Active not recruitingPHASE2, PHASE3INTERVENTIONAL

A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG

This study is checking if a new medicine, GLM101, is safe and helpful for people of all ages (from 4 years old) who have a rare genetic condition called PMM2-CDG. This condition can affect how the body moves and coordinates. Researchers will compare GLM101 to a 'dummy' treatment (placebo) to see if it improves these symptoms. The study has two main parts. In the first part, some people will get GLM101 and others will get the placebo for 24 weeks. This part is 'double-blind,' meaning no one knows who is getting which treatment. After that, in the second part, everyone who wants to continue will receive GLM101 for another 24 weeks. The main goal is to see if GLM101 helps with balance and coordination.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

Glycomine, Inc.

Enrolment target

Start

09 Jul 2025

Estimated completion

30 Apr 2027

Phosphomannomutase 2 Deficiency PMM2-CDG

What is this study about?

This study is about a rare genetic condition called PMM2-CDG. People with this condition can have problems with their balance, movement, and coordination. Researchers are testing a new medicine, GLM101, to see if it can help improve these symptoms and if it's safe to use.

To do this, the study will involve children, teenagers, and adults who have PMM2-CDG. They will be given either GLM101 or a 'dummy' medicine (called a placebo) for 24 weeks. This helps the researchers understand if any improvements are genuinely from GLM101 or if they might have happened anyway. After this first period, everyone in the study will then have the chance to receive GLM101 for another 24 weeks.

The main thing the researchers are looking for is whether GLM101 can make a difference in how well people can move and coordinate their bodies, especially focusing on balance. They'll use a special scale to measure these changes. They will also keep a close eye on any side effects to make sure the medicine is safe.

Key takeaways

This study is testing a new medicine (GLM101) for PMM2-CDG.
It aims to see if GLM101 is safe and helps with movement and coordination.
Participants will receive either GLM101 or a dummy treatment (placebo) for 24 weeks, then everyone will get GLM101 for another 24 weeks.
People aged 4 years and older with a confirmed diagnosis of PMM2-CDG can take part.
Regular clinic visits and assessments will be part of the study.
You can stop participating at any time.

Who may be eligible?

To join this study, people need to be at least 4 years old. They must have been officially diagnosed with PMM2-CDG, and there needs to be a record of this diagnosis.

Also, participants need to be able to complete a specific assessment about their movement and balance. There's a certain range of scores on this assessment that helps confirm they are a good fit for the study.

If you are a woman who could become pregnant, you'll need to use reliable birth control during the study and for a short time afterwards. This is to make sure you don't become pregnant while taking the study medicine, as its effects on pregnancy are not yet known. If you are unable to become pregnant (for example, if you are very young, past menopause, or have had certain surgeries), these measures won't apply to you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 4 years old or older?
Do you have a confirmed diagnosis of PMM2-CDG?
Can you complete balance and movement assessments?
If you are a woman who could become pregnant, are you willing to use effective birth control during the study?
Are you able to attend clinic visits for infusions and assessments?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll first have a screening period that lasts about 4 weeks. After that, the main study begins. For the first 24 weeks (Part A), you’ll receive weekly infusions of either the study medicine, GLM101, or a placebo (a dummy medicine through a drip). Neither you nor your doctor will know which one you are getting. Following this, if you wish to continue, you'll enter Part B, where everyone receives GLM101 infusions weekly for another 24 weeks. During the study, you'll have regular visits to the clinic for assessments, which will include checks on your balance and movement to see how you are doing. After your last infusion, there will be one final safety check-up visit about 4 weeks later. The total time you could be involved in the study is about a year.

Potential risks and benefits

Taking part in any study has potential benefits and risks. You might benefit if GLM101 proves to be effective in improving your PMM2-CDG symptoms, but there's no guarantee it will work for everyone, or at all. There's also a chance you could receive the placebo during the first part of the study. As with all medicines, GLM101 might cause side effects, which the study team will monitor closely. You are free to leave the study at any time, for any reason, without it affecting your medical care.

Locations (15)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Minnesota
Verified postcode
Minneapolis, United States
Icahn School of Medicine at Mount Sinai
Verified postcode
New York, United States
The Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States
Seattle Children's Hospital
Verified postcode
Seattle, United States
UZ Leuven, Campus Gasthuisberg
Verified postcode
Leuven, Belgium
Vseobecna fakultni nemocnice v Praze
Verified postcode
Prague, Czechia
AP-HP Hopital Necker-Enfants Malades
Verified postcode
Paris, France
Universitaetsklinikum Muenster
Verified postcode
Münster, Germany
Azienda Ospedaliero Universitaria Policlinico G. Rodolico-San Marco
Verified postcode
Catania, Italy
Azienda Ospedaliero Universitaria Pisana
Verified postcode
Pisa, Italy
Instytut Matki i Dziecka
Verified postcode
Warsaw, Poland
Unidade Local de Saúde de Santo António
Verified postcode
Porto, Portugal

Common questions

What is PMM2-CDG?

PMM2-CDG is a rare genetic condition that can affect how your body moves and coordinates actions, among other things.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active drug. It helps researchers see if the medicine itself is causing any changes.

How will I receive the study medicine?

The medicine (GLM101 or placebo) will be given to you weekly through an infusion into your vein, similar to a drip.

Will I know if I'm getting the real medicine or the placebo?

During the first 24 weeks, neither you nor your doctor will know if you're receiving GLM101 or the placebo. After that, everyone will receive GLM101.

Can children join this study?

Yes, children aged 4 years and older, as well as teenagers and adults, may be able to join.