Prospective, Open-label, Uncontrolled, Phase III Study to Assess the Efficacy and Safety of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery in Subjects With Congenital Fibrinogen Deficiency Read more about Prospective, Open-label, Uncontrolled, Phase III Study to Assess the Efficacy and Safety of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery in Subjects With Congenital Fibrinogen Deficiency
Prophylactic Acetaminophen (Panadol) To Prevent the Intraventricular Haemorrhage of Premature Babies. Read more about Prophylactic Acetaminophen (Panadol) To Prevent the Intraventricular Haemorrhage of Premature Babies.
Phase IIIb, Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness & Safety of the use of Carbaglu® in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA) Read more about Phase IIIb, Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness & Safety of the use of Carbaglu® in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)
Phase III, Single-arm, Open-label, International, Multi-centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients with Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid-lowering Therapy Read more about Phase III, Single-arm, Open-label, International, Multi-centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients with Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid-lowering Therapy
Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%) Read more about Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%)
Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe hemophilia B (FIX:C<2%) (BeneGene-2) Read more about Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe hemophilia B (FIX:C
Perioperative Ischemic Evaluation-3 Tril (POISE-3) Read more about Perioperative Ischemic Evaluation-3 Tril (POISE-3)
Perioperative Ischemic Evaluation-3 Tril (POISE-3) Read more about Perioperative Ischemic Evaluation-3 Tril (POISE-3)
Open Label, Multinational, Multicenter, Real World Treatment Study of Single Agent AZD9291 for Patients with Advanced/Metastatic Epidermal Growth Factor Receptor (EGFR)T790M Mutation-Positive Non-Small Cell Lung Cancer (NSCLC) Who Have Received Prior Therapy with an EGFR Tyrosine Kinase Inhibitor(EGFR-TKI) Read more about Open Label, Multinational, Multicenter, Real World Treatment Study of Single Agent AZD9291 for Patients with Advanced/Metastatic Epidermal Growth Factor Receptor (EGFR)T790M Mutation-Positive Non-Small Cell Lung Cancer (NSCLC) Who Have Received Prior Therapy with an EGFR Tyrosine Kinase Inhibitor(EGFR-TKI)
Multicentre, Randomised, Controlled, Open-label Study Comparing the Efficacy and Safety of Slow Repeated IV Infusions of 2 Doses of Doxorubicin Transdrug™ (DT) (20mg/m2 and 30mg/m2) to Those of Best Standard of Care (BSC) in Patients With Advanced Hepatocellular Carcinoma (HCC) After Failure or Intolerance to Sorafenib. ReLive Study. Read more about Multicentre, Randomised, Controlled, Open-label Study Comparing the Efficacy and Safety of Slow Repeated IV Infusions of 2 Doses of Doxorubicin Transdrug™ (DT) (20mg/m2 and 30mg/m2) to Those of Best Standard of Care (BSC) in Patients With Advanced Hepatocellular Carcinoma (HCC) After Failure or Intolerance to Sorafenib. ReLive Study.
