-01·Annals of Neurology

Correction to Lyden P, Pryor KE, Coffey CS, et al. Final results of the RHAPSODY trial: a multi‐center, phase 2 trial using a continual reassessment method to determine the safety and tolerability of 3K3A‐APC, a recombinant variant of human activated protein C, in combination with tissue plasminogen activator, mechanical thrombectomy or both in moderate to severe acute ischemic stroke. Ann Neurol 2019;85:125–136.

2023-06-03·medRxiv : the preprint server for health sciences

Opioid use does not limit potent low-dose HIV-1 latency reversal agent boosting.

作者: Lilie, Tyler ; Clayton, Kiera ; Lin, Nina ; Tsibris, Athe ; Jordan, Hannah ; Asundi, Archana ; Corry, Heather ; Olson, Alex ; Bouzy, Jennifer ; Roche, Samantha ; Coxen, Kendyll ; Taylor, Jessica

The combined effects of the HIV-1 and opioid epidemics on virus reservoir dynamics are less well characterized. To assess the impact of opioid use on HIV-1 latency reversal, we studied forty-seven suppressed participants with HIV-1 and observed that lower concentrations of combination latency reversal agents (LRA) led to synergistic virus reactivation ex vivo, regardless of opioid use. The use of a Smac mimetic or low-dose protein kinase C agonist, compounds that did not reverse latency alone, in combination with low-dose histone deacetylase inhibitors generated significantly more HIV-1 transcription than phorbol 12-myristate 13-acetate (PMA) with ionomycin, the maximal known HIV-1 reactivator. This LRA boosting did not differ by sex or race and associated with greater histone acetylation in CD4⁺ T cells and modulation of T cell phenotype. Virion production and the frequency of multiply spliced HIV-1 transcripts did not increase, suggesting a post-transcriptional block still limits potent HIV-1 LRA boosting.

2022-12-09·Hematology

Thrombosis questions from the inpatient wards

Review

作者: Goshua, George ; Lee, Alfred Ian ; Bendapudi, Pavan K.

AbstractThe multifaceted pathophysiologic processes that comprise thrombosis and thromboembolic diseases take on a particular urgency in the hospitalized setting. In this review, we explore 3 cases of thrombosis from the inpatient wards: purpura fulminans, cancer-associated thrombosis with thrombocytopenia, and coronavirus disease 2019 (COVID-19) and the use of dose-escalated anticoagulation therapy and antiplatelet agents. We discuss the evaluation and management of purpura fulminans and the roles of plasma transfusion, protein C and antithrombin replacement, and anticoagulation in treating this disease. We present a framework for evaluating the etiologies of thrombocytopenia in cancer and review 2 strategies for anticoagulation management in patients with cancer-associated thrombosis and thrombocytopenia, including recent prospective data supporting the use of dose-modified anticoagulation based on platelet count. Last, we dissect the major clinical trials of therapeutic- and intermediate-dose anticoagulation and antiplatelet therapy in hospitalized patients with COVID-19, reviewing key recommendations from consensus guidelines while highlighting ways in which institutional and patient-tailored practices regarding antithrombotic therapies in COVID-19 may differ. Together, the cases highlight the diverse and dramatic presentations of macro- and microvascular thrombosis as encountered on the inpatient wards.

项与蛋白质C 相关的新闻（医药）

2022-11-11

·ema

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 7-10 November 2022

Four new medicines recommended for approval EMA’s human medicines committee ( CHMP ) recommended four medicines for approval at its November 2022 meeting. The CHMP recommended authorising the COVID-19 vaccine VidPrevtynBeta (COVID-19 vaccine (recombinant, adjuvanted)) as a booster in adults previously vaccinated with an mRNA or adenoviral vector COVID-19 vaccine. It is the seventh vaccine recommended in the European Union (EU) for protecting against COVID-19 and, together with the vaccines already authorised, will support vaccination campaigns in EU Member States during the pandemic. See more information in the news announcement in the grid below. The committee adopted a positive opinion for a biosimilar medicine , Kauliv (teriparatide), for the treatment of osteoporosis, a health condition that weakens bones, making them fragile and more likely to break. A generic medicine , Pirfenidone Viatris (pirfenidone), received a positive opinion for the treatment of idiopathic pulmonary fibrosis, a chronic and progressive condition in which the lungs become scarred and breathing becomes increasingly difficult. The CHMP adopted a positive opinion for a generic medicine , Sugammadex Amomed (sugammadex), intended for the reversal of neuromuscular blockade induced by rocuronium in adults and children or vecuronium in adults. Rocuronium and vecuronium are muscle relaxants used during some types of surgeries. Sugammadex is used to speed up the recovery from the effects of the muscle relaxant. Recommendations on extensions of therapeutic indication for 11 medicines The committee recommended 11 extensions of indication for medicines that are already authorised in the EU: Ceprotin , Comirnaty , DuoPlavin , Dupixent , Enhertu , Eylea , Imfinzi , Iscover , Lynparza , Plavix and Xofluza . Withdrawals of applications Two applications for marketing authorisation were withdrawn: Orepaxam * for the treatment of pulmonary arterial hypertension, and Febseltiq * for the treatment of cholangiocarcinoma (cancer of the bile ducts). Two applications for extensions of therapeutic indications were withdrawn: Gavreto for the treatment of certain types of thyroid cancer, and Ilaris for the treatment of Schnitzler syndrome, a rare inflammatory disease causing long-term urticaria, recurrent fever, bone and joint pain, and swollen lymph nodes. Question-and-answer documents on the withdrawals are available in the grid below. COVID-19 update The committee recommended extending the use of COVID-19 vaccine Comirnaty targeting the original strain and Omicron subvariants BA.4 and BA.5 in children between 5 to 11 years of age. An overview of all the COVID-19 vaccines authorised in the EU is available on EMA’s website. Safety update The CHMP endorsed the measures recommended by the Pharmacovigilance Risk Assessment Committee ( PRAC ) to minimise the risk of serious side effects with Janus kinase (JAK) inhibitors used to treat several chronic inflammatory disorders. These side effects include cardiovascular conditions, blood clots, cancer and serious infections. This recommendation is the outcome of an article 20 referral procedure, which is triggered for medicines that have been authorised via the centralised procedure in case ofquality, safety or efficacy issues. A public health communication on this referral is available in the grid below. Agenda and minutes The agenda of the November 2022 CHMP meeting is published on EMA's website. Minutes of the October 2022 CHMP meeting will be published in the coming weeks. CHMP statistics Key figures from the November 2022 CHMP meeting are represented in the graphic below. *This product was designated as an orphan medicine during its development. Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products ( COMP ) at the time of approval to determine whether the information available to date allows maintaining the medicine’s orphan status and granting the medicine ten years of market exclusivity . Positive recommendation on new medicines Name of medicine VidPrevtyn Beta International non-proprietary name (INN) COVID-19 vaccine (recombinant, adjuvanted) Marketing-authorisation applicant Sanofi Pasteur Therapeutic indication VidPrevtyn Beta is indicated as a booster for active immunisation to prevent COVID-19 in adults who have previously received a mRNA or adenoviral vector COVID-19 vaccine More information VidPrevtyn Beta: Pending EC decision News announcement: EMA recommends approval of VidPrevtyn Beta as a COVID 19 booster vaccine Positive recommendation on new biosimilar medicine Name of medicine Kauliv INN teriparatide Marketing-authorisation applicant Strides Pharma Cyprus Therapeutic indication Treatment of osteoporosis More information Kauliv: Pending EC decision Positive recommendations on new generic medicines Name of medicine Pirfenidone Viatris INN pirfenidone Marketing-authorisation applicant Viatris Limited Therapeutic indication Treatment of idiopathic pulmonary fibrosis More information Pirfenidone Viatris: Pending EC decision Name of medicine Sugammadex Amomed INN sugammadex Marketing-authorisation applicant AOP Orphan Pharmaceuticals GmbH Therapeutic indication Reversal of neuromuscular blockade induced by rocuronium or vecuronium More information Sugammadex Amomed: Pending EC decision Positive recommendations on extensions of indications Name of medicine Ceprotin INN human protein C Marketing-authorisation holder Takeda Manufacturing Austria AG More information Ceprotin: Pending EC decision Name of medicine Comirnaty INN tozinameran Marketing-authorisation holder BioNTech Manufacturing GmbH More information Comirnaty: Pending EC decision Name of medicine DuoPlavin INN clopidogrel / acetylsalicylic acid Marketing-authorisationholder Sanofi-aventis groupe More information DuoPlavin: Pending EC decision Name of medicine Dupixent INN dupilumab Marketing-authorisation holder Sanofi-aventis groupe More information Dupixent: Pending EC decision Name of medicine Enhertu INN trastuzumab deruxtecan Marketing-authorisation holder Daiichi Sankyo Europe GmbH More information Enhertu: Pending EC decision Name of medicine Eylea INN aflibercept Marketing-authorisation holder Bayer AG More information Eylea: Pending EC decision Name of medicine Imfinzi INN durvalumab Marketing-authorisation holder AstraZeneca AB More information Imfinzi: Pending EC decision Name of medicine Iscover INN clopidogrel Marketing-authorisationholder Sanofi-aventis groupe More information Iscover: Pending EC decision Name of medicine Lynparza INN olaparib Marketing-authorisationholder AstraZeneca AB More information Lynparza: Pending EC decision Name of medicine Plavix INN clopidogrel Marketing-authorisationholder Sanofi-aventis groupe More information Plavix: Pending EC decision Name of medicine Xofluza INN baloxavir marboxil Marketing-authorisationholder Roche Registration GmbH More information Xofluza: Pending EC decision Withdrawal of initial marketing authorisation application Name of medicine Febseltiq INN infigratinib Marketing-authorisation applicant Helsinn Birex Pharmaceuticals Limited More information Febseltiq: Withdrawn application Name of medicine Orepaxam INN treprostinil diolamine Marketing-authorisationapplicant Ferrer Internacional S.A. More information Orepaxam: Withdrawn application Withdrawals of post-authorisation marketing authorisation applications Name of medicine Gavreto INN pralsetinib More information Gavreto: Withdrawn application Name of medicine Ilaris INN canakinumab More information Ilaris: Withdrawn application Conclusion of referral Name of medicine Janus kinase (JAK) inhibitors More information Janus Kinase inhibitors (JAKi) Other updates List item Scientific advice and protocol assistance adopted during the CHMP meeting 7-10 November 2022 (PDF/246.92 KB) (new) Adopted First published: 11/11/2022 EMA/CHMP/SAWP/872304/2022 List item Start of Union reviews adopted during the CHMP meeting of 7-10 November 2022 (PDF/106.5 KB) (new) Adopted First published: 11/11/2022 EMA/846051/2022

疫苗上市批准孤儿药

100 项与蛋白质C 相关的药物交易

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适应症	国家/地区	公司	日期
暴发性紫癜	日本	Takeda Pharmaceutical Co., Ltd.	2024-03-26
静脉血栓栓塞	日本	Takeda Pharmaceutical Co., Ltd.	2024-03-26
蛋白质C缺乏	冰岛	Takeda Manufacturing Austria AG	2001-07-16
蛋白质C缺乏	挪威	Takeda Manufacturing Austria AG	2001-07-16
蛋白质C缺乏	列支敦士登	Takeda Manufacturing Austria AG	2001-07-16
蛋白质C缺乏	欧盟	Takeda Manufacturing Austria AG	2001-07-16

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适应症	最高研发状态	国家/地区	公司	日期
蛋白质C缺乏	临床2期	日本	Takeda Pharmaceutical Co., Ltd.	2021-09-07

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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


ISTH2023	N/A	蛋白质C缺乏	13	Subcutaneous protein C concentrate	齋窪醖壓夢淵廠鬱鹹蓋(糧簾鑰窪簾鹹觸窪製糧) = 網鏇鹽構夢簾顧淵簾蓋壓膚艱顧製糧衊醖壓簾 (繭觸鬱願鑰鹹膚遞憲壓 )	-	2023-06-24
NCT04984889 (CTgov)	临床1/2期	蛋白质C缺乏	5	TAK-662	願窪蓋鬱構觸蓋鹹鬱網(蓋鏇鹹願蓋構製選糧顧) = 網網鬱鹹簾鹹鹹選艱顧遞鬱鬱淵蓋遞選積糧範 (夢廠構淵鬱蓋衊遞窪鬱, 鬱襯蓋積選壓蓋繭網餘 ~ 餘糧艱鑰廠鹽鏇憲繭鏇) 更多	-	2024-01-05