A5102949061- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Immunodeficiency and Autoimmune Disorders
- Peripheral Neuropathies and Disorders
- Platelet Disorders and Treatments
- Hemophilia Treatment and Research
- Hereditary Neurological Disorders
- Blood groups and transfusion
- Chronic Myeloid Leukemia Treatments
- Autoimmune Bullous Skin Diseases
- Myasthenia Gravis and Thymoma
- Peripheral Nerve Disorders
- Blood disorders and treatments
- Biosimilars and Bioanalytical Methods
- Pneumocystis jirovecii pneumonia detection and treatment
- Chronic Lymphocytic Leukemia Research
- PI3K/AKT/mTOR signaling in cancer
- Mast cells and histamine
- Interdisciplinary Research and Collaboration
- Monoclonal and Polyclonal Antibodies Research
- Genetics, Bioinformatics, and Biomedical Research
- Horticultural and Viticultural Research
- Statistical Methods in Clinical Trials
- Vitamin K Research Studies
- Health Systems, Economic Evaluations, Quality of Life
- Pharmaceutical studies and practices
Behringwerke (Germany)
2013-2024
Baxter (Austria)
2010-2014
CSL (Switzerland)
2013
Hereditary angioedema is a disabling, potentially fatal condition caused by deficiency (type I) or dysfunction II) of the C1 inhibitor protein. In phase 2 trial, use CSL830, nanofiltered preparation that suitable for subcutaneous injection, resulted in functional levels activity would be expected to provide effective prophylaxis attacks.We conducted an international, prospective, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, 3 trial evaluate efficacy and safety...
Hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) impairs health-related quality of life (HRQoL).The objective this study was to assess HRQoL outcomes in patients self-administering subcutaneous C1-INH (C1-INH[SC]; HAEGARDA) for routine prevention HAE attacks.Post hoc analysis data from the placebo-controlled, crossover phase III COMPACT (Clinical Studies Optimal Management Preventing Angioedema Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy). Ninety C1-INH-HAE were...
Hereditary angioedema (HAE) is a chronic, unpredictable disease. Long-term prophylactic treatments that offer durable efficacy, safety, and convenience are required to assist patients in achieving complete disease control, per international guidelines. We report an interim analysis of ongoing phase 3 (VANGUARD) open-label extension (OLE) study evaluating the long-term safety efficacy garadacimab for HAE prophylaxis. Adults adolescents aged ≥12 years with previously participating 2 pivotal...
Development of neutralizing antibodies remains the most problematic complication in treating congenital hemophilia. Control and prevention bleeding events such patients with recombinant factor VIIa (rFVIIa) is limited by short half-life available product. Here, we report on pharmacokinetics safety a novel, fusion protein linking coagulation FVIIa albumin (rVIIa-FP) first-in-human study healthy male subjects.Forty subjects between 18 35 years age were included dosed five consecutive cohorts....
Abstract Background Long-term prophylaxis with subcutaneous C1-inhibitor (C1-INH[SC]; HAEGARDA, CSL Behring) in patients hereditary angioedema (HAE) due to C1-INH deficiency (C1-INH-HAE) was evaluated an open-label extension follow-up study the international, double-blind, placebo-controlled COMPACT study. The current analysis patient-reported health-related quality of life (HRQoL) data from 126 randomized treatment C1-INH(SC) 40 IU/kg (n = 63) or 60 twice weekly for 52 weeks. HRQoL at...
Intravenous (IVIG) and subcutaneous (SCIG) immunoglobulin infusions are widely used for the treatment of patients with primary immunodeficiency (PID) worldwide. This prospective, multicenter, open-label, single-arm Phase III study evaluated efficacy, tolerability, safety IgPro20 (Hizentra®; L-proline-stabilized 20 % human SCIG) in adult pediatric Japanese PID.Patients received three IVIG at 3-4-week intervals followed by a dose-equivalent switch to weekly SCIG infusions. A 12-week...
Hizentra(®) (20% subcutaneous immunoglobulin [SCIG]) was administered to subjects with primary immunodeficiency disease in two extension studies the EU and US assess long-term efficacy tolerability. Subjects (aged 4-69 years) were treated for 148 weeks (N = 40; 5405 infusions) 87 21; 1735 infusions). Weekly doses 116.0 mg/kg (EU) 193.2 (US); IgG levels 7.97 g/L 11.98 (US). Annualized rates of serious bacterial infections 0.05 infections/subject/year 0.06 Rates any infection 3.33 2.38 The...
For prophylaxis of hereditary angioedema (HAE) attacks, replacement therapy with human C1-inhibitor (C1-INH) treatment is approved and available as intravenous [C1-INH(IV)] (Cinryze®) subcutaneous [C1-INH(SC)] HAEGARDA® preparations. In the absence a head-to-head comparative study two modalities, an indirect comparison data from 2 independent but similar clinical trials was undertaken. Two randomized, double-blind, placebo-controlled, crossover studies were identified which evaluated either...
Chronic inflammatory demyelinating polyneuropathy (CIDP) causes weakness which adversely impacts function and quality of life (QOL). CIDP often requires long-term management with intravenous or subcutaneous immunoglobulin. The Polyneuropathy Treatment Hizentra® (PATH) study showed that immunoglobulin (SCIG) was efficacious in maintenance. Here, patient-reported outcomes patients on SCIG are assessed.Subjects stabilized were randomly allocated to receive weekly 0.2 0.4 g/kg bodyweight 20%...
Nonclinical in vivo animal studies have to be completed before starting clinical of the pharmacokinetic behavior a drug humans. The exposure is often measured by area under concentration versus time curve (AUC). classical complete data design where each sampled for analysis at every point applicable large animals only. In case small animals, blood sampling restricted, batch or serial need considered. designs, samples are taken more than once from animal, but not all points. only one sample...
To assess electrophysiology parameters that can reflect patients' clinical status and show changes in nerve function with treatment, a study of subcutaneous immunoglobulin chronic inflammatory demyelinating polyneuropathy.Nerve conduction studies (latency, velocity, block compound muscle action potential [CMAP] on upper limb median, ulnar, lower peroneal motor nerves) were conducted the placebo-controlled PATH (Polyneuropathy And Treatment Hizentra) two doses maintenance (SCIG) IgPro20...
Primary (PID) and secondary immune deficiencies (SID) represent diverse groups of diagnoses, yet both can be effectively treated with intravenous immunoglobulin (IVIG) replacement therapy. Guidelines for the use IVIG in SID vary due to paucity data. The objective was analyze available Privigen® (IgPro10, CSL Behring, Bern, Switzerland) data on Efficiency Index (EI) pharmacokinetic (PK) parameters patients PID SID.Three studies (NCT00168025, NCT00322556, observational study IgPro10_5001) were...
To assess axonal function prior to subcutaneous immunoglobulin (SCIG) therapy or placebo in relation relapse chronic inflammatory demyelinating polyneuropathy (CIDP) determine whether damage can predict response.Relapse rates patients from the Polyneuropathy and Treatment with Hizentra (PATH) study, where were treated SCIG (IgPro20), analyzed by baseline (post-intravenous stabilization) (≤1 mV peroneal compound muscle action potential) status.In non-axonal damage, relapses significantly...
BACKGROUND Hemolysis is an infrequent but recognized and potentially serious adverse effect of intravenous immunoglobulin (IVIG). Relatively elevated hemolysis reporting rates were seen with some IVIG products high anti‐A/B isoagglutinin content, among which IgPro10 (Privigen, CSL Behring). For IgPro10, two reduction measures successively implemented: 1) anti‐A donor screening 2) immunoaffinity chromatography (IAC; Ig IsoLo)–based step included in the production process. The aim this...