Abstract
What is this summary about?
This summarizes an article about the clinical study ‘VANGUARD’ that was published in The Lancet journal in February 2023. Hereditary angioedema (HAE) is a rare genetic disease that causes swellings throughout the body (called HAE attacks). HAE attacks in the upper airways (including the tongue and vocal cords) can be life threatening by making breathing difficult. HAE attacks may occur frequently and without warning, and people with HAE have a lower quality of life than other people.
In the VANGUARD study, researchers tested the safety of a new medicine called garadacimab and whether it could prevent HAE attacks. Garadacimab was injected subcutaneously (just under the skin) with a needle once a month.
What were the results?
In the VANGUARD study, patients took garadacimab or placebo (an identical-looking dummy substance with no medical effect, used for comparison). The aim was to see if garadacimab could prevent HAE attacks better than placebo.
Patients taking garadacimab had very few or no HAE attacks, but those taking placebo carried on having attacks. Garadacimab gave protection from HAE attacks shortly after it was first used, and this carried on for the 6 months of treatment. Most patients taking garadacimab (62%) had no HAE attacks throughout the 6 months of treatment (were attack free), but 100% of patients taking placebo had HAE attacks throughout the study. More patients taking garadacimab (82%) than placebo (33%) had a ‘good’ or ‘excellent’ experience living with HAE. Patients taking garadacimab and placebo had similar rates of side effects. Only 5% (2 out of 39) of patients taking garadacimab had discomfort or skin changes at the place of injection compared with 12% (3 out of 25) taking placebo.
What do the results mean?
Taking garadacimab once a month helps prevent HAE attacks from happening, with most patients being attack free throughout the 6 months of treatment. Garadacimab had very few and mostly mild or moderate side effects. Overall, garadacimab is a beneficial treatment for preventing HAE attacks.
Plain language summary
Garadacimab provided protection against #HAE attacks throughout treatment and improved patients' experiences when living with HAE. Learn more in this plain language summary. (Link) #Hereditaryangioedema #angioedema #garadacimab
This is an abstract of the Plain Language Summary of Publication article.
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Link to original article here
Writing disclosure
Medical writing assistance was provided by Jed Avissar, MSc (OPEN Health Communications, London, UK) and was funded by CSL Behring.
ifrd_a_2340387_sm1269.pdf
Download PDF (2.2 MB)Acknowledgments
We thank the study investigators, trial site coordinators, and patients who participated in this trial; the independent data monitoring committee members Bruce Zuraw (chairperson), Konrad Bork, and Danny Cohn for their support in the conduct of the study; John-Philip Lawo, Ingo Pragst, and Mary Scozzafava of CSL Behring for coordinating the trial and providing invaluable assistance in reviewing the manuscript.
Competing interests disclosure
Avner Reshef has received research grants as a principal investigator, speaker, and advisor for BioCryst, CSL Behring, Ionis, Pharming, Pharvaris, Shulov Innovative Science, and Takeda/Shire. Bruce Ritchie has been a speaker and advisory board member for CSL Behring and Takeda but has not received personal reimbursement for these activities; has participated in multiple clinical trials involving investigational drugs for BioCryst, CSL Behring, Dyax, Pharming, and Takeda (he does not hold patents or investments with these companies or involving this product); and serves as a volunteer medical scientific advisor to Hereditary Angioedema (HAE) Canada, a patient organization. Emel Aygören-Pürsün has received honoraria as a speaker/advisor and/or grant support/clinical trial investigator support from BioCryst, BioMarin, Centogene, CSL Behring, KalVista, Pharming, Pharvaris, and Takeda/Shire. Erik S G Stroes has received lecturing/advisory board fees from Amgen, AstraZeneca, Esperion, Ionis/Akcea, Merck, Novartis, and Sanofi, paid to their institution. Gordon L Sussman has been an advisory board member for CSL Behring and has participated in clinical trials for investigational drugs for Aimmune, Amgen, Aslan Pharmaceuticals, AstraZeneca, CSL Behring, DBV Technologies, Innovaderm Research, KalVista, Novartis, and Pharvaris. Henriette Farkas has received research grants from CSL Behring, Pharming, and Takeda; served as an advisor for BioCryst, CSL Behring, KalVista, ONO Pharmaceutical, Pharming, and Takeda; and participated in clinical trials/registries for BioCryst, CSL Behring, KalVista, Pharming, Pharvaris, and Takeda. Henrike Feuersenger is a full-time employee of CSL Behring Innovation GmbH and shareholder of CSL Ltd. H Henry Li is a speaker for BioCryst, CSL Behring, Pharming, and Takeda, and has received research and consultancy grants from BioCryst, BioMarin, CSL Behring, Ionis, Pharming, Phavaris, and Takeda. Inmaculada Martinez Saguer has received honoraria, research funding, and travel grants from BioCryst, CSL Behring, KalVista, Octapharma, Pharming, and Takeda/Shire, and/or has served as a consultant and/or participated in advisory boards for these companies. Iris Jacobs is a full-time employee of CSL Behring LLC and shareholder of CSL Ltd. Isao Ohsawa has received honoraria and/or served as a consultant and/or participated in advisory boards for CSL Behring, Takeda/Shire, and Torii Pharmaceutical. John Anderson is a speaker bureau member for BioCryst, CSL Behring, Pharming, and Takeda; has received consulting fees from and is a clinical trial investigator for BioCryst, BioMarin, CSL Behring, Kalvista, Pharming, Pharvaris, and Takeda; and has received consulting fees from Cycle Pharmaceuticals. Jonathan A Bernstein is a consultant and principal investigator for BioCryst, BioMarin, CSL Behring, Ionis, KalVista, Pharming, Pharvaris, and Takeda/Shire; is a consultant for Astria, Cycle Pharmaceuticals, and ONO Pharmaceutical. Joshua S Jacobs is a speaker for AstraZeneca, CSL Behring, GSK, Regeneron, Sanofi Genzyme, Takeda/Shire, and Teva, and has received research funding/consultancy fees from Allakos, AstraZeneca, Astria Therapeutics, BioCryst, CSL Behring, Fresenius Kabi, Genentech, GSK, Pharvaris, Novartis, Regeneron, and Takeda/Shire. Kimito Kawahata has not received any consulting fees, honoraria, or grants; is not a shareholder of any pharmaceutical company; and has been a clinical trial investigator for CSL Behring. Lolis Wieman is a full-time employee of CSL Behring LLC and shareholder of CSL Ltd. Markus Magerl has received financial support from CSL Behring for acting as a study center investigator during the conduct of the study and personal fees from Astria, BioCryst, CSL Behring, Kalvista, Pharming, Pharvaris, and Takeda. Michael E Manning is a speaker for Amgen, AstraZeneca, BioCryst, Blueprint, CSL Behring, Genentech, GSK, Pharming, Sanofi/Regeneron, and Takeda; has received research grants from Allakos, BioCryst, CSL Behring, GSK, KalVista, Merck, Novartis, Pharming, Pharvaris, and Takeda; and has served as a consultant for BioCryst, CSL Behring, Cycle Pharmaceuticals, KalVista, Pharming, and Takeda. Petra Staubach has received honoraria, research funding, and travel grants and/or has served as a consultant and/or participated in advisory boards for CSL Behring, Octapharma, Pharming, Shire, and Takeda. Raffi Tachdjian is a speaker for AstraZeneca, BioCryst, CSL Behring, Grifols, GSK, Pharming, Sanofi/Regeneron, and Takeda; has served as a consultant for BioCryst, CSL Behring, KalVista, Pharming, and Takeda; and has received grants/research support from BioCryst, CSL Behring, Ionis, KalVista, Pharvaris, and Takeda. Regina Treudler has received honoraria, travel grants, and/or has participated in clinical trials and/or advisory boards for CSL Behring, Shire, and Takeda. Timothy J Craig is a speaker for Astria Therapeutics, BioMarin, CSL Behring, Grifols, Regeneron, and Takeda and has received research and consultancy grants from Astria Therapeutics, BioCryst, BioMarin, CSL Behring, GSK, Intellia, Ionis, KalVista, Pharvaris, and Takeda. He is a member of the US Hereditary Angioedema Association Medical Advisory Board and Director of the ACARE International Angioedema Center at Penn State University in Hershey, PA, USA. William H Yang has been a speaker and advisory board member for, and has received honoraria from, CSL Behring, Merck, Novartis, Sanofi Genzyme, and Takeda/Shire; has received research grants from Aimmune, ALK, Amgen, AnaptysBio, Aslan Therapeutics, AstraZeneca, BioCryst, Celgene, CSL Behring, DBV Technologies, Dermira, Eli Lilly, Galderma, Genentech/Roche, Glenmark, GSK, Haleon, Ionis, Incyte Biosciences, Novartis, Novavax, Pharming, Pharvaris, Providence, Regeneron, Sanofi Genzyme, Takeda/Shire, and VBI; serves as a medical advisor (volunteer) for HAE Canada, a patient organization; and is a member of Angioedema Centers of Reference and Excellence. William R Lumry is a speaker for AstraZeneca, CSL Behring, Grifols, GSK, Optinose, Pharming, Sanofi/Regeneron, and Takeda/Shire; has served as a consultant for Astria Therapeutics, BioCryst, BioMarin, CSL Behring, Fresenius Kabi, Intellia, KalVista, Pharming, Pharvaris, and Takeda/Shire; is a board member of the US Hereditary Angioedema Association Medical Advisory Board; and has received grants/research support from BioCryst, BioMarin, CSL Behring, Grifols, Ionis, KalVista, Takeda/Shire, and Teva. Yusuke Suzuki has received speaker fees from AstraZeneca, Novartis, Takeda/Shire, and Torii Pharmaceutical, and has been a clinical trial investigator for CSL Behring. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
