GLM101 for Phosphomannomutase 2 Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
The trial aims to explore the long-term effects of GLM101 for individuals with Phosphomannomutase 2 Deficiency (PMM2-CDG), a rare genetic disorder affecting protein production. Participants will receive weekly infusions of GLM101, continuing the treatment from previous studies. This trial suits those who have completed GLM101 treatment in an earlier study and have a confirmed PMM2-CDG diagnosis. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.
Do I need to stop my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.
Is there any evidence suggesting that GLM101 is likely to be safe for humans?
A previous study administered GLM101 over 200 times to patients with PMM2-CDG. The results showed that GLM101 is generally safe and well-tolerated, with no serious side effects reported. Another study confirmed that most participants did not experience significant negative effects, indicating a favorable safety profile. These findings suggest that GLM101 is likely safe for human use based on the available data.12345
Why do researchers think this study treatment might be promising for PMM2 deficiency?
Unlike the standard treatments for Phosphomannomutase 2 Deficiency, which often involve dietary management and supplements like mannose, GLM101 offers a new approach. GLM101 is administered through weekly intravenous infusions, potentially providing more consistent therapeutic levels compared to oral treatments. Researchers are particularly excited because GLM101 directly targets the underlying enzyme deficiency, which could lead to more effective management of the condition's symptoms. This targeted approach holds promise for improving the quality of life for patients who have limited treatment options.
What evidence suggests that GLM101 might be an effective treatment for PMM2-CDG?
Research has shown that GLM101, the investigational treatment in this trial, may help treat Phosphomannomutase 2 Deficiency (PMM2-CDG). In earlier studies, patients who took GLM101 showed noticeable improvements in symptoms like ataxia, which affects coordination. On average, they improved by 11.9 points on a scale measuring movement difficulties. This treatment was generally safe and well-tolerated by patients. Early results from adult patients demonstrated improvements, suggesting that GLM101 can help manage this rare genetic disorder. Overall, these findings support the potential effectiveness of GLM101 in treating PMM2-CDG.12356
Who Is on the Research Team?
Chief Medical Officer
Principal Investigator
Glycomine, Inc.
Are You a Good Fit for This Trial?
This trial is for people at least 2 years old with a confirmed diagnosis of PMM2-CDG who have already completed treatment with GLM101 in a previous study. Participants must be able to give consent and agree to use birth control if they could become pregnant or father a child.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive weekly intravenous infusions of GLM101 at 30 mg/kg, with dose adjustments based on safety and efficacy data
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants continue to receive GLM101 to assess long-term safety and efficacy
What Are the Treatments Tested in This Trial?
Interventions
- GLM101
Trial Overview
The study provides ongoing weekly infusions of the drug GLM101 at the same dose as before, aiming to learn about its long-term effects in treating PMM2-CDG. Only those previously treated with GLM101 can join.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
GLM101 IV infusions, given weekly
Find a Clinic Near You
Who Is Running the Clinical Trial?
Glycomine, Inc.
Lead Sponsor
Citations
1.
glycomine.com
glycomine.com/glycomine-announces-encouraging-efficacy-data-from-ongoing-phase-2-clinical-study-in-pmm2-cdg/Glycomine Announces Encouraging Efficacy Data from ...
Notable improvements were seen in ataxia, an average improvement of 12 points after 12 weeks (n=4) of treatment. improvement of 6 points after ...
GLM101 for Phosphomannomutase 2 Deficiency
Early results from adult patients suggest that this treatment can improve symptoms. Patient reported outcomes
NCT06892288 | A Study to Assess the Efficacy and Safety ...
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG,
4.
glycomine.com
glycomine.com/glycomine-completes-enrollment-in-global-phase-2b-polar-study-of-glm101-for-the-treatment-of-pmm2-cdg/Glycomine Completes Enrollment in Global Phase 2b ...
GLM101, which demonstrated meaningful improvements in ataxia and other clinical endpoints with a favorable safety profile,”
PMM2-CDG Clinical Trials
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG,
Open-Label Extension Study to Assess GLM101 in PMM2- ...
This study is designed to monitor long-term safety and treatment effect of GLM101 and provide continued access to study treatment. Participants will receive 30 ...
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.