A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
NCT ID: NCT06892288
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
50 participants
INTERVENTIONAL
2025-07-09
2026-08-31
Brief Summary
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The study includes two treatment parts: a 24-week double blind placebo-controlled treatment period (Part A), and a 24-week open-label phase where every participant will receive GLM101(Part B).
Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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GLM101- 30 mg/kg weekly administered IV in Part A (double-blind) and Part B (open-label)
GLM101 (Part A, Double-blind)
IV infusions, 30 mg/kg once weekly for 24 weeks, for participants randomized to GLM101 in Part A
GLM101 (Part B, Open-label)
IV infusions, 30 mg/kg once weekly from week 25 to 48, to all participants
Placebo weekly admin. IV in Part A (double-blind); 30 mg/kg weekly admin. IV in Part B (open-label)
Placebo (Part A, Double-blind)
IV infusions, 30 mg/kg once weekly for 24 weeks, for participants randomized to Placebo in Part A
GLM101 (Part B, Open-label)
IV infusions, 30 mg/kg once weekly from week 25 to 48, to all participants
Interventions
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GLM101 (Part A, Double-blind)
IV infusions, 30 mg/kg once weekly for 24 weeks, for participants randomized to GLM101 in Part A
Placebo (Part A, Double-blind)
IV infusions, 30 mg/kg once weekly for 24 weeks, for participants randomized to Placebo in Part A
GLM101 (Part B, Open-label)
IV infusions, 30 mg/kg once weekly from week 25 to 48, to all participants
Eligibility Criteria
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Inclusion Criteria
* 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.
* Participant is willing and capable of completing the ICARS in its entirety without any assessment deemed as "not evaluable".
* Participant screening total ICARS score is ≥ 20 and ≤ 80 .
* Male or female participant has appropriate measures in place to prevent pregnancy:
* If the participant is a woman of childbearing potential, i.e., fertile, following menarche and until becoming postmenopausal unless permanently sterile (permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy), she must not be pregnant (confirmed by a negative serum pregnancy test), is using a medically accepted method of contraception (abstinence, a hormonal contraceptive associated with inhibition of ovulation in conjunction with a barrier method, or use of an intrauterine device), and must agree to continue using this method for 50 days after the last infusion. Note: sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the participant. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception.
* If the participant is a female of non-childbearing potential, she must be premenarchal, surgically sterile, or must have an ovarian dysfunction confirmed by a follicle stimulating hormone \> 40 IU/ L (or higher per local institutional guidelines) and absence of menses for 12 months after last menstrual bleeding without an alternative medical cause.
* If the participant is a sexually active male with female partners, the participant agrees to use a medically acceptable method of contraception (abstinence, the partner taking a hormonal contraceptive in conjunction with male participant using male condom, or use by the partner of an intrauterine device with a male participant using male condom) and agrees to continue using this method for 50 days after the last infusion.
* If the participant is male, he must agree to refrain from donating sperm during the study and 50 days after the last infusion.
* The participant is willing and able to provide informed consent/assent, directly or through his/her legally authorized representative.
* The participant has a caregiver who is willing and able to complete questionnaires and provide informed consent.
Exclusion Criteria
* Diagnosis of congenital disorder of glycosylation (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.
* Has a history of liver transplant.
* Has an active infection requiring parenteral antibiotics, antivirals, antifungals or treatment with systemic steroids within 7 days prior to screening.
* Has a history of drug or alcohol use disorder within 12 months prior to screening.
* Has had a major surgical procedure within 30 days prior to screening or an upcoming planned major surgery.
* Previous history of GLM101 administration.
* 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 half-lives (whichever is longer) before enrollment.
* Have consumed products or supplements containing mannose or biotin within 2 weeks prior to screening.
* Elevated liver function tests: alanine aminotransferase or aspartate aminotransferase \> 3 × upper limit of normal (ULN) OR total bilirubin \> 2 × ULN or international normalized ratio \> 1.5.
* Has screening laboratory value(s) considered clinically significant and not related to PMM2-CDG based on the investigator judgment.
* Has serology positive for hepatitis B surface antigen or hepatitis C antibody during screening.
* Has a QT interval by Fridericia (QTcF) ≥ 450 ms, or other electrocardiogram abnormalities judged as clinically significant by the investigator.
* 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.
* Participant weighs above 75 kg.
* Participant has a known or suspected hypersensitivity to GLM101 or any components of the formulation used.
* Any other reason for which, in the investigator's opinion, makes the participant unsuitable for study participation.
4 Years
ALL
No
Sponsors
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Glycomine, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Chief Medical Officer
Role: STUDY_DIRECTOR
Glycomine, Inc.
Locations
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University of Minnesota
Minneapolis, Minnesota, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Seattle Children's Hospital
Seattle, Washington, United States
UZ Leuven, Campus Gasthuisberg
Leuven, , Belgium
Vseobecna fakultni nemocnice v Praze
Prague, , Czechia
AP-HP Hopital Necker-Enfants Malades
Paris, , France
Universitaetsklinikum Muenster
Münster, , Germany
Azienda Ospedaliero Universitaria Policlinico G. Rodolico-San Marco
Catania, , Italy
Azienda Ospedaliero Universitaria Pisana
Pisa, , Italy
Instytut Matki i Dziecka
Warsaw, , Poland
Unidade Local de Saúde de Santo António
Porto, , Portugal
Hospital Sant Joan de Déu
Esplugues de Llobregat, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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GLM101-003
Identifier Type: -
Identifier Source: org_study_id