Octapharma News - LARVOL Sigma

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|||||||||| Nuwiq (simoctocog alfa) / Octapharma, Octanate (human Factor VIII/von Willebrand Factor) / Octapharma
Clinical, Review, Journal: Personalised Prophylaxis in a Child with Haemophilia A and Type 1 Diabetes. (Pubmed Central) - Jun 3, 2021
rFVIII was well tolerated, with no treatment-related adverse events observed. This case illustrates the importance of treatment personalisation for young patients and their families managing concomitant diseases.

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma
[VIRTUAL] A CLINICAL CASE OF X-LINKED HYPER-IGM SYNDROME () - Jun 2, 2021 - Abstract #ESPID2021ESPID_1432;
Against the background of the treatment (antibiotic therapy, octagam), a slight improvement in the condition of the boy was noted, however, after a short period of relief, complaints again appeared about fever, diarrhea, aphthous stomatitis, weight loss...The CD40LG gene is associated with X-linked hyper-IgM syndrome (HIGM). Learning Points/ Since primary immunodeficiency states can manifest themselves with malabsorption syndrome, diarrhea, IBD, pediatricians should be more alert and take into account in the differential diagnosis of these conditions and the choice of treatment regimens.

|||||||||| Journal: Quantitative HLA-class-II/Factor VIII (FVIII) Peptidomic Variation in Dendritic Cells Correlates with the Immunogenic Potential of Therapeutic FVIII Proteins in Hemophilia A. (Pubmed Central) - May 15, 2021
Learning Points/ Since primary immunodeficiency states can manifest themselves with malabsorption syndrome, diarrhea, IBD, pediatricians should be more alert and take into account in the differential diagnosis of these conditions and the choice of treatment regimens. Our results provide HLAcII peptidomic level explanations for several important clinical observations/issues including the differential immunogenicity of distinct FVIIIs and the role of HLAcII genetics in inhibitor development.

|||||||||| Fibryga (fibrinogen concentrate (human)) / Octapharma
Clinical, Journal: Fibrinogen concentrate for treatment of bleeding and surgical prophylaxis in congenital fibrinogen deficiency patients. (Pubmed Central) - May 15, 2021
Our results provide HLAcII peptidomic level explanations for several important clinical observations/issues including the differential immunogenicity of distinct FVIIIs and the role of HLAcII genetics in inhibitor development. HFC was efficacious for on-demand treatment of bleeding and as surgical prophylaxis, with a favorable safety profile, in patients with congenital afibrinogenemia.

||||||||||  Fibryga (fibrinogen concentrate (human)) / Octapharma
Clinical data, Trial completion, Real-world evidence, Real-world:  Use of Fibryga, a Fibrinogen Concentrate in Real World: Retrospective Collection of Clinical Data (clinicaltrials.gov) -  May 13, 2021
P=N/A, N=200, Completed,  Sponsor: Octapharma
HFC was efficacious for on-demand treatment of bleeding and as surgical prophylaxis, with a favorable safety profile, in patients with congenital afibrinogenemia. Recruiting --> Completed

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma, Zarzio (filgrastim biosimilar) / Novartis
[VIRTUAL] PROSID STUDY: EVALUATING EFFICACY AND SAFETY OF INTRAVENOUS IMMUNOGLOBULIN (IVIG) 10% IN PRIMARY INFECTION PROPHYLAXIS IN PATIENTS WITH CHRONIC LYMPHOCYTIC LEUKEMIA – STUDY DESIGN () - May 13, 2021 - Abstract #EHA2021EHA_1282;
Results Interim analysis will be conducted once at least 100 patients are available for evaluation of the primary endpoint in the ITT population). Conclusion This study will evaluate the efficacy and safety of IVIg 10% as primary prophylaxis of infections inCLL patients with SID in a large clinical setting for the first time.

|||||||||| Kcentra (plasma-derived prothrombin complex concentrate) / CSL Behring, Octaplex (human prothrombin complex concentrate) / Octapharma, Pfizer
[VIRTUAL] Reversal of anticoagulation with PCC – every minute counts () - May 12, 2021 - Abstract #BSH2021BSH_467;
Lack of knowledge about PCC by staff treating these emergency cases and delays caused by patient transfers are avoidable. Every effort must be made to improve local systems and reduce the ‘door to needle’ time for administering PCC in these patients.

||||||||||  Gammanorm (human immune globulin subcutaneous) / Octapharma
Trial completion date, Trial primary completion date:  Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases (clinicaltrials.gov) -  May 12, 2021
P3, N=65, Recruiting,  Sponsor: Octapharma
Every effort must be made to improve local systems and reduce the ‘door to needle’ time for administering PCC in these patients. Trial completion date: Mar 2021 --> Jul 2021 | Trial primary completion date: Mar 2021 --> Jul 2021

||||||||||  Octaplex (human prothrombin complex concentrate) / Octapharma, Pfizer
New P3 trial:  Study of OCTAPLEX in Patients With Acute Major Bleeding on DOAC Therapy With Factor Xa Inhibitor (clinicaltrials.gov) -  Apr 29, 2021
P3, N=200, Not yet recruiting,  Sponsor: Octapharma

||||||||||  Hemlibra (emicizumab-kxwh) / Roche, Nuwiq (simoctocog alfa) / Octapharma
Trial completion date, Trial primary completion date:  The Atlanta Study: Emicizumab PUPs and Nuwiq ITI Study (clinicaltrials.gov) -  Apr 28, 2021
P3, N=60, Not yet recruiting,  Sponsor: Emory University
Trial completion date: Mar 2021 --> Jul 2021 | Trial primary completion date: Mar 2021 --> Jul 2021 Trial completion date: Oct 2024 --> Apr 2025 | Trial primary completion date: Oct 2024 --> Apr 2025

|||||||||| Gammanorm (human immune globulin subcutaneous) / Octapharma
[VIRTUAL] Medium-to-Long-Term Safety, Efficacy, and Tolerability of Subcutaneous Human Immunoglobulin 16.5% in Patients with Primary Immunodeficiencies () - Apr 23, 2021 - Abstract #CIS2021CIS_240;
PID patients received weekly or bi-weekly doses of SCIg 16.5% (Cutaquig) with the option of increased infusion volume (up to 60 mL/site) and rate (up to 240 mL/hr/all sites) over a 42-month period...The product was well tolerated, with 96.6% of all infusions having no infusion site reactions. This study demonstrated that subcutaneous human normal immunoglobulin 16.5% is well-tolerated, safe and effective for long-term use in patients with primary immunodeficiency diseases.

|||||||||| Gammanorm (human immune globulin subcutaneous) / Octapharma
[VIRTUAL] Efficacy and safety of administration of cutaquig (16.5% IGSC) in pediatric patients with primary immunodeficiencies (PI): Data from two phase 3 studies () - Apr 23, 2021 - Abstract #CIS2021CIS_138;
P3
Infusion site reactions were reported in 25 (66%) pediatric patients during the pivotal study and in 7 (70%) in the extension study (all except one of mild/moderate intensity). Data from both studies demonstrate similar efficacy and safety of cutaquig administration in pediatric patients as in adults.

|||||||||| Fibryga (fibrinogen concentrate (human)) / Octapharma
[VIRTUAL] FIBRINOGEN CONCENTRATE IN PEDIATRIC AND ADOLESCENT PATIENTS WITH CONGENITAL FIBRINOGEN DEFICIENCY () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_230;
Hemostatic efficacy of HFC was comparable for adolescent and pediatric patients, with a favorable safety profile. Studies funded by Octapharma.

|||||||||| Fibryga (fibrinogen concentrate (human)) / Octapharma
[VIRTUAL] FIBRINOGEN CONCENTRATE FOR SURGICAL PROPHYLAXIS IN PATIENTS WITH CONGENITAL FIBRINOGEN DEFICIENCY () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_229;
In conclusion, HFC administration for bleeding prophylaxis during surgery was efficacious for this ultra-rare disease in adult, adolescent and pediatric patients with CFD. Studies funded by Octapharma.

|||||||||| Fibryga (fibrinogen concentrate (human)) / Octapharma
[VIRTUAL] FIBRINOGEN CONCENTRATE PHARMACOKINETICS AND DOSING IN PATIENTS WITH CONGENITAL FIBRINOGEN DEFICIENCY () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_227;
Pediatric patients received a numerically higher HFC dose compared to adult/adolescent patients. Despite differences in PK and dosing, HFC was efficacious for on-demand BE treatment and surgical prophylaxis in CFD patients across age groups.

|||||||||| Gammanorm (human immune globulin subcutaneous) / Octapharma
[VIRTUAL] LONG-TERM EFFICACY AND TOLERABILITY OF SUBCUTANEOUS IMMUNOGLOBULIN (GAMMANORM) IN INFLAMMATORY MYOPATHIES: A 5-YEAR FOLLOW-UP IN 30 PATIENTS (HALL E) - Apr 11, 2021 - Abstract #AUTO2021AUTO_352;
No serious adverse event was reported.ConclusionsThese results suggest that the use of high-dose SCIg is feasible, beneficial and safe for long-term treatment of patients with inflammatory myopathies. Thereby, SCIg represents an alternative to IVIg, in particularly in patients with difficult venous access or insufficient response, and/or in patients preferring home care setting.

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma
@odile_launay bjr Mme, j'ai lu l'article sur immunodep., Je le suis et en traitement mensuel immuno, Octagam, g fait les 2 doses Pfizer, sero 1 mois après et taux AC à 385 UA/ml, c une bonne réponse? Merci d'avance (Twitter) - Apr 9, 2021

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma
Retrospective data, Journal: Effects of adjunct treatment with intravenous Octagam on the course of severe COVID-19: results from a retrospective cohort study. (Pubmed Central) - Apr 7, 2021
Thereby, SCIg represents an alternative to IVIg, in particularly in patients with difficult venous access or insufficient response, and/or in patients preferring home care setting. To evaluate the effect of adjunct treatment with Octagam, an intravenous immunoglobulin (IVIG) product, on clinical outcomes and biomarkers in critically ill COVID-19 patients...Clinically relevant benefits through adjunct Octagam treatment in COVID-19 need to be confirmed in a randomized, controlled trial.

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma
[VIRTUAL] A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE III TRIAL OF IVIG 10% IN PATIENTS WITH DERMATOMYOSITIS. THE PRODERM STUDY: RESULTS ON EFFICACY AND SAFETY (Room 1) - Apr 6, 2021 - Abstract #EULAR2021EULAR_1148;
To evaluate the effect of adjunct treatment with Octagam, an intravenous immunoglobulin (IVIG) product, on clinical outcomes and biomarkers in critically ill COVID-19 patients...Clinically relevant benefits through adjunct Octagam treatment in COVID-19 need to be confirmed in a randomized, controlled trial. This is the first large international phase III randomized, placebo-controlled trial demonstrating the efficacy and safety of IVIg as a treatment for patients with DM.

||||||||||  Octaplex (human prothrombin complex concentrate) / Octapharma, Pfizer, Fibryga (fibrinogen concentrate (human)) / Octapharma
Enrollment open, Head-to-Head:  FiiRST-2: Factor In the Initial Resuscitation of Severe Trauma 2 Patients (clinicaltrials.gov) -  Apr 4, 2021
P4, N=350, Recruiting,  Sponsor: University Health Network, Toronto
This is the first large international phase III randomized, placebo-controlled trial demonstrating the efficacy and safety of IVIg as a treatment for patients with DM. Not yet recruiting --> Recruiting

|||||||||| Nuwiq (simoctocog alfa) / Octapharma
Clinical, PK/PD data, Journal: Efficacy of Nuwiq (Simoctocog Alfa) in Patients with Hemophilia A Who Changed and Adhered to a Pharmacokinetic-Guided Prophylaxis Regimen in the NuPreviq Study. (Pubmed Central) - Mar 16, 2021
Not yet recruiting --> Recruiting No abstract available

||||||||||  Gammanorm (human immune globulin subcutaneous) / Octapharma
Trial termination:  Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases (clinicaltrials.gov) -  Mar 10, 2021
P=N/A, N=60, Terminated,  Sponsor: Octapharma
No abstract available Recruiting --> Terminated

||||||||||  Octagam (intravenous normal human immunoglobulin) / Octapharma
Trial completion:  Octagam 10% Therapy in COVID-19 Patients With Severe Disease Progression (clinicaltrials.gov) -  Mar 10, 2021
P3, N=208, Completed,  Sponsor: Octapharma
Recruiting --> Terminated Active, not recruiting --> Completed

||||||||||  Fibryga (fibrinogen concentrate (human)) / Octapharma
Enrollment open, Trial completion date, Trial primary completion date:  Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency (clinicaltrials.gov) -  Mar 5, 2021
P=N/A, N=25, Recruiting,  Sponsor: Octapharma
Active, not recruiting --> Completed Not yet recruiting --> Recruiting | Trial completion date: Jan 2024 --> Oct 2027 | Trial primary completion date: Jan 2024 --> Oct 2027

||||||||||  Atenativ (human antithrombin III) / Octapharma
Trial completion date, Trial primary completion date:  Observational, Retrospective Study to Evaluate Coagulation Changes and the Influence of Antithrombin III Treatment in Patients With Severe COVID-19 Infection (clinicaltrials.gov) -  Feb 26, 2021
P=N/A, N=300, Recruiting,  Sponsor: Octapharma
Not yet recruiting --> Recruiting | Trial completion date: Jan 2024 --> Oct 2027 | Trial primary completion date: Jan 2024 --> Oct 2027 Trial completion date: Jan 2021 --> Jun 2021 | Trial primary completion date: Jan 2021 --> Jun 2021

|||||||||| Panzyga (human immune globulin intravenous 10% stabilised with glycine) / Octapharma
FDA event: Pfizer's PANZYGA Wins FDA Approval For Rare Peripheral Nerves Disorder $PFE @Pharmdca @BioStocks @bioILLOGICAL (Twitter) - Feb 16, 2021

||||||||||  Wilate (human factor VIII/von Willebrand factor) / Octapharma, Nuwiq (simoctocog alfa) / Octapharma
Trial completion date, Trial termination, Trial primary completion date:  PREVAIL: Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq (clinicaltrials.gov) -  Feb 15, 2021
P=N/A, N=80, Terminated,  Sponsor: Octapharma
Trial completion date: Jan 2021 --> Jun 2021 | Trial primary completion date: Jan 2021 --> Jun 2021 Trial completion date: Dec 2027 --> Nov 2020 | Recruiting --> Terminated | Trial primary completion date: Dec 2027 --> Nov 2020; Study terminated as enrolled patients rolled over into a large international study in the same indication

||||||||||  Gammanorm (human immune globulin subcutaneous) / Octapharma
[VIRTUAL] Real-World Evaluation of Immune Globulin Subcutaneous 16.5% in the Treatment of Primary Immunodeficiency () -  Feb 3, 2021 - Abstract #AAAAI2021AAAAI_989;
We report efficacy, safety, and tolerability of IGSC 16.5% (Cutaquig®) in a real-world setting.  Methods We conducted a retrospective review of patients receiving IGSC 16.5% from product availability through 7/2020...Overall, 35 (39%) patients experienced 53 mild to moderate bacterial respiratory tract infections, predominantly sinus infections. Three were serious bacterial infections.  Conclusions These data demonstrate IGSC 16.5% for PID is effective and well-tolerated in a real-world setting.

||||||||||  Gammanorm (human immune globulin subcutaneous) / Octapharma
[VIRTUAL] Long-Term Safety, Efficacy, and Tolerability of Subcutaneous Human Immunoglobulin 16.5% in Patients with Primary Immunodeficiencies () -  Feb 3, 2021 - Abstract #AAAAI2021AAAAI_897;
Patients received weekly or bi-weekly doses of SCIg 16.5% (Cutaquig) with the option of increased infusion volume (up to 60 mL/site) and rate (up to 240 mL/hr/all sites) over a 42-month period.  Results Twenty-seven patients received a mean dose of 0.169 g/kg overall (range among different age groups: 0.127 – 0.166 g/kg)...44.4% of patients experienced infusion site reactions, with most being mild-to-moderate in intensity. Serum IgG trough levels remained relatively constant (> 5g/L) throughout the study.  Conclusions This study demonstrated that subcutaneous human normal immunoglobulin 16.5% is well-tolerated, safe and effective for long-term use in patients with primary immunodeficiency diseases.

||||||||||  Octagam (intravenous normal human immunoglobulin) / Octapharma
[VIRTUAL] Intravenous Immunoglobulin for Treatment of Severe Coronavirus Disease 19 (COVID-19) – A Literature Overview () -  Feb 3, 2021 - Abstract #AAAAI2021AAAAI_886;
An observational study conducted in 10 COVID-19 patients demonstrated short-term moderate-dose corticosteroid combined with high-dose IVIg reversed severe, deteriorating COVID-19 patients who failed initial low-dose therapy. Three published papers were identified using IVIg in pediatric COVID-19 patients who presented with Kawasaki-like or PMIS (Pediatric COVID-associated Multi-system Inflammatory Syndrome).  Conclusions It may be useful to consider high-dose IVIg at the time of initiation of respiratory distress to potentially promote satisfactory clinical recovery and reduce burden of care for COVID-19 patients.

||||||||||  Gammanorm (human immune globulin subcutaneous) / Octapharma
[VIRTUAL] Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Human Immunoglobulin 16.5% Administered at Modified Dosing Regimens in Patients with Primary Immunodeficiency Diseases – Study Design () -  Feb 3, 2021 - Abstract #AAAAI2021AAAAI_819;
Sixty-five adult and pediatric patients with a confirmed diagnosis of PID currently on a stable dose of SCIG treatment will be enrolled and assigned to 1 of 3 cohorts to receive doses of SCIg 16.5% (Cutaquig) over a 32-week period (Cohort 1: increased volume at each infusion site (up to 60 mL/site); Cohort 2: increased infusion rate (up to 240 mL/hr/all sites); Cohort 3: every other week dosing).  Results The co-primary objectives are to compare total IgG trough levels achieved by weekly infusions to every other week infusions, to assess safety and tolerability of SCIg 16.5% being administered according to 3 different infusion parameters, and to assess efficacy parameters when switching from weekly infusions to every other week infusions. Secondary objectives will evaluate quality of life, along with additional evaluations of safety and efficacy of SCIg 16.5% administration.  Conclusions The investigators of this study hope to provide modified infusion regimens to offer patients dosing flexibility to accommodate their personal schedules or reduce the number of injection

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma
Clinical, Observational data, Retrospective data, Review, Clinical Trial,Phase III, Journal: Use of Intravenous Immunoglobulin (Prevagen or Octagam) for the Treatment of COVID-19: Retrospective Case Series. (Pubmed Central) - Jan 29, 2021
No thrombotic events occurred and IVIG was well tolerated, despite most cases demonstrating very elevated D-dimer suggestive of active intravascular fibrinolysis. We believe that IVIG warrants immediate clinical trial evaluation in COVID-19 to confirm its role as a mainstay treatment of moderate to severe COVID-19 infection as a means to reduce hospital stay and utilization of ICU resources, including mechanical ventilation, and potentially reduce mortality.

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma
Clinical, P3 data, Journal: Prospective, double-blind, randomized, placebo-controlled phase III study evaluating efficacy and safety of octagam 10% in patients with dermatomyositis ("ProDERM Study"). (Pubmed Central) - Jan 26, 2021
P3
The ProDERM study was the first to assess the long-term efficacy and safety of IVIg (Octagam 10%) in a placebo-controlled, blinded, randomized trial in DM. The study aimed to inform on the use of IVIg in the treatment of DM, and results are expected in Q3 2020.

||||||||||  Wilate (human factor VIII/von Willebrand factor) / Octapharma, Nuwiq (simoctocog alfa) / Octapharma, Octanate (human Factor VIII/von Willebrand Factor) / Octapharma
Trial completion date, Trial primary completion date:  Protect-NOW: Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients (clinicaltrials.gov) -  Jan 25, 2021
P=N/A, N=140, Recruiting,  Sponsor: Octapharma
The study aimed to inform on the use of IVIg in the treatment of DM, and results are expected in Q3 2020. Trial completion date: Jun 2022 --> Jun 2023 | Trial primary completion date: Jun 2022 --> Jun 2023

||||||||||  simoctocog alpha/von Willebrand Factor (OCTA101) / Octapharma
Enrollment open, Trial completion date, Trial primary completion date:  Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A (clinicaltrials.gov) -  Jan 22, 2021
P1/2, N=36, Recruiting,  Sponsor: Octapharma
Trial completion date: Jun 2022 --> Jun 2023 | Trial primary completion date: Jun 2022 --> Jun 2023 Active, not recruiting --> Recruiting | Trial completion date: Sep 2021 --> Sep 2022 | Trial primary completion date: Sep 2021 --> Sep 2022

||||||||||  Octagam (intravenous normal human immunoglobulin) / Octapharma
Enrollment closed:  Octagam 10% Therapy in COVID-19 Patients With Severe Disease Progression (clinicaltrials.gov) -  Jan 13, 2021
P3, N=208, Active, not recruiting,  Sponsor: Octapharma
Active, not recruiting --> Recruiting | Trial completion date: Sep 2021 --> Sep 2022 | Trial primary completion date: Sep 2021 --> Sep 2022 Recruiting --> Active, not recruiting

|||||||||| Nuwiq (simoctocog alfa) / Octapharma
[VIRTUAL] A family journey with Nuwiq early treatment decisions for long-term health () - Jan 8, 2021 - Abstract #EAHAD2021EAHAD_461;
Recruiting --> Active, not recruiting Sponsored by OCTAPHARMA

|||||||||| Fibryga (fibrinogen concentrate (human)) / Octapharma
[VIRTUAL] EFFICACY AND SAFETY OF HUMAN FIBRINOGEN CONCENTRATE IN PATIENTS WITH CONGENITAL FIBRINOGEN DEFICIENCY: RESULTS OF THE FORMA‐02 AND FORMA‐04 TRIALS () - Jan 8, 2021 - Abstract #EAHAD2021EAHAD_87;
Data from these two efficacy and safety trials indicate that HFC was effective for on‐demand BE treatment and perioperative prophylaxis in CFD patients. Efficacy was comparable for adult, adolescent and paediatric patients, with a favourable safety profile.

|||||||||| [VIRTUAL] VWF‐CONTAINING FVIII CONCENTRATES IMPROVE FVIII RECOVERY IN A MOUSE MODEL OF SEVERE HAEMOPHILIA WITH INHIBITORS () - Jan 8, 2021 - Abstract #EAHAD2021EAHAD_30;
In agreement to previously reported data, native pdFVIII/VWF concentrate was more efficient in the restoration of FVIII circulating levels in FVIIInull E16 KO mice even in the presence of high inhibitor titer. This finding is similar for the different isolated FVIII concentrates analyzed, regardless their origin, molecular form, cell line or involved modifications.

|||||||||| [VIRTUAL] PHARMACOKINETIC DIFFERENCES IN HEMOPHILIA A BASED ON FVIII PRODUCT AND ON ASSAY TYPE () - Jan 8, 2021 - Abstract #EAHAD2021EAHAD_16;
It is still uncertain whether “Elocta” correlates with higher FVIII levels on CSA and if this fact was relevant for our statistically significant results in t1/2 of EHL in CSA. Interestingly, plasma‐derived product presented higher t1/2 than recombinant products, even though n.s. Although the number of studied patients was low to draft definitive conclusions, making comparison analysis between products more difficult, we observed minimal FVIII measurement discrepancies among OSA and CSA.

|||||||||| [VIRTUAL] IN‐HOUSE VALIDATION OF FACTOR VIII ACTIVITY FOR STANDARD AND EXTENDED HALF‐LIFE PRODUCTS AND CORRELATION TO THROMBIN GENERATION ASSAYS () - Jan 8, 2021 - Abstract #EAHAD2021EAHAD_11;
The CSA‐1 method gave the least variability compared to the assigned values. In addition, thrombin generation using spiked samples gave excellent correlation to CSA factor activity and identified a thrombin generation method with greater sensitivity towards FVIII in terms of dose response in lag time.

|||||||||| bortezomib / Generic mfg., dexamethasone / Generic mfg.
[VIRTUAL] A case of acquired von Willebrand syndrome becoming IVIg responsive following bortezomib treatment despite minimal paraprotein response () - Jan 5, 2021 - Abstract #BSHI2020BSH-I_22;
Alternatively, the M‐band may have been a ‘bystander’ paraprotein as is seen in many autoimmune conditions as a result of immune dysregulation. This case also suggest bortezomib may be effective even in aVWS cases without a measurable paraprotein.

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma, Kiovig (human normal immunoglobulin 10%) / Takeda
[VIRTUAL] Evaluation of dosing strategies for intravenous immunoglobulin to reduce total immunoglobulin dose and improve cost savings in a community hospital setting () - Dec 26, 2020 - Abstract #ASHP2020ASHP_4627;
For critically ill patients whose clinical status depends on volume status, this cost savings can also provide a volume sparing effect to improve outcomes. Additionally, IVIG cost savings can reduce the need for IVIG rationing in times of drug shortages, which may especially be harmful for patients who need frequent administration of IVIG.

||||||||||  Octaplex (human prothrombin complex concentrate) / Octapharma, Pfizer, Fibryga (fibrinogen concentrate (human)) / Octapharma
Trial completion date, Trial initiation date, Trial primary completion date, Head-to-Head:  FiiRST-2: Factor In the Initial Resuscitation of Severe Trauma 2 Patients (clinicaltrials.gov) -  Dec 19, 2020
P4, N=350, Not yet recruiting,  Sponsor: University Health Network, Toronto
Additionally, IVIG cost savings can reduce the need for IVIG rationing in times of drug shortages, which may especially be harmful for patients who need frequent administration of IVIG. Trial completion date: Jun 2023 --> Jan 2024 | Initiation date: Oct 2020 --> Jan 2021 | Trial primary completion date: May 2023 --> Dec 2023

|||||||||| Octagam (intravenous normal human immunoglobulin) / Octapharma
Journal: Reduction of Amyloid in the Brain and Retina After Treatment With IVIG for Mild Cognitive Impairment. (Pubmed Central) - Dec 18, 2020
All were administered IVIG (Octagam 10%) at 0.4 g/kg every 14 days for a total of 5 infusions...Three subjects had a reduction in amyloid SUVR and all 5 subjects had a reduction in amyloid retinal deposits in at least 1 eye. A short course of IVIG over 2 months removes a measurable amount of amyloid from the CNS in persons with MCI.

|||||||||| Fibryga (fibrinogen concentrate (human)) / Octapharma
[VIRTUAL] Human Fibrinogen Concentrate for Bleeding Prophylaxis During Surgery in Paediatric Patients with Congenital Fibrinogen Deficiency () - Dec 18, 2020 - Abstract #GTH2021GTH_239;
Efficacy of HFC (Fibryga® Octapharma) in surgical prophylaxis was assessed at the end of surgery by the surgeon and post-operatively by the haematologist using a four-point objective scale (Excellent, Good, Moderate, None), and by an Independent Data Monitoring & Endpoint Adjudication Committee (IDMEAC)...No allergic/hypersensitivity reactions or deaths were observed. Conclusion HFC administration for bleeding prophylaxis during surgery was efficacious for this ultra-rare disease in a paediatric population with congenital afibrinogenemia and showed a favourable safety profile.

|||||||||| Fibryga (fibrinogen concentrate (human)) / Octapharma
[VIRTUAL] Efficacy and Safety of Human Fibrinogen Concentrate in Patients with Congenital Fibrinogen Deficiency: Combined Results of the FORMA-02 and FORMA-04 Clinical Trials () - Dec 18, 2020 - Abstract #GTH2021GTH_144;
Methods FORMA-02 and FORMA-04 were multicentre, prospective, open-label Phase 3 studies of the efficacy and safety of HFC (Fibryga®, Octapharma) in adult/adolescent and paediatric patients with CFD...Conclusion Across two Phase 3 clinical trials HFC was efficacious for on-demand treatment of BEs and perioperative prophylaxis in patients with CFD. Efficacy of HFC was comparable for adult, adolescent and paediatric patients, with a favourable safety profile.

|||||||||| Enrollment change, Trial withdrawal: RESIST NAIVE: Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive (clinicaltrials.gov) - Dec 17, 2020
P=N/A, N=0, Withdrawn, Sponsor: City of Hope Medical Center
Efficacy of HFC was comparable for adult, adolescent and paediatric patients, with a favourable safety profile. N=148 --> 0 | Active, not recruiting --> Withdrawn

|||||||||| Trial completion, Trial completion date, Trial primary completion date: RESIST EXP: Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced) (clinicaltrials.gov) - Dec 17, 2020
P=N/A, N=3, Completed, Sponsor: City of Hope Medical Center
N=148 --> 0 | Active, not recruiting --> Withdrawn Active, not recruiting --> Completed | Trial completion date: Jun 2020 --> Oct 2020 | Trial primary completion date: Jun 2020 --> Oct 2020

||||||||||  ASP7317 / Octapharma, CHA Bio & Diostech, Astellas
Enrollment change, Trial completion date, Trial primary completion date:  A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration (clinicaltrials.gov) -  Dec 10, 2020
P1b, N=18, Active, not recruiting,  Sponsor: Astellas Institute for Regenerative Medicine
Active, not recruiting --> Completed | Trial completion date: Jun 2020 --> Oct 2020 | Trial primary completion date: Jun 2020 --> Oct 2020 N=150 --> 18 | Trial completion date: Jan 2027 --> Nov 2022 | Trial primary completion date: Jan 2027 --> Nov 2022



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