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M1P Dipotassium Salt

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.

Table of contents

Trial overview

Two interventional studies are investigating ALFA-D-MANNOSE 1-PHOSPHATE DIPOTASSIUM through the study drug GLM101 in people with PMM2-CDG.[1][2] Both studies are Phase 2 and are listed as authorised.[1][2]

One study is focused on weekly doses and compares GLM101 with placebo to see whether symptoms of ataxia change after 24 weeks.[1] The other is a long-term study that follows patients who previously received GLM101 to learn more about safety over time.[2]

Who is being studied

The target condition in both trials is PMM2-CDG, which is the only condition named in the source data.[1][2] The long-term study includes patients who already received GLM101 before joining this follow-up study.[2]

The first trial plans to enroll 50 participants, and the long-term study plans to enroll 80 participants.[1][2] This means the research is being done in relatively small groups, which is common in early testing stages.[1][2]

What the trials measure

The main outcome in the weekly-dose study is the change from baseline in ICARS at 24 weeks.[1] Baseline means the starting point before treatment begins, and ICARS is a score used to measure ataxia, which means problems with balance and coordination.[1]

The long-term study mainly collects safety information.[2] 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.[2]

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.[1][2] Both studies are marked as authorised in the source data.[1][2]

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.[1][2] These two designs help researchers look at both short-term effect and longer-term safety.[1][2]

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.[1] The source data lists the dose as 30 mg/kg for both GLM101 and the placebo infusion description.[1]

In the long-term study, GLM101 is also given by intravenous use.[2] 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.[2]

Key patient terms

Ataxia means trouble with balance and coordination, which can affect walking and movement.[1]

Placebo means a look-alike treatment used for comparison in a study, so researchers can see whether the study drug has a real effect.[1]

Vital signs are basic health checks such as blood pressure, heart rate, and body temperature.[2]

ECG is a heart tracing test that shows the heart’s electrical activity.[2]

Urinalysis and blood tests help researchers watch for changes in body function during the study.[2]

Trial ID Phase Condition studied Status Enrollment
2024-520109-37-00 Phase 2 PMM2-CDG Authorised 50
NCT06657859 Phase 2 PMM2-CDG Authorised 80

FAQ

  • What condition is being studied in these trials? The trials are studying PMM2-CDG, a rare condition. The research focuses on how GLM101 is used in people with this disease.
  • Who can join these studies? The studies involve people with PMM2-CDG. One trial includes participants with PMM2-CDG, and the long-term study includes patients who previously received GLM101.
  • What are the trials trying to find out? One trial is looking at whether weekly doses can improve ataxia, which means problems with balance and coordination. The other trial is checking long-term safety.
  • What phase are these trials? Both studies are Phase 2 trials. This means they are still testing the treatment in a small group to learn more about its effects and safety.
  • What results are being measured? One study measures change in ICARS at 24 weeks, which is a scale for ataxia. The long-term study measures side effects, deaths, stopping treatment because of side effects, blood and urine tests, ECG, vital signs, and physical exam changes.

Ongoing Clinical Trials on M1P Dipotassium Salt

  • Long-Term Safety Study of GLM101 for Patients with PMM2-CDG

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Italy Portugal Spain

  • Study on the Effects and Safety of GLM101 for Patients with PMM2-CDG

    Not recruiting

    2 1 1
    Investigated drugs:
    Belgium Czechia France Germany Italy Poland +2

Glossary

  • PMM2-CDG: A rare condition studied in these trials. The studies focus on people who have this disease.
  • Phase 2: A trial stage that tests a treatment in a smaller group to learn more about how well it works and how safe it is.
  • Interventional study: A study where participants receive a treatment or placebo so researchers can compare results.
  • Placebo: A look-alike treatment with no active study drug. It is used for comparison in research.
  • Ataxia: Problems with balance and coordination.
  • ICARS: A rating scale used to measure ataxia. A change in the score shows whether symptoms got better or worse.
  • Safety information: Details about side effects, serious problems, deaths, and whether people stop the study because of side effects.
  • ECG: A heart tracing test that checks the heart’s electrical activity.
  • Vital signs: Basic body checks such as blood pressure, heart rate, and body temperature.
  • Hematology: Blood tests that help doctors check different parts of the blood.
  • Urinalysis: A urine test used to look for signs of health problems.

References