A Phase 2b, Multicenter, Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 Administered Intravenously to Participants with PMM2-CDG
GLM101
What is the goal of the study?
Phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG, formerly known as CDG Type Ia) is a rare, predominantly pediatric, frequently lethal, inherited autosomal recessive metabolic disease affecting protein function, with no approved therapies. Currently, treatment of PMM2-CDG relies on the use of non-standardized, palliative measures to manage the clinical manifestations of the disease. Given the high unmet medical need, Glycomine is developing GLM101 for systemic delivery in patients with PMM2-CDG. PMM2-CDG is caused by a deficiency in phosphomannomutase 2 (PMM2), the enzyme that converts mannose-6-phosphate (M6P) into mannose-1-phosphate (M1P). GLM101 is a liposomal formulation encapsulating the active pharmaceutical ingredient M1P inside the aqueous core of the lipid vesicles. It is being developed for the potential use as M1P substrate replacement therapy for PMM2-CDG patients. Encapsulation of the hydrophilic M1P into the architecture of a liposome is intended to protect it from degradation in the systemic circulation and deliver it inside the target cells. Glycomine has previously conducted a study to assess the safety of GLM101 in healthy participants (Study GLM101-001). Dosing in the present study, GLM101-003 will be performed in participants with PMM2-CDG and is based on the safety findings of Study GLM101-001 and Study GLM101-002, a separate Phase 2 study in participants with PMM2-CDG (NCT05549219 and EU CT number 2024-513119-29-00). It is expected that the results from the current study and Study GLM101-002 will inform safety and efficacy of GLM101 in participants with PMM2-CDG
Who can participate in the study?
Please contact the study team listed below to learn more.