GLM101-003
Research type
Research Study
Full title
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
IRAS ID
1011535
Contact name
Rose Marino
Contact email
Sponsor organisation
Glycomine, Inc.
Eudract number
2024-520109-37
Clinicaltrials.gov Identifier
Research summary
This is an open label 56-week study of GLM101 for PMM2-CDG patients with a confirmed diagnosis of PMM2-CDG. PMM2-CDG is a rare, genetic metabolic disorder. Patients with PMM2-CDG have low levels of mannose-1-phosphate (M1P) which can cause organs to not work properly. There are no approved therapies. Treatment of PMM2-CDG currently relies on the monitoring and managing disease complications.
Given the high unmet medical need, Glycomine is developing GLM101 as an investigational replacement therapy made of M1P, which is intended to replace the deficient M1P in patients with PMM2-CDG. The purpose of this study is to evaluate the clinical effects and safety of GLM101 in patients with PMM2-CDG.
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).
This trial is for male or female patients aged ≥ 4 years old, with a confirmed diagnosis of PMM2-CDG. Approximately 36 participants (≥ 12 years old) with PMM2-CDG and approximately 14 participants (≥ 4 to < 12 years old) with PMM2-CDG are planned to be enrolled at clinical sites globally.Patients will attend weekly study visits to receive either GLM101 or placebo for 24 weeks during part A (Double-blind Treatment Period) or GLM101 for 24 weeks during part B (Open-label Extension Period) with a 4 week safety follow-up period.
Participants will have weekly infusions of GLM101 (or placebo during part A) at 30 mg/kg body weight.Blood and urine will be collected to examine safety, to measure the presence or progress of PMM2-CDG and to understand how GLM101 may be working. Physical tests and questionnaires will be given to see if GLM101 changes how PMM2-CDG patients function and feel.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
25/EE/0043
Date of REC Opinion
30 Apr 2025
REC opinion
Further Information Favourable Opinion