Furthermore, GLM101 is Glycomine’s first drug from its platform and is designed to intracellularly deliver mannose-1-phosphate as a potential treatment for PMM2-CDG (also known as CDG-1a). Glycomine finished a clinical study in healthy volunteers, and will soon start GLM101 clinical trials in PMM2-CDG patients in several countries and has received Orphan Drug Designation (ODD) in the U.S. and Europe and Rare Pediatric Disease Designation (RPDD) in the U.S. The purpose of this first study in PMM2-CDG patients sponsored by Glycomine, Inc., is to help researchers understand what effects GLM101, has on people with PMM2-CDG. The information learned in the study may help doctors and benefit patients with PMM2-CDG in the future.
Learn more about the orphan designation (EU/3/18/2047)granted by the European Commission to Glycomine SARL, France, for liposomal mannose-1-phosphate for the treatment of phosphomannomutase 2-congenital disorder of glycosylation. Read here.
Clinical trial of GLM101 for the treatment of PMM2-CDG
The company recently finished a study dosing healthy volunteers in an open-label Phase 1 clinical study to evaluate safety and tolerability. Read more here.
A natural history for PMM2-CDG
To learn more about how PMM2-CDG affects patients, Glycomine has initiated a natural history study in 2018, which is the largest longitudinal study ever undertaken for this disease, called study GLY-000. In this study, 139 participants with PMM2-CDG were enrolled. In that study, Glycomine discovered that a proportion of patients with PMM2-CDG have low cortisol levels. This is important to know, because low cortisol levels can be treated (Read more about these findings in the following article entitled “Should patients with Phosphomannomutase 2-CDG (PMM2-CDG) be screened for adrenal insufficiency?” and that you can access HERE).
For more information about this ongoing study (GLY-000) please visit clinicaltrials.gov and read at https://clinicaltrials.gov/ct2/show/NCT03173300
But what is a natural study?
A natural history study helps researchers understand how a disease may affect people with the disorder over time. Natural history studies are often the first step in developing a research program for a disease because they help provide the foundational science that informs how to care for people, identify and measure signs and symptoms that potential therapies can treat or modify, and help us design clinical trials that are likely to provide good quality information on how candidate therapeutics affect the disease. In this webinar, an introduction to natural history study design and content, and how this information can be used to advance research will be provided, as well as how to find available resources to help start and build a natural history study.
