simoctocog alpha/von Willebrand Factor (OCTA101) / Octapharma - LARVOL DELTA

Safety, pharmacokinetics and efficacy of a subcutaneous recombinant FVIII (OCTA101) in adult patients with severe haemophilia A. (PubMed, Haemophilia) - P1/2 | "Despite promising PK and efficacy results, the trial was terminated due to the incidence of FVIII inhibitors. The occurrence of inhibitors at two dose levels suggests that their development may be related to the subcutaneous route of administration."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Rare Diseases

Safety and pharmacokinetics of a subcutaneous recombinant FVIII (OCTA101) in adult patients with severe haemophilia A (ISTH 2022) - P1/2 | "Background: Regular, prophylactic intravenous FVIII is the standard of care for severe haemophilia A, but can be challenging for some patients. Twenty patients were enrolled in the PK part of the study. Half-life and recovery were as expected from pre-clinical studies, and trough plasma FVIII levels ≥10% were achieved. Two patients developed inhibitory antibodies to OCTA101, and the trial was put on hold during discussion with the DMC."

Clinical • PK/PD data • Cardiovascular • Hematological Disorders • Hemophilia • Rare Diseases

Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A (clinicaltrials.gov) - P1/2 | N=30 | Terminated | Sponsor: Octapharma | Trial completion date: Sep 2022 ➔ Feb 2022 | Active, not recruiting ➔ Terminated | Trial primary completion date: Sep 2022 ➔ Feb 2022; Study was terminated due to SADRs

Trial completion date • Trial primary completion date • Trial termination • Hematological Disorders • Hemophilia • Rare Diseases

Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A (clinicaltrials.gov) - P1/2; N=36; Active, not recruiting; Sponsor: Octapharma; Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Hemophilia • Rare Diseases

Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A (clinicaltrials.gov) - P1/2; N=36; Recruiting; Sponsor: Octapharma; Active, not recruiting ➔ Recruiting; Trial completion date: Sep 2021 ➔ Sep 2022; Trial primary completion date: Sep 2021 ➔ Sep 2022

Clinical • Enrollment open • Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Rare Diseases

Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A (clinicaltrials.gov) - P1/2; N=28; Active, not recruiting; Sponsor: Octapharma; Recruiting ➔ Active, not recruiting; Trial completion date: Mar 2020 ➔ Sep 2021; Trial primary completion date: Mar 2020 ➔ Sep 2021

Clinical • Enrollment closed • Trial completion date • Trial primary completion date

Induced pluripotent stem cells show success in treating hemophilia A in mice (Yahoo Finance) - "In a new study released today in STEM CELLS Translational Medicine (SCTM), researchers at the University of California, Davis and the Chinese Academy of Science demonstrate how induced pluripotent stem cells can provide a renewable supply of endothelial cells. These cells can then be genetically modified to produce high levels of a clotting protein that was used to successfully treat hemophilia A in mice....In the study published in SCTM, the researchers explored endothelial cells (ECs) derived from hemophilia A patients' induced pluripotent stem cells (HA-iPSC-ECs) as a way to deliver FVIII."

Preclinical