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

Verified siteInvestigationalNo placebo
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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, which stands for 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. The study involves a treatment called GLM101, which is administered through an injection or infusion directly into the bloodstream. GLM101 contains a substance known as alfa-d-mannopyranosyl phosphate dipotassium, which is designed to help manage the symptoms of PMM2-CDG.

The purpose of this study is to examine the safety of GLM101 in patients who have previously received this treatment. Participants will receive GLM101 over a period of time, and their health will be monitored to gather information on any side effects or changes in their condition. The study will also look at how the treatment affects certain symptoms of PMM2-CDG, such as coordination and balance issues, which are measured using a tool called the International Co-operative Ataxia Rating Scale (ICARS). Additionally, the study will measure the levels of a sugar called Mannose-1-Phosphate in the blood, which is a component of GLM101.

Throughout the study, participants will undergo regular health checks, including blood and urine tests, heart monitoring with an ECG (a test that records the electrical activity of the heart), and assessments of vital signs like blood pressure and body temperature. These evaluations will help researchers understand the effects of GLM101 and ensure the safety of the participants. The study is expected to continue for several years, allowing for a comprehensive assessment of the treatment's long-term impact on individuals with PMM2-CDG.

The research process

The trial runs in 4 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, the participant must have successfully completed a previous treatment period with GLM101.

    The participant must be between 12 and 65 years old and meet specific criteria regarding reproductive health and contraception.

  2. Step 2

    Treatment administration

    GLM101 is administered through an intravenous injection or infusion.

    The treatment involves the administration of GLM101, which contains the active substance alfa-d-mannopyranosyl phosphate dipotassium.

  3. Step 3

    Monitoring and assessments

    Regular monitoring includes collecting safety information such as side effects, blood and urine tests, ECG, vital signs, and physical exams.

    The International Co-operative Ataxia Rating Scale (ICARS) is evaluated every 3 to 6 months to assess changes compared to before treatment.

    The amount of Mannose-1-Phosphate in the blood is evaluated at Month 6.

  4. Step 4

    Completion of study

    The study is estimated to end by March 2, 2029.

    Participants are required to comply with the study protocol throughout the duration of the trial.

Who can join the trial?

8 criteria

  • The patient must be willing and able to give their agreement to participate, either directly or through a legal representative.
  • The patient must have successfully completed a previous treatment period with the study drug, GLM101.
  • The patient must be between 12 and 65 years old at the time of agreeing to participate.
  • If the patient is a female who can become pregnant, she must not be pregnant, confirmed by a negative pregnancy test. She must use a medically accepted method of birth control, such as abstinence, hormonal contraceptives with a barrier method, or an intrauterine device, and agree to continue using it for 50 days after the last dose of GLM101.
  • If the patient is a female who cannot become pregnant, she must be pre-pubertal, surgically sterile, or have a confirmed ovarian dysfunction with specific hormone levels and no menstrual periods for 12 months without another medical reason.
  • If the patient is a sexually active male with female partners, he must agree to use a medically accepted method of birth control, such as abstinence, the partner using hormonal contraceptives with a male condom, or the partner using an intrauterine device with a male condom, and continue using it for 50 days after the last dose of GLM101.
  • If the patient is male, he must agree not to donate sperm during the study and for 50 days after the last dose of GLM101.
  • The patient must be willing and able to follow the study's rules and procedures.
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Investigated drugs

GLM101 is a medication being studied for its safety and effectiveness in patients with PMM2-CDG, a rare genetic disorder. In this clinical trial, GLM101 is administered intravenously to participants who have previously received the medication. The main goal of the study is to ensure that GLM101 is safe for long-term use in these patients.

What is already known about the treatment

GLM101 – This medication is administered intravenously and is currently being studied in clinical trials for its safety and efficacy in treating PMM2-CDG, a rare genetic disorder. It is in the experimental phase, with ongoing research to understand its potential benefits and risks. The main therapeutic indication is for PMM2-CDG, aiming to address the underlying metabolic issues associated with this condition. At the molecular level, GLM101 is designed to interact with specific pathways to correct or improve the metabolic dysfunctions caused by the genetic mutation. It falls under the pharmacological classification of investigational drugs, as it is still being evaluated for its therapeutic potential.

Investigated diseases

PMM2-CDG – PMM2-CDG, also known as Phosphomannomutase 2 Congenital Disorder of Glycosylation, is a rare genetic disorder that affects the body's ability to properly process certain sugars, leading to a wide range of symptoms. It is caused by mutations in the PMM2 gene, which is crucial for the production of glycoproteins. The disease can manifest with developmental delays, neurological issues, and problems with various organs. Symptoms often appear in infancy or early childhood and can include poor muscle tone, difficulty feeding, and growth delays. As the child grows, additional symptoms such as ataxia, seizures, and vision problems may develop. The severity and progression of symptoms can vary widely among individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-512171-12-00Protocol codeGLM101-007Estimated enrolment99 patientsSponsorGlycomine Inc.

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