This study is conducted in Japan of Freeze-dried Human Protein C Concentrate (TAK-662) used to treat participants with congenital protein C deficiency.
The main aim of the study is to evaluate for adverse events and effectiveness of congenital protein C deficiency (TAK-662).
During the study, participants with congenital protein C deficiency will be administered with TAK-662 intravenous injection in under routine normal practice. The investigators will evaluate adverse events due to TAK-662 for 12 months. For participants who will be administered in long-term supplementation of TAK-662 after acute treatment or short-term supplementation, the investigator will evaluate for 24 months as a maximum. The study sponsor will not be involved in how the participants are administered but will be recorded what happens during the study.

Start Date06 Sep 2024

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

NCT04984889

/ CompletedPhase 1/2

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.

Start Date07 Sep 2021

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

NCT01705808

/ TerminatedPhase 3IIT

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.

Start Date01 Sep 2012

Sponsor / Collaborator

Università Vita-Salute San Raffaele

100 Clinical Results associated with Human activated protein C(Takeda Pharmaceutical)

100 Translational Medicine associated with Human activated protein C(Takeda Pharmaceutical)

100 Patents (Medical) associated with Human activated protein C(Takeda Pharmaceutical)

Literatures (Medical) associated with Human activated protein C(Takeda Pharmaceutical)

01 Jan 2025·BLOOD COAGULATION & FIBRINOLYSIS

Challenges in managing severe homozygous protein c deficiency: a case report

Article

Author: Alsharidah, Sondus ; Obaid, Mohamad Adel ; Almatter, Eman ; Bourhama, Mona ; Bourusly, Maha

Protein C deficiency is a rare autosomal recessive disorder associated with a high risk of thromboembolic complications. This case report describes the challenges in managing a 23-year-old woman with severe homozygous protein C type 1 deficiency diagnosed since early infancy.Her medical history included misdiagnosed cellulitis, recurrent thrombosis, and permanent vision loss in one eye. The laboratory workup confirmed a diagnosis of severe protein C deficiency. Management involved a combination of fresh frozen plasma (FFP), protein C concentrate, warfarin, and heparin, with ongoing challenges due to recurrent thrombosis and anaphylaxis to FFP.This case highlights the challenges in the diagnosis and management of severe protein C deficiency. Although current treatment options provide partial control, further research is crucial to develop safer and more effective therapies to improve long-term outcomes for affected patients.

01 Aug 2024·Research and Practice in Thrombosis and Haemostasis

Comprehensive literature review of protein C concentrate use in patients with severe congenital protein C deficiency

Review

Author: Wadhwa, Abhinav ; Gazda, Hanna T ; Turecek, Peter L ; Siffel, Csaba ; Sliwka, Henrik ; Ogongo, Margaret Katana ; Tongbram, Vanita

Severe congenital protein C deficiency (SCPCD) is a rare disorder associated with life-threatening purpura fulminans and disseminated intravascular coagulation that typically present within hours after birth. Treatment options for patients with SCPCD include replacement therapy with a plasma-derived protein C concentrate. In this targeted literature review, we summarize information on the use of protein C concentrate as long-term prophylaxis (>1 week of treatment) for patients with SCPCD. In total, 18 publications were included in the review, of which 15 were case studies. Treatment with protein C concentrate (Ceprotin; Baxalta US Inc, a Takeda company; Takeda Manufacturing Austria AG) was reported in 11 publications, and treatment with protein C concentrate (Protexel; LFB Biomedicaments) was reported in 2 publications. One publication reported on both Ceprotin and Protexel. Details of protein C concentrate treatment regimens, including the dose, administration frequency, and route of administration, were reported in 11 publications. Dosing regimens varied across all 11 publications, possibly due to different protein C trough levels among patients or the administration of concomitant medications. Seven of the 11 publications reported on patients who initially received intravenous protein C concentrate and subsequently switched to subcutaneous administration. Treatment outcomes with protein C concentrate were generally favorable, including the prevention of coagulopathy and thrombosis and the healing of cutaneous lesions. Three adverse events in 1 publication were identified as being possibly related to Ceprotin administration. Although published data are limited, this review provides valuable insights into the treatment of patients with SCPCD in clinical practice, including protein C concentrate dosing regimens, administration routes, and associated clinical outcomes.

01 Dec 2021·BMC pediatricsQ4 · MEDICINE

Severe protein C deficiency in a newborn caused by a homozygous pathogenic variant in the PROC gene: a case report

Q4 · MEDICINE

ArticleOA

Author: Ju, Young Seok ; Shin, Seung Han ; Shim, So-Yeon ; Ryu, Young Hye ; Hong, Kiteak ; Lee, Soyoung ; Lee, Jeong Seok ; Park, Seongyeol ; Song, Uisook

Abstract:

Background:

Severe protein C deficiency is a rare and inherited cause of thrombophilia in neonates. Protein C acts as an anticoagulant, and its deficiency results in vascular thrombosis. Herein, we report a case of protein C deficiency with a homozygous pathogenic variant in a term neonate, with good outcomes after proper treatment.

Case presentation:

A four-day-old male newborn was transferred to the Seoul National University Hospital on account of dark red to black skin lesions. He was born full-term with an average birth weight without perinatal problems. There were no abnormal findings in the prenatal tests, including intrauterine sonography. The first skin lesion was observed on his right toes and rapidly progressed to proximal areas, such as the lower legs, left arm, and buttock. Under the impression of thromboembolism or vasculitis, we performed a coagulopathy workup, which revealed a high D-dimer level of 23.05 μg/ml. A skin biopsy showed fibrin clots in most capillaries, and his protein C activity level was below 10%, from which we diagnosed protein C deficiency. On postnatal day 6, he experienced an apnea event with desaturation and an abnormal right pupillary light reflex. Brain computed tomography showed multifocal patchy intracranial hemorrhage and intraventricular hemorrhage with an old ischemic lesion. Ophthalmic examination revealed bilateral retinal traction detachments with retinal folds. Protein C concentrate replacement therapy was added to previous treatments including steroids, prostaglandin E1, and anticoagulation. After replacement therapy, there were no new skin lesions, and the previous lesions recovered with scarring. Although there were no new brain hemorrhagic infarctions, there was ongoing ischemic tissue loss, which required further rehabilitation. Ophthalmic surgical interventions were performed to treat the bilateral retinal traction detachments with retinal folds. Molecular analysis revealed a homozygous pathogenic variant in the PROC gene.

Conclusion:

Severe protein C deficiency can manifest as a fatal coagulopathy in any organ. Early diagnosis and proper treatment, including protein C concentrate replacement, may improve outcomes without serious sequelae.

News (Medical) associated with Human activated protein C(Takeda Pharmaceutical)

11 Nov 2022

·www.ema.europa.eu

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

VaccineDrug ApprovalOrphan Drug

100 Deals associated with Human activated protein C(Takeda Pharmaceutical)

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Purpura Fulminans	Japan	Takeda Pharmaceutical Co., Ltd.	26 Mar 2024
Venous Thromboembolism	Japan	Takeda Pharmaceutical Co., Ltd.	26 Mar 2024
Protein C Deficiency	European Union	Takeda Manufacturing Austria AG	16 Jul 2001
Protein C Deficiency	Iceland	Takeda Manufacturing Austria AG	16 Jul 2001
Protein C Deficiency	Liechtenstein	Takeda Manufacturing Austria AG	16 Jul 2001
Protein C Deficiency	Norway	Takeda Manufacturing Austria AG	16 Jul 2001

Clinical Result

Indication

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04984889 (CTgov)	Phase 1/2	Protein C Deficiency	5	TAK-662	bxxncbmezz(dpzhgzpaau) = vxyzdmvqhy pyxkbnnjmx (kkalucszre, 0.000) View more	-	05 Jan 2024
ISTH2023	Not Applicable	Protein C Deficiency	13	Subcutaneous protein C concentrate	ijqznomcle(gxmhojsbdv) = krfovtpcpv fztmeylzey (pukkpojlbt )	-	24 Jun 2023

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