Pharmacokinetics, Efficacy and Safety of IMMUNATE® Solvent/Detergent (IMMUNATE® S/D) in Previously Treated Patients with Severe Hemophilia A: Results of a Prospective, Multicenter, Open-Label Phase III Study

2008 ◽  
Vol 119 (2) ◽  
pp. 89-97 ◽  
Author(s):  
L. Nemes ◽  
T. Lissitchkov ◽  
G. Dobaczewski ◽  
A. Klukowska ◽  
V. Komrska ◽  
...  
Keyword(s):  
Hemophilia A ◽  
Phase Iii ◽  
Severe Hemophilia ◽  
Efficacy And Safety ◽  
Open Label ◽  
Open Label Phase ◽  
Phase Iii Study ◽  
Previously Treated Patients ◽  
Previously Treated

scholarly journals Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program

2019 ◽  
Vol 10 ◽  
pp. 204062071985847 ◽  
Author(s):  
Toshko Lissitchkov ◽  
Anna Klukowska ◽  
John Pasi ◽  
Craig M. Kessler ◽  
Robert Klamroth ◽  
...  
Keyword(s):  
Hemophilia A ◽  
High Titer ◽  
Clinical Trial Data ◽  
Severe Hemophilia ◽  
Efficacy And Safety ◽  
Previously Treated Patients ◽  
Previously Treated ◽  
Recombinant Fviii ◽  
Actual Incidence ◽  
Human Specific

Simoctocog alfa (human-cl rhFVIII, Nuwiq®) is a 4th generation recombinant FVIII (rFVIII), without chemical modification or fusion with any other protein/fragment. Nuwiq® is produced in a human embryonic kidney cell line (HEK293F), which ensures human-specific post-translational protein processing. Nuwiq® was evaluated in seven prospective clinical studies in 201 adult and pediatric previously treated patients (PTPs) with severe hemophilia A. The NuProtect study in 110 previously untreated patients (PUPs) is ongoing. The mean half-life of Nuwiq® was 15.1–17.1 h in PTP studies with adults and adolescents, and 12.5 h in children aged 2–12 years. Clinical trials in PTPs demonstrated the efficacy and safety of Nuwiq® in the prevention and treatment of bleeds and as surgical prophylaxis. In the NuPreviq study of pharmacokinetic (PK)-guided personalized prophylaxis in 66 adult PTPs, 83% of patients had no spontaneous bleeds during 6 months of personalized prophylaxis and 57% were treated ⩽2 per week. No FVIII inhibitors were detected in PTPs after treatment with 43,267 injections and >80 million IU of Nuwiq®. Interim data for 66 PUPs with ⩾20 exposure days to Nuwiq® in NuProtect demonstrated a low cumulative high-titer inhibitor rate of 12.8% [actual incidence 12.1% (8/66)] and convincing efficacy and safety.

scholarly journals Efficacy and safety of esaxerenone (CS-3150) in Japanese patients with type 2 diabetes and macroalbuminuria: a multicenter, single-arm, open-label phase III study

2021 ◽  
Author(s):  
Sadayoshi Ito ◽  
Naoki Kashihara ◽  
Kenichi Shikata ◽  
Masaomi Nangaku ◽  
Takashi Wada ◽  
...  
Keyword(s):  
Type 2 Diabetes ◽  
Renin Angiotensin System ◽  
Japanese Patients ◽  
Phase Iii ◽  
Efficacy And Safety ◽  
Open Label ◽  
Clinical Trial Registration ◽  
Open Label Phase ◽  
Phase Iii Study

Abstract Background Esaxerenone has potential renoprotective effects and reduces the urinary albumin-to-creatinine ratio (UACR) in patients with diabetic kidney disease and overt nephropathy. We investigated the efficacy and safety of esaxerenone in Japanese patients with type 2 diabetes (T2D) and macroalbuminuria (UACR ≥ 300 mg/g creatinine). Methods We conducted a multicenter, single-arm, open-label phase III study in 56 patients with T2D and UACR ≥ 300 mg/g creatinine with estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2 and treated with a renin–angiotensin system inhibitor. Patients received esaxerenone for 28 weeks at 1.25 mg/day initially with titration to 2.5 mg/day based on serum potassium (K+) monitoring. Efficacy was evaluated as the change in UACR from baseline to week 28. Safety endpoints included adverse events (AEs), incidence of serum K+ increase, and change in eGFR from baseline. Results UACR decreased by 54.6% (95% CI 46.9%, 61.3%) on average from baseline (544.1 mg/g creatinine) to the end of treatment (246.8 mg/g creatinine); 51.8% of patients showed improvement to early nephropathy. AE incidence was 69.6%. Three patients (5.4%) had serum K+ levels ≥ 6.0 mEq/L or ≥ 5.5 mEq/L on two consecutive occasions. Hyperkalemia in two patients was transient and resolved during the treatment period. One patient discontinued following two consecutive serum K+ values ≥ 5.5 mEq/L. The maximum change from baseline in eGFR was − 8.3 mL/min/1.73 m2 at week 24. Conclusions Esaxerenone reduced UACR in Japanese patients with T2D and UACR ≥ 300 mg/g creatinine; more than half experienced a transition from UACR ≥ 300 mg/g creatinine to UACR < 300 mg/g creatinine. Clinical trial registration JapicCTI-173696

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