You are here: Resources / Research Opportunities

Research Announcements

Posted February 2022:

Links: 

To learn more about the study or refer patients, please see clinicaltrials.gov (NCT04732429) to contact the nearest study site.

For families that want to learn more, the HERO website - mma-pahero.com - provides patient-friendly information For questions, please contact Chief Medical Officer Patrick Horn, MD, PhD at horn@hemoshear.com

 

Arcturus is seeking participants ≥ 18 years of age with stable OTCD to take part in an ongoing clinical safety study of ARCT-810

Arcturus Therapeutics is developing a messenger RNA (mRNA) therapy for ornithine transcarbamylase deficiency (OTCD). Using mRNA is a new and fundamentally different approach to disease management than the use of traditional ammonia chelators and diet. The investigational ARCT-810 therapy contains mRNA with instructions to make fully functional OTC in the liver, thus addressing the root cause of OTCD with the potential to correct the urea cycle defect. mRNA technology uses lipid nanoparticles (LNPs) to protect the mRNA from degradation in the blood as it is distributed to the liver. The LNPs are designed to be taken up by hepatocytes and once inside the cell, they release the mRNA which can be translated to the OTC enzyme by the cells’ natural processes. Delivery of human OTC mRNA may help normalize ammonia and the other biochemical markers of ureagenesis.

This is an early Phase Ib single-dose study designed to assess the safety, tolerability and pharmacokinetics (metabolism and clearance) of ARCT-810. As such, participants who enroll must be adults with mild, stable OTC disease and good cognitive function. This may include women with only one OTCD gene (heterozygous). The information obtained from this study may allow the progression of ARCT-810 into studies of younger and sicker patients with OTCD. ARCT-810 is not a gene therapy, i.e., there is no alteration of the recipient’s genes and there are no viral components.

For more information about the Phase Ib clinical trial of ARCT-810 visit the study page on ClinicalTrials.gov: https://www.clinicaltrials.gov/ct2/show/NCT04442347?term=Arcturus&draw=2&rank=3

If you have questions or need help contacting one of the participating academic sites, please contact Manely Yafeh at manely@arcturusrx.com

David Geller, M.D. davidg@arcturusrx.com Vice President Pulmonary & Rare Diseases Arcturus Therapeutics     

 

Posted May 2020:

Updated list of clinical trials

 

 

Click here to return to Research Committee page

Contact Us

For more information about GMDI  or MetabolicPro
please contact us:
info@gmdi.org

 

GMDI
P.O. Box 1462
Hillsborough, NC 27278

Network

         

 

Copyright 2022 by Genetic Metabolic Dietitians International