Extension Study to Assess Safety and Efficacy of GLM101 in Participants with PMM2-CDG
Research type
Research Study
Full title
A Phase 2, Open-Label Extension Study to Assess the Safety and Efficacy of GLM101 Administered Intravenously to Participants with PMM2-CDG
IRAS ID
1011727
Contact name
Rose Marino
Contact email
Sponsor organisation
Glcyomine Incorporated
Clinicaltrials.gov Identifier
Research summary
This research study is evaluating the long-term safety and effectiveness of the drug GLM101 in participants with phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG). Participants will have already received GLM101 in a prior parent study. The Sponsor of this study is Glycomine Incorporated.
PMM2-CDG 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 is developing GLM101.
In this extension study, participants will receive GLM101 by intravenous (IV) infusion for up to 4 years. Weekly infusions of GLM101 will be at 30 mg/kg, which is the same maximal dose used in the currently ongoing study GLM101-002 and in the upcoming study GLM101-003 (under review; IRAS ID:1011535).
GLM101 has received Orphan Drug Designation from both the USA and Europe. The Sponsor has also been granted a Rare Paediatric Disease Designation and Fast-track in the United States.
REC name
North of Scotland Research Ethics Committee 1
REC reference
25/NS/0043
Date of REC Opinion
28 May 2025
REC opinion
Further Information Favourable Opinion