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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare genetic disorder called PMM2-CDG (Phosphomannomutase 2 Congenital Disorder of Glycosylation). This condition affects the body's ability to properly process certain sugars, leading to a variety of health issues, including problems with movement and coordination, known as ataxia. The study is testing a new treatment called GLM101, which is given through an intravenous infusion, meaning it is administered directly into the bloodstream. The purpose of the study is to evaluate the effectiveness and safety of GLM101 in improving symptoms of PMM2-CDG, particularly focusing on changes in ataxia over a period of 24 weeks.

Participants in the study will receive weekly doses of either GLM101 or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure that the results are not biased. The study will last for up to 48 weeks, with participants being monitored for any changes in their condition and any potential side effects. The main goal is to see if GLM101 can improve movement and coordination in people with PMM2-CDG compared to those who receive a placebo.

Throughout the study, participants will undergo various assessments to track their progress. These assessments will include evaluations of their movement and coordination, as well as overall health checks to ensure safety. The study will also compare the effects of starting GLM101 treatment early versus later in the study. By the end of the trial, researchers hope to gather valuable information on how GLM101 can help manage symptoms of PMM2-CDG and improve the quality of life for those affected by this rare disorder.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be randomly assigned to receive either the study medication GLM101 or a placebo. A placebo is a substance with no active medication, used to compare the effects of the actual drug.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to establish a baseline for your condition. This includes evaluating your ataxia, which is a lack of muscle coordination, using a scale called ICARS (International Cooperative Ataxia Rating Scale).

  3. Step 3

    Weekly treatment

    You will receive weekly doses of the study medication GLM101 or placebo through an intravenous infusion. This means the medication will be administered directly into your vein. The treatment will continue for 24 weeks.

  4. Step 4

    Mid-study assessment

    At the 24-week mark, your condition will be reassessed to measure any changes in your ataxia using the ICARS scale. This will help determine the effectiveness of the treatment.

  5. Step 5

    Continuation or switch

    If you were initially receiving the placebo, you may be switched to the active treatment with GLM101 for an additional 24 weeks. If you were already receiving GLM101, you will continue with the treatment for the same duration.

  6. Step 6

    Final assessment

    At the end of the 48-week period, a final assessment will be conducted to evaluate the overall changes in your condition. This will include another evaluation using the ICARS scale and other relevant measures.

  7. Step 7

    Safety monitoring

    Throughout the study, your safety will be closely monitored. This includes regular checks of your vital signs, laboratory tests, and any side effects you may experience. It is important to report any new symptoms or concerns to the study team immediately.

Who can join the trial?

8 criteria

  • The participant must be 4 years old or older at the time of signing the consent form.
  • The participant must have a molecular diagnosis of PMM2-CDG. This means they have specific genetic changes related to the condition, confirmed by a lab report.
  • The participant must be willing and able to complete the ICARS test fully. ICARS is a test that measures movement and coordination.
  • The participant's total score on the ICARS test must be between 20 and 80.
  • Both male and female participants must have appropriate measures in place to prevent pregnancy during the study.
  • If the participant is male, he must agree not to donate sperm during the study and for 50 days after the last treatment.
  • The participant must be willing and able to provide informed consent or assent, either directly or through a legally authorized representative.
  • The participant must have a caregiver who is willing and able to complete questionnaires and provide informed consent.

Who cannot join the trial?

10 criteria

  • Participants cannot join if they have any other serious health conditions that might interfere with the study.
  • Participants who are pregnant or breastfeeding are not allowed to take part in the study.
  • Individuals who have participated in another clinical trial within the last 30 days are excluded.
  • Participants who are unable to follow the study procedures or attend the required visits cannot participate.
  • Anyone with a known allergy to the study medication or its ingredients is not eligible.
  • Participants who have a history of drug or alcohol abuse within the past year are excluded.
  • Individuals with a mental health condition that might affect their ability to participate are not allowed to join.
  • Participants who are taking certain medications that could interfere with the study are not eligible.
  • Anyone with a history of certain heart conditions is excluded from the study.
  • Participants who have had a major surgery within the last 3 months are not allowed to take part.
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Investigated drugs

GLM101 is a medication being tested in this clinical trial. It is given to participants through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein. The purpose of this medication is to help improve symptoms of a condition called PMM2-CDG, which affects movement and coordination. The trial aims to see if GLM101 can reduce problems with balance and coordination, known as ataxia, over a period of 24 weeks. Participants in the trial will receive this medication once a week, and researchers will compare their progress to those who do not receive the medication to determine its effectiveness and safety.

What is already known about the treatment

GLM101 – GLM101 is administered through an intravenous infusion, meaning it is delivered directly into the bloodstream via a vein. This medication is currently being studied in clinical trials, specifically in a Phase 2b study, to evaluate its effectiveness and safety for treating PMM2-CDG, a rare genetic disorder. The main goal of using GLM101 is to improve symptoms of ataxia, which affects coordination and balance, in individuals with PMM2-CDG. At the molecular level, GLM101 works by targeting specific pathways involved in the disease process, although the exact mechanism is still under investigation. It is classified pharmacologically as an investigational drug, meaning it is still being tested and is not yet approved for general medical use.

Investigated diseases

PMM2-CDG – PMM2-CDG, also known as Phosphomannomutase 2 Congenital Disorder of Glycosylation, is a rare genetic disorder affecting the body's ability to properly glycosylate proteins. This condition is caused by mutations in the PMM2 gene, leading to a deficiency in the enzyme phosphomannomutase 2. The disease primarily affects the nervous system, liver, and other organs, resulting in a wide range of symptoms. Common manifestations include developmental delays, ataxia, hypotonia, and abnormal liver function. As the disease progresses, individuals may experience worsening of neurological symptoms and other systemic complications. The severity and progression of symptoms can vary significantly among affected individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-520109-37-00Protocol codeGLM101-003Estimated enrolment50 patientsSponsorGlycomine Inc.

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