Try the modernized ClinicalTrials. Learn more about the modernization effort. Hide glossary Glossary Study record managers: refer to the Data Element Definitions if submitting registration or results information. Search for terms. Explore , research studies in all 50 states and in countries. Find a study all fields optional Saved Studies. Recruiting and not yet recruiting studies All studies. If you choose to participate, you must go through our informed consent process, where you will learn key facts about the study.
You'll have plenty of time to ask questions, and the research team will walk you through details like: why they are doing the study, possible risks and benefits, how long it will last and the needed tests. This process will continue throughout the study.
It is not a contract. You can always ask questions, and you will always be able to leave the study at any time for any reason. King: Like existing vaccines against seasonal influenza, FluMos-v1 Table 1 is meant to create antibodies to several different influenza virus strains. Unlike existing influenza shots, the new vaccine uses a mosaic nanoparticle immunogen that co-displays antigens from multiple influenza viruses on the same nanoparticle surface.
In our pre-clinical work, we found that the nanoparticle immunogen elicited a greater diversity of protective antibodies than did current vaccines against influenza. We are particularly interested in learning about how the vaccine will interact with immune systems that have encountered influenza viruses or vaccines before.
Despite this setback, the full results are not yet out and are critically important. The trial results will be a first real look at fundamental aspects of RNA-lowering strategies, including how the drug impacted various biomarkers across hundreds of participants. The results could transform the clinical-trial landscape for other conditions treatable by ASOs. Julian Gillmore: I have been looking after patients with transthyretin amyloidosis for 25 years, and until 5 years ago, I just watched their health decline.
Some years ago, gene silencers became available and improved outcomes for patients, which was a very substantial advance. Preliminary data from this trial have shown for the first time that CRISPR-based gene therapies can be administered by intravenous infusion to edit a specific gene in liver cells.
Full results are expected next year. This new gene-editing therapy offers patients the prospect of meaningful clinical improvements after a single dose of the drug. Julian Gillmore is a reader in medicine and an honorary consultant nephrologist at Royal Free London Hospital.
This trial tests new combination regimens of miltefosine and paromomycin in Ethiopia, Sudan, Kenya and Uganda in eastern Africa. The aim is to replace the toxic sodium stibogluconate component of the current injectable treatment for kala-azar with the orally administered miltefosine.
This is a much-needed improvement patients have been waiting for and we have been working toward for years, as oral treatments can be administered at local health centers, closer to affected communities.
Patients diagnosed with kala-azar will die if they are not treated. The new, less toxic and more easily administered regimen will also be good for children, who are disproportionately affected by visceral leishmaniasis.
Blair Leavitt: There are several ongoing studies of pridopidine that target disease progression in neurodegenerative disorders. Pridopidine is an oral small-molecule therapeutic that selectively targets the sigma 1 receptor and has a well-established safety record from previous human clinical trials. Kevin M. Flanigan: A small number of boys with Duchenne muscular dystrophy have a disease caused by a duplication of exon 2 of the DMD gene which encodes dystrophin. This exon duplication can be removed through the use of an adeno-associated virus 9—based vector, which carries copies of the non-coding small nuclear RNA U7 with a sequence directed toward splice donor and acceptor sites within the duplicated exon.
This exon-skipping approach is a first in-human trial and results in exclusion of a copy of DMD exon 2, and expression of the full-length, wild-type dystrophin. Preliminary data have been presented showing successful expression, for the first time, of full-length dystrophin in response to this viral gene therapy.
There are four more gene therapies for Duchenne muscular dystrophy being tested, but each of these requires delivery of a gene encoding an engineered microdystrophin, which is not present in nature and which is expected to result in substantial functional improvement.
David Nutt: Psilocybin, a psychedelic derived from fungi, is showing great promise in the treatment of mental-health conditions. First, it tackles the huge problem of depression that is resistant to existing treatments.
Finally, it will also give some readouts on the dose—effect relationship, as they are testing three greatly differing doses of psilocybin, at 1 mg, 10 mg and 25 mg.
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