A Multicenter, Post-Marketing Registry Study of COAGADEX® in the Peri-operative Management of Patients With Moderate or Severe Hereditary Factor X Deficiency Undergoing Major Surgery

This is a non-interventional, multicenter, post-marketing registry study in three patients with moderate or severe hereditary FX deficiency, to assess Coagadex administered peri-operatively for hemostatic cover in major surgery during routine post-marketing use.

Start Date27 Feb 2018

Sponsor / Collaborator

Bio Products Laboratory USA, Inc.

NCT01721681

/ CompletedPhase 3

A Phase III Open, Multicentre Study to Investigate the Safety, Pharmacokinetics and Efficacy of BPL's High Purity Factor X in the Prophylaxis of Bleeding in Factor X Deficient Children Under the Age of 12 Years

The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months.
The secondary objectives of the study are:
To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.
To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks).

Start Date01 Apr 2015

Sponsor / Collaborator

Bio Products Laboratory USA, Inc.

NCT01086852

/ TerminatedPhase 3

A Phase III Open, Multicentre Study to Investigate the Safety and Efficacy of BPL's High Purity FACTOR X in the Treatment of Factor X Deficient Subjects Undergoing Surgery

To primary efficacy variable is to assess the presence or absence of excessive blood loss during and after surgery.
The secondary efficacy endpoints are as follows:
A subjective overall assessment by the investigator of FACTOR X in the control of bleeding during surgery.
The incidence of bleeding episodes during treatment with FACTOR X while the subject is at risk of post-operative bleeding, including location and duration.
Incremental recovery of FX:C and FX:Ag after the pre-surgery bolus infusion.
Assessment of FX:C and FX:Ag levels on each day post-surgery.
Assessment of the cumulative weight-adjusted doses of FACTOR X as measured by FX:C (IU/kg body weight) administered to each subject to maintain haemostasis.
Assessment of the cumulative doses of FACTOR X as measured by FX:C (IU) administered to each subject to maintain haemostasis.
Amount of weight-adjusted FACTOR X as measured by FX:C (IU/kg body weight) administered daily (day of surgery and each post-operative day) to maintain haemostasis.

Start Date01 Mar 2011

Sponsor / Collaborator

Bio Products Laboratory USA, Inc.

100 Clinical Results associated with Human Coagulation Factor X(Bio Products)

100 Translational Medicine associated with Human Coagulation Factor X(Bio Products)

100 Patents (Medical) associated with Human Coagulation Factor X(Bio Products)

Literatures (Medical) associated with Human Coagulation Factor X(Bio Products)

01 Jul 2022·HAEMOPHILIAQ2 · MEDICINE

A review of the pharmacokinetics, efficacy and safety of high‐purity factor X for the prophylactic treatment of hereditary factor X deficiency

Q2 · MEDICINE

Review

Author: Leavitt, Andrew D. ; Payne, Jeanette ; Batsuli, Glaivy ; Mathias, Mary ; McGuinn, Catherine E.

Abstract:

Introduction:

Hereditary factor X (FX) deficiency (FXD) is a rare autosomal recessive bleeding disorder. Plasma‐derived FX (pdFX) is a high‐purity FX concentrate approved in the United States and Europe for the treatment and prophylaxis of bleeding episodes and for peri‐operative management in patients with hereditary FXD (HFXD).

Aim:

To review pharmacokinetic dosing, efficacy, and safety data for pdFX as routine prophylaxis for HFXD.

Methods:

Summary of the published pharmacokinetic and safety data from TEN01, TEN02, TEN05, and real‐world publications of pdFX for prophylaxis.

Results:

Pharmacokinetic modelling data from the phase 3 TEN01 study supported administration of pdFX 25 IU/kg twice weekly for routine prophylaxis in adolescents/adults (aged ≥12 years). Results from nine paediatric patients in the phase 3 TEN02 study and eight adolescents/adults (aged ≥12 years) in the retrospective data‐collection TEN05 study, along with real‐world evidence, showed that routine prophylaxis with pdFX ≈40 IU/kg twice weekly in patients aged <12 years and pdFX ≈25 IU/kg twice weekly in patients aged ≥12 years was effective in bleeding prevention.

Conclusions:

pdFX was well tolerated in clinical studies, with no new safety signals identified during routine prophylactic use. Based on current evidence, it is recommended that routine prophylaxis with pdFX be initiated at 25 IU/kg twice weekly in adults/adolescents ≥12 years of age, and at a dosage of 40 IU/kg twice weekly in children <12 years of age. Thereafter, FX levels should be closely monitored, and dosages should be adjusted according to clinical response and to maintain trough levels ≥5 IU/dl.

01 Jan 2022·SAGE open medical case reports

A case report of congenital factor X deficiency in an adult patient

Author: Aljarah, Nasser ; AL-Ansari, Rehab Y ; Woodman, Alexander ; Alofi, Ghufran

Factor X deficiency is one of the rarest coagulation disorders representing 10% of all rare bleeding diseases with a frequency of 1:1,000,000. A 39-year-old male patient with no previous medical conditions was admitted to the hospital with a left carpal ganglion for surgical excision. Routine preoperative laboratory examination revealed a high international normalized ratio of 5.4 IU (0.8–1.1) and a prothrombin time of 72.2 s (10.9–13.6), with an isolated factor X level of less than 5%. Genetic testing for congenital factor X deficiency identified a homozygous mutation c.271 > A (p.Glu91). Vitamin K supplementation did not improve his international normalized ratio or increase factor X levels; hence, surgery was delayed. The patient was re-hospitalized to remove a wisdom tooth, during which fresh frozen plasma was administered. An allergic reaction complicated this procedure in the form of a rash on the body. As a result, the tooth was removed without active bleeding. This report presents a unique factor X deficiency case with limited treatment options to improve factor X levels after failed vitamin K administration and an allergic reaction to fresh frozen plasma. A physician’s observation and ongoing follow-up were the only reasonable approaches in treating the patient with mild to moderate factor X deficiency due to lack of prothrombin complex concentrates or factor X replacement at the center at the time.

01 Jul 2021·Research and practice in thrombosis and haemostasis

Plasma‐derived factor X concentrate compassionate use for hereditary factor X deficiency: Long‐term safety and efficacy in a retrospective data‐collection study

Article

Author: Akanezi, Chioma ; Huang, James N ; Kavakli, Kaan ; Austin, Steven K ; Liesner, Ri

BACKGROUND:

Coagadex is a high-purity plasma-derived factor X concentrate (pdFX) developed to treat hereditary factor X deficiency (FXD).

OBJECTIVE:

Evaluate the efficacy and safety of pdFX administered to patients with hereditary FXD.

METHODS:

This was an open-label, multicenter, retrospective analysis of patients receiving pdFX for compassionate use. Efficacy end points included treatments administered, the number and treatment of bleeds, and investigator assessments. Adverse drug reactions (ADRs) were monitored.

RESULTS:

Fifteen patients were included: seven received routine prophylaxis, seven received on-demand treatment, and one alternated. Most were aged ≥12 years (n = 13) and had severe hereditary FXD (n = 12). The median follow-up time was 19.2 months (range, 3.5-48.8). The number of infusions per patient per month was higher for the routine prophylaxis group (median [range], 5.4 [1.4-10.1]) than for the on-demand group (0.8 [0.1-2.3]), as was the dose per infusion (27.9 [21.9-53.6] IU/kg vs 20.0 [13.6-27.7] IU/kg). Patients experienced 88 bleeds (34 minor, 7 major, 47 unclassified). The monthly bleed rate per patient was 0.04 in the routine prophylaxis group (based on 17 bleeds in four patients) and 0.8 in the on-demand group (based on 71 bleeds in eight patients). pdFX was used to treat 79 bleeds and was rated effective in all instances. In an overall assessment, investigators rated pdFX as excellent for 14 patients (93.3%) and good for 1 patient (6.3%). No ADRs or safety concerns were reported.

CONCLUSIONS:

This analysis supports the use of pdFX as a safe, effective treatment for hereditary FXD. Routine prophylaxis with pdFX may reduce bleed frequency.

News (Medical) associated with Human Coagulation Factor X(Bio Products)

07 Jun 2016

·www.pharmatimes.com

UK launch for first hereditary factor X deficiency treatment

The first and only treatment licensed specifically for the rare bleeding disorder hereditary factor X deficiency has now been launched across the UK. Bio Products Laboratory's Coagadex was approved in Europe in March for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with the condition. "Our previous focus has been on treating Factor X deficiency with blood infusions of plasma or a concentrate of clotting factors," noted Dr Steve Austin, Haemophilia Centre director of St George's University Hospitals NHS Foundation Trust, London, welcoming the therapy's launch. "For the first time, patients with this rare bleeding disorder can receive a specific factor X replacement that has been proven safe and effective in clinical studies." Hereditary factor X deficiency affects around 700 patients in Europe, and is characterised by a lack of factor X protein in the blood, which plays a crucial role in coagulation. People with this deficiency are at increased risk of bleeding, including inside the brain, lungs or gastrointestinal tract in the most severe cases, which can be life-threatening. The UK is the first country in Europe where Coagadex will be made available, via a Commercial Medicines Unit tender, which starts on July 1st. The CMU works on behalf of the Department of Health and the NHS, and in partnership with the people who buy pharmaceuticals for hospitals across the NHS in the UK.

Collaborate

100 Deals associated with Human Coagulation Factor X(Bio Products)

External Link

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-	-	B02BD13	DB13148

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Factor X Deficiency	United States	Bio Products Laboratory USA, Inc.	20 Oct 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ISTH2022	Not Applicable	Factor X Deficiency	-	Human coagulation factor X	vqnaycvqjc(sbrilcwuak) = ubauylbmde tvvezwxsaz (prieyzsvmj ) View more	-	09 Jul 2022
ISTH2019	Not Applicable	-	-	Factor X	dabvtnpnso(yqliqpokik) = hkxoppdqgv vjfwcthabk (cglltuppcz )	-	10 Jun 2019
NCT00930176 (CTgov)	Phase 3	Factor X Deficiency	16	Human Coagulation FACTOR X	pthiahtwvd(thdbuurwht) = vymialldkx ujdoucsqcf (hpgcdwtpqh, 21.01) View more	-	12 Dec 2014

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