Not recruitingRare disease

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

Verified siteInvestigational

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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 Phosphomannomutase 2 deficiency (PMM2-CDG). PMM2-CDG is a condition that affects various parts of the body and can lead to problems with movement, balance, and coordination, known as ataxia. The trial will investigate a treatment called GLM101, which is administered through an injection or infusion directly into the bloodstream. The active ingredient in GLM101 is a chemical compound known as alfa-d-mannopyranosyl phosphate dipotassium.

The purpose of this study is to understand how GLM101 affects the symptoms of PMM2-CDG, particularly focusing on changes in ataxia over a period of 12 and 24 weeks. Participants in the study will receive multiple doses of GLM101, and the trial will monitor how the body processes the medication, as well as its safety and tolerability. Some participants may receive a placebo instead of GLM101 to help compare the effects of the treatment.

The study will last for 24 weeks, during which participants will undergo regular assessments to track their progress and any changes in their condition. These assessments will include various tests and observations to ensure the safety of the participants and to gather information on how the treatment is working. The trial aims to provide valuable insights into the potential benefits of GLM101 for individuals with PMM2-CDG.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the participant will be informed about the trial's purpose and procedures. The participant will provide informed consent, either directly or through a legally authorized representative.
Step 2
Initial assessment
An initial assessment will be conducted to confirm eligibility. This includes verifying age, diagnosis of PMM2-CDG, and ensuring compliance with specific health criteria.
Step 3
Treatment administration
The participant will receive the medication GLM101 through an intravenous infusion. The treatment will be administered over a period of 24 weeks.
The dosage and frequency of the infusions will be determined by the study protocol, and the participant will be monitored for any immediate reactions.
Step 4
Monitoring and assessments
Throughout the study, regular assessments will be conducted to monitor the participant's health and response to the treatment.
These assessments will include evaluations of ataxia, safety parameters, and potential biomarkers. Blood tests and other clinical evaluations will be performed to gather necessary data.
Step 5
Final evaluation
At the end of the 24-week period, a final evaluation will be conducted to assess the overall impact of the treatment.
The participant's health and any changes in symptoms will be documented, and the data will be analyzed to determine the effectiveness and safety of GLM101.

Who can join the trial?

8 criteria

The participant can be male or female.
For certain groups, the participant must be between 18 to 65 years old, 12 to 17 years old, or 2 to 11 years old at the time of screening.
The participant must have a confirmed diagnosis of **PMM2-CDG**. This means they have specific genetic changes and enzyme activity that match this condition. Previous lab reports confirming this diagnosis are acceptable.
If the participant is a female who can have children, she must not be pregnant, confirmed by a test. She must use a reliable method of birth control and agree to continue using it for 50 days after the last treatment.
If the participant is a female who cannot have children, she must be pre-pubertal, have had surgery to prevent pregnancy, or have a medical condition confirmed by specific hormone levels and no periods for 12 months without another medical reason.
If the participant is a sexually active male with female partners, he must use a reliable method of birth control and agree to continue using it for 50 days after the last treatment. Males are considered unable to father children if they have had certain surgeries at least 3 months before screening.
If the participant is male, he must agree not to donate sperm during the study and for 60 days after the last treatment.
The participant must be willing and able to give informed consent, either directly or through a legally authorized representative.

Who cannot join the trial?

4 criteria

Participants who do not have the specific medical condition called PMM2-CDG cannot join the study.
Participants who are not within the specified age range cannot participate. The study is open to certain age groups only.
Participants who are part of a vulnerable population, meaning they might need special protection or care, are not eligible.
Participants who do not meet other specific health or medical criteria set by the study cannot take part.

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Verified sites

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Verified
Hospital Sant Joan De Deu Barcelona
Esplugues De Llobregat, Spain

Trial locations

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Investigated drugs

GLM101 is an investigational medication being studied for its potential effects on ataxia in individuals with PMM2-CDG. This medication is administered intravenously, meaning it is given directly into a vein. The study aims to understand how the body processes GLM101, its safety, and how well it is tolerated by participants. The trial will also assess any changes in symptoms after 12 and 24 weeks of treatment.

Alfa-D-Mannopyranosyl Phosphate Dipotassium

What is already known about the treatment

GLM101 – GLM101 is administered intravenously and is currently being studied in a Phase 2 clinical trial. It is being investigated for its potential to treat PMM2-CDG, a rare genetic disorder. The main goal of the trial is to assess changes in ataxia, a condition characterized by lack of muscle control, after 12 and 24 weeks of treatment. GLM101 is classified as an investigational drug, and its mechanism of action involves targeting specific pathways to improve symptoms associated with PMM2-CDG.

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 glycosylate proteins. This condition is caused by mutations in the PMM2 gene, leading to a deficiency in the enzyme phosphomannomutase 2. As a result, individuals with PMM2-CDG may experience a wide range of symptoms, including developmental delays, ataxia (lack of muscle coordination), and other neurological issues. The disease can also affect other systems in the body, leading to problems such as liver dysfunction, coagulation disorders, and growth retardation. Symptoms and severity can vary greatly among affected individuals, making it a complex condition to manage.