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A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants with PMM2-CDG
EU CTIS ID: 2024-520109-37-00
What this study is testing
To characterize the change from Baseline in ataxia at 24 weeks, comparing GLM101 to placebo in participants with PMM2-CDG as assessed by ICARS
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1.Participant is aged ≥ 4 years old at the time of signing the consent. Note: At clinical site(s) in Czechia, participants must be aged ≥12 years, per Czech national authority requirements
- 2.Participant with molecular diagnosis of PMM2-CDG. Diagnosis is defined as biallelic pathogenic and/or likely pathogenic variants, or, in the case of variants of uncertain pathogenicity, demonstration of biallelic variants and PMM2 enzyme activity consistent with a diagnosis of PMM2-CDG. Diagnosis with laboratory report(s) on file is required.
- 3.Participant is willing and capable of completing the ICARS in its entirety without any assessment deemed as “not evaluable”.
- 4.Participant screening total ICARS score is ≥ 20 and ≤ 80
- 5.Male or female participant has appropriate measures in place to prevent pregnancy
- 6.If the participant is male, he must agree to refrain from donating sperm during the study and 50 days after the last infusion
You likely can't join if
- 1.Has uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric or other significant disease based on the investigator judgment
- 2.Diagnosis of CDG other than PMM2; Diagnosis is defined as biallelic pathogenic and/or likely pathogenic variants, or, in the case of variants of uncertain pathogenicity, demonstration of biallelic variants and the defined CDG enzyme activity consistent with a diagnosis of the CDG other than PMM2 CDG
- 3.Has a history of liver transplant
- 4.Has an active infection requiring parenteral antibiotics, antivirals, antifungals or treatment with systemic steroids within 7 days prior to screening
- 5.Has a history of drug or alcohol use disorder within 12 months prior to screening
- 6.Has had a major surgical procedure within 30 days prior to screening or an upcoming planned major surgery
See the full eligibility criteria
Who can join
- 1.Participant is aged ≥ 4 years old at the time of signing the consent. Note: At clinical site(s) in Czechia, participants must be aged ≥12 years, per Czech national authority requirements
- 2.Participant with molecular diagnosis of PMM2-CDG. Diagnosis is defined as biallelic pathogenic and/or likely pathogenic variants, or, in the case of variants of uncertain pathogenicity, demonstration of biallelic variants and PMM2 enzyme activity consistent with a diagnosis of PMM2-CDG. Diagnosis with laboratory report(s) on file is required.
- 3.Participant is willing and capable of completing the ICARS in its entirety without any assessment deemed as “not evaluable”.
- 4.Participant screening total ICARS score is ≥ 20 and ≤ 80
- 5.Male or female participant has appropriate measures in place to prevent pregnancy
- 6.If the participant is male, he must agree to refrain from donating sperm during the study and 50 days after the last infusion
- 7.The participant is willing and able to provide informed consent/assent, directly or through his/her legally authorized representative (LAR)
- 8.The participant has a caregiver who is willing and able to complete questionnaires and provide the informed consent
What rules you out
- 1.Has uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric or other significant disease based on the investigator judgment
- 2.Diagnosis of CDG other than PMM2; Diagnosis is defined as biallelic pathogenic and/or likely pathogenic variants, or, in the case of variants of uncertain pathogenicity, demonstration of biallelic variants and the defined CDG enzyme activity consistent with a diagnosis of the CDG other than PMM2 CDG
- 3.Has a history of liver transplant
- 4.Has an active infection requiring parenteral antibiotics, antivirals, antifungals or treatment with systemic steroids within 7 days prior to screening
- 5.Has a history of drug or alcohol use disorder within 12 months prior to screening
- 6.Has had a major surgical procedure within 30 days prior to screening or an upcoming planned major surgery
- 7.Previous history of GLM101 administration
- 8.Is currently participating in another interventional clinical study or has completed another clinical study with an investigational drug or device within 30 days or 5 halflives (whichever is longer) before enrollment.
- 9.Have consumed products or supplements containing mannose or biotin within 2 weeks prior to screening
- 18.If female, must not be breastfeeding
- 19.Estimated glomerular filtration rate (eGFR) <45 mL/min/ 1.73 m2 calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation for participants ≥ 18 years or Schwartz equation for participants <18 years of age at screening
- 10.Elevated liver function tests: ALT or AST > 3 × ULN OR total bilirubin > 2 × ULN or INR > 1.5 (if no anti-coagulation treatment) or INR > 4 (if participant on anti-coagulation treatment)
- 20.Persons who have been committed to an institution by virtue of an order issued either by the judicial or the administrative authorities
- 11. Has screening laboratory value(s) considered clinically significant and not related to PMM2-CDG based on the investigator judgment
- 12. Has serology positive for hepatitis B surface antigen or hepatitis C antibody during screening
- 13.Has a QT interval by Fridericia (QTcF) ≥ 450 ms, or other electrocardiogram (ECG) abnormalities judged as clinically significant by the investigator
- 14.Has history or presence, upon clinical evaluation, of any illness that might impact the safety of GLM101 infusion or evaluability of drug effect based on the investigator’s and Sponsor’s Medical Monitor’s discretion
- 15.Participant weighs above 120 kg
- 16.Participant has a known or suspected hypersensitivity to GLM101 or any components of the formulation used
- 17.Any other reason for which, in the investigator’s opinion, makes the participant unsuitable for study participation
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.