PMM2-CDG
Conditions
Brief summary
Collection of the safety information from the collection of Side effects (or Adverse Events), deaths, and stopping the study due to side effects; Tests from blood and urine (hematology, chemistry, and urinalysis) (compared to before treatment with GLM101); ECG (heart tracings), vital signs (blood pressure, heart rate, and body temperature), and physical exam changes (compared to before treatment with GLM101)
Detailed description
Changes in International Co-operative Ataxia Rating Scale, ICARS (evaluated every 3-6 months, compared to before treatment with GLM101), The amount of Mannose-1-Phosphate (the sugar contained in a lipid bubble, which together make up GLM101) in blood (evaluated at Month 6)
Interventions
DRUGGLM101
Sponsors
Glycomine Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Collection of the safety information from the collection of Side effects (or Adverse Events), deaths, and stopping the study due to side effects; Tests from blood and urine (hematology, chemistry, and urinalysis) (compared to before treatment with GLM101); ECG (heart tracings), vital signs (blood pressure, heart rate, and body temperature), and physical exam changes (compared to before treatment with GLM101) | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in International Co-operative Ataxia Rating Scale, ICARS (evaluated every 3-6 months, compared to before treatment with GLM101), The amount of Mannose-1-Phosphate (the sugar contained in a lipid bubble, which together make up GLM101) in blood (evaluated at Month 6) | — |
Countries
Spain
Outcome results
None listed