Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid
Inserm · Université Paris-Est Créteil · +18 more institutions
Abstract
Transthyretin amyloid (ATTR) cardiomyopathy is a progressive and fatal disease caused by misfolded transthyretin. Despite advances in slowing disease progression, there is no available treatment that depletes ATTR from the heart for the amelioration of cardiac dysfunction. NI006 is a recombinant human anti-ATTR antibody that was developed for the removal of ATTR by phagocytic immune cells.
In this phase 1, double-blind trial, we randomly assigned (in a 2:1 ratio) 40 patients with wild-type or variant ATTR cardiomyopathy and chronic heart failure to receive intravenous infusions of either NI006 or placebo every 4 weeks for 4 months. Patients were sequentially enrolled in six cohorts that received ascending doses (ranging from 0.3 to 60 mg per kilogram of body weight). After four infusions, patients were enrolled in an open-label extension phase in which they received eight infusions of NI006 with stepwise increases in the dose. The safety and pharmacokinetic profiles of NI006 were assessed, and cardiac imaging studies were performed.
Citation impact
- FWCI
- 33.17
- Percentile
- 100%
- References
- 36
Authors
14- PGPablo García‐PavíaCorresponding
Inserm, Université Paris-Est Créteil, Heidelberg University, Universidad Francisco de Vitoria, University Hospital Heidelberg, Centro de Investigación en Red en Enfermedades Cardiovasculares, Hospital Universitario Puerta de Hierro Majadahonda, Laboratoire Traitement du Signal et de l'Image
- FAFabian aus dem Siepen
Inserm, Université Paris-Est Créteil, Heidelberg University, University Hospital Heidelberg, Hospital Universitario Puerta de Hierro Majadahonda, Laboratoire Traitement du Signal et de l'Image
- EDErwan Donal
Inserm, Université Paris-Est Créteil, Heidelberg University, University Hospital Heidelberg, Hospital Universitario Puerta de Hierro Majadahonda, Laboratoire Traitement du Signal et de l'Image, Université de Rennes
- OLOlivier Lairez
Inserm, Université Paris-Est Créteil, Heidelberg University, University Hospital Heidelberg, Centre Hospitalier Universitaire de Toulouse, Hospital Universitario Puerta de Hierro Majadahonda, Laboratoire Traitement du Signal et de l'Image, Hôpital Rangueil
- PVPeter van der Meer
University Medical Center Groningen, Inserm, University of Groningen, Université Paris-Est Créteil, Heidelberg University, University Hospital Heidelberg, Hospital Universitario Puerta de Hierro Majadahonda, Laboratoire Traitement du Signal et de l'Image
Topics & keywords
- Medicine
- Transthyretin
- Pharmacodynamics
- Cardiomyopathy
- Heart failure
- Pharmacokinetics
- Internal medicine
- Placebo
- Good health and well-being