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A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE2/PHASE3

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

A Phase 2b, Multicenter, Double-blind, Randomized, Placebo Controlled Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 Administered Intravenously to Participants With PMM2-CDG (POLAR Trial)

NCT06892288•Glycomine, Inc.

View on ClinicalTrials.gov

Summary

This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults. Researchers will compare participants receiving GLM101 to those receiving a placebo to see if GLM101 improves symptoms of PMM2-CDG. 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

This Phase 2b, multicenter, randomized, double-blind, placebo-controlled clinical study is designed to evaluate the efficacy, safety, and pharmacokinetics (PK) of GLM101 in adult, adolescent, and pediatric participants with PMM2-CDG. The study is structured into: a 4-week Screening Period, a 24-week Double-blind Treatment Period (Part A) to assess primary efficacy, a 24-week Open-label Extension Period (Part B), and a safety follow-up visit conducted 4 weeks after the last infusion. In Part A, participants will be randomly assigned to receive weekly intravenous infusions of either GLM101 at 30 mg/kg or a placebo for 24 weeks. After the end of Part A, participants will transition into Part B, a 24-week open-label extension where all participants will receive GLM101. The primary objective of the trial is to identify changes from baseline in coordination and muscle movement (ataxia) using the International Co-operative Ataxia Rating Scale (ICARS) after 24 weeks of taking GLM101 compared to a placebo in PMM2-CDG patients.

Eligibility

Age

4 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Participant is aged ≥ 4 years old at the time of signing the consent.
•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.
+1 more criteria

Exclusion Criteria

•Has uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric or other significant disease based on the investigator judgment.
•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: ALT or AST \> 3 × ULN OR total bilirubin \> 2 × ULN or international normalized ratio (INR) \> 1.5 (if no anti-coagulation treatment) or INR \> 4 (if participant on anti-coagulation treatment).
+10 more criteria

Locations (15)

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

Key Dates

Start Date

July 9, 2025

Primary Completion Date

September 30, 2026

Completion Date

April 30, 2027

Last Updated

February 27, 2026

Enrollment

ESTIMATED participants

Conditions

Phosphomannomutase 2 DeficiencyPMM2-CDG

Interventions

GLM101 (Part A, Double-blind)

DRUG

Placebo (Part A, Double-blind)

DRUG

GLM101 (Part B, Open-label)

DRUG

Natural History Study Protocol in PMM2-CDG (CDG-Ia)

NCT03173300

Phosphomannomutase 2 Deficiency

View study

COMPLETEDPHASE2

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG

NCT05549219

Pmm2-CDGPhosphomannomutase 2 Deficiency

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 27, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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