A Randomised, Open-Label, 24-week study of GLM101 for participants with PMM2-CDG
Research type
Research Study
Full title
A Phase 2, Randomised, Open-Label, 24-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 administered intravenously to adult, adolescent and paediatric participants with PMM2-CDG
IRAS ID
1008717
Contact name
Madeline Fielder
Contact email
Sponsor organisation
Glycomine Incorporated
Eudract number
2022-000565-40
Clinicaltrials.gov Identifier
Research summary
PMM2-CDG (phosphomannomutase 2 congenital disorder of glycosylation) is a rare inherited metabolic disease with no approved therapies. Patients with PMM2-CDG have low levels of mannose-1-phosphate (M1P). Current treatment of PMM2-CDG relies on the use of non-standardised, palliative measures to manage the disease. Given the high unmet medical need, the Sponsor, Glycomine Incorporated, is developing GLM101 for systemic delivery in patients with PMM2-CDG over a 24 week period to evaluate changes in coagulation and antithrombosis factors. Safety and tolerability of GLM101 will be carefully monitored, along with quality of life of the patients and the changes in caregiver burden from the start of the treatment to post week 24. This study looks at a new investigational drug, GLM101 for the treatment of PMM2-CDG. 'Investigational drug' means that the United States (US) Food and Drug Administration (FDA), European Medicines Agency (EMA), or other regulatory authorities in other countries have not yet approved GLM101, the study drug. The information collected from this study will help researchers understand what effect GLM101has on people with PMM2-CDG. This study may provide information about the future care and treatment of PMM2-CDG with GLM101. The purposes of this study are to evaluate the safety of GLM101, to evaluate the changes in ataxia (muscle control), to study any immune response to GLM101 (how the body reacts to GLM101), to study the effect of GLM101 on biomarkers. Biomarkers are found in a person's blood and measured to assess your health and how they change when you receive the study drug, to collect information with respect to quality of life changes. The Sponsor has received Orphan Drug Designation from both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of PMM2-CDG. The Sponsor has also been granted a Rare Paediatric Disease Designation in the United States.
REC name
London - Riverside Research Ethics Committee
REC reference
24/LO/0154
Date of REC Opinion
19 Apr 2024
REC opinion
Further Information Favourable Opinion