In short
Clinical trials investigating ALFA-D-MANNOSE 1-PHOSPHATE DIPOTASSIUM are studying GLM101 in people with PMM2-CDG. These trials look at both safety and effectiveness, including changes in ataxia and long-term safety checks in participants who have already received treatment.
Key points
- Clinical trials of ALFA-D-MANNOSE 1-PHOSPHATE DIPOTASSIUM are studying GLM101 in people with PMM2-CDG. Both trials are Phase 2 and are authorised. One study looks at whether weekly treatment can improve ataxia over 24 weeks, using ICARS as the main outcome. The other study follows patients longer to collect safety information, including side effects, blood and urine tests, ECG, vital signs, and physical exam changes. The target population is people with PMM2-CDG, including patients who previously received GLM101.
Trial overview
Two interventional studies are investigating ALFA-D-MANNOSE 1-PHOSPHATE DIPOTASSIUM through the study drug GLM101 in people with PMM2-CDG. Both studies are Phase 2 and are listed as authorised.
One study is focused on weekly doses and compares GLM101 with placebo to see whether symptoms of ataxia change after 24 weeks. The other is a long-term study that follows patients who previously received GLM101 to learn more about safety over time.
Who is being studied
The target condition in both trials is PMM2-CDG, which is the only condition named in the source data. The long-term study includes patients who already received GLM101 before joining this follow-up study.
The first trial plans to enroll 50 participants, and the long-term study plans to enroll 80 participants. This means the research is being done in relatively small groups, which is common in early testing stages.
What the trials measure
The main outcome in the weekly-dose study is the change from baseline in ICARS at 24 weeks. Baseline means the starting point before treatment begins, and ICARS is a score used to measure ataxia, which means problems with balance and coordination.
The long-term study mainly collects safety information. This includes side effects, deaths, stopping the study because of side effects, blood and urine tests, ECG results, vital signs, and changes in the physical exam compared with before treatment.
Trial phases and status
Both trials are in Phase 2, which usually means the treatment is being studied in a smaller group of patients to learn more about safety and possible benefit. Both studies are marked as authorised in the source data.
The first trial is designed to compare GLM101 with placebo, while the second trial follows patients over a longer period with GLM101 exposure already in their history. These two designs help researchers look at both short-term effect and longer-term safety.
Trial designs and treatment plans
In the first study, participants receive weekly doses of GLM101 or placebo by intravenous administration, which means the treatment is given into a vein. The source data lists the dose as 30 mg/kg for both GLM101 and the placebo infusion description.
In the long-term study, GLM101 is also given by intravenous use. The study is not focused on a new comparison with placebo; instead, it is built to watch what happens over time in patients who have already received the treatment.
Key patient terms
Ataxia means trouble with balance and coordination, which can affect walking and movement.
Placebo means a look-alike treatment used for comparison in a study, so researchers can see whether the study drug has a real effect.
Vital signs are basic health checks such as blood pressure, heart rate, and body temperature.
ECG is a heart tracing test that shows the heart’s electrical activity.
Urinalysis and blood tests help researchers watch for changes in body function during the study.