Extended study of GLM101 for PMM2-CDG patients
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This study is for people who already completed a previous trial of GLM101 for PMM2-CDG. It offers continued access to the treatment while doctors monitor safety and effects.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have finished treatment with GLM101 in a prior clinical study.
- You must be 2 years old or older.
- You must have a confirmed diagnosis of PMM2-CDG through genetic testing or enzyme activity tests.
- If you are capable of becoming pregnant, you must use effective birth control during the study and for 50 days after the last dose.
- If you are male and sexually active, you must use effective birth control and avoid donating sperm during the study and for 50 days after the last dose.
- You must follow study rules and attend all visits.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This Phase 2 study tests whether taking D-galactose is safe and helps symptoms or health markers in people with PGM1-CDG, a rare genetic condition. Participants must already be on a stable D-galactose dose to compare outcomes during the trial.
This study tests a new experimental medicine called PMG1016 to see how safe it is and how it moves through the body in healthy volunteers. You'll receive a single dose and have your blood checked and heart monitored to make sure the medicine is well-tolerated.
This study follows children with Pelizaeus-Merzbacher disease (PMD) who have a specific genetic change (PLP1 duplication). It aims to learn which body measurements (“biomarkers”) and symptoms change over time, to better understand disease progression.
This trial is testing an experimental drug (ION356) given by a lumbar puncture to see if it is safe and how it works in boys with Pelizaeus Merzbacher disease (PMD). The drug is designed to target the genetic cause of PMD.
This study follows people of all ages with a confirmed POLG-related disease to learn how the condition changes over time. The information collected may help design better care and future treatments.
This study aims to learn more about rare disorders of purine and pyrimidine metabolism (DPPM), which can affect the brain, immune system, kidneys, and muscles. Researchers will study people with these disorders, their family members, and healthy volunteers to better understand the condition over time.
Hear when a new Pmm2-CDG trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.