Pmm2-CDGPhase 2NCT05549219
24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG
A Phase 2 study testing GLM101 in people with Pmm2-CDG and Phosphomannomutase 2 Deficiency, run by Glycomine, Inc. at 4 sites in 3 countries.
Trial summary
This is a Phase 2, randomized, open-label, 24-week treatment study to evaluate the potential pharmacodynamic (PD) activity, safety, tolerability, and pharmacokinetics (PK) of GLM101 in adult, adolescent, and pediatric, patients with a confirmed diagnosis of PMM2-CDG. Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Pmm2-CDG, Phosphomannomutase 2 Deficiency
- What is being tested?
- GLM101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 29 Nov 2022to 18 Nov 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Is male or female, 18 to 65 years of age, inclusive, at Screening (Cohorts 1-3, 7), 12-17 years of age, inclusive, at Screening (Cohort 4) or 2-11 years of age, inclusive, at Screening (Cohorts 5 and 6);
- Molecularly confirmed 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 bi-allelic variants AND phosphomannomutase-2 (PMM2) enzyme activity consistent with a diagnosis of PMM2-CDG. Historical diagnosis with lab report(s) on file is permitted;
- If the participant is a female of childbearing potential (i.e. fertile, following menarche and until becoming post-menopausal 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 of GLM101; 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 subject. 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 pre-pubertal, surgically sterile, or must have an ovarian dysfunction confirmed by a follicle stimulating hormone (FSH) >40 IU/L and absence of menses for 12 months without an alternative medical cause;
- If the participant is a sexually active male with female partners, the sexually mature, nonsterile male participant agrees to use a medically acceptable method of contraception (abstinence, the partner taking a hormonal contraceptive in conjunction with a male condom, or use by the partner of an intrauterine device with a male condom) and agrees to continue using this method for 50 days after the last infusion of GLM101. Males are considered surgically sterile if they have undergone bilateral orchiectomy or vasectomy at least 3 months prior to Screening;
- If the participant is male, he must agree to refrain from donating sperm during the study and 50 days after the last infusion of GLM101;
- Is willing and able to provide informed consent/assent, directly or through his/her legally authorized representative.
Who cannot join
- 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 bi-allelic variants AND the defined CDG enzyme activity consistent with a diagnosis of the CDG other than PMM2 CDG.
- Has an active infection requiring parenteral antibiotics, antivirals, or antifungals or treatment with systemic steroids within 7 days prior to Screening;
- Has confirmed active coronavirus disease-2019 (COVID-19) or tests positive for severe acute respiratory syndrome coronavirus 2 (SARS CoV 2) at Screening or check in to the clinical site;
- ALT or AST >3× ULN OR total bilirubin >2× ULN or INR >1.5
- Has a history of a severe allergic reaction to any drug or excipients of GLM101 (as listed in the GLM101 Investigator's Brochure);
- Has a known history of poor venous access;
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- Has a history of liver transplant;
- 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;
- Has Screening or eligibility confirmation laboratory value(s) outside the laboratory reference range considered clinically significant and not related to PMM2-CDG;
- If female, has a positive serum pregnancy test during Screening;
- If female, must not be breastfeeding;
- Has serology positive for hepatitis B surface antigen or hepatitis C antibody during Screening;
- 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 Medical Monitor's discretion;
- Has a QTc ≥ 450 ms, or other clinically significant ECG abnormalities;
- Has uncontrolled cardiovascular, hepatic, pulmonary, gastro-intestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric or other significant disease;
- 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 before GLM101 infusion.
- Weight exceeds 75 kg.
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
United States2 sites
Mayo Clinic
Rochester, Minnesota, 55905
Clinical Research of West Florida
Tampa, Florida, 33606
About this study
Official title: A Phase 2, Randomized, Open-Label, 24-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 Administered Intravenously to Adult, Adolescent, and Pediatric Participants With PMM2-CDG
This is a Phase 2, randomized, open-label, 24-week treatment study to evaluate the potential pharmacodynamic (PD) activity, safety, tolerability, and pharmacokinetics (PK) of GLM101 in adult, adolescent, and pediatric, patients with a confirmed diagnosis of PMM2-CDG. The planned doses of GLM101 to be investigated are 10, 20, and 30 mg/kg. The study will consist of a Screening Period, a 24-week (6-month) Treatment Period, and a 30-day (1-month) Follow-Up Period.
- Conditions
- Pmm2-CDG, Phosphomannomutase 2 Deficiency
- Being tested
- GLM101
- Keywords
- Pmm2-CDG, Pmm2, CDG, GLM101
- Medical subject headings
- Congenital disorder of glycosylation type 1A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GLM101Drug
GLM101 IV Infusion
| Study group | Role | What they receive |
|---|---|---|
| 10 mg/kg GLM101 | Experimental | GLM101 IV infusions, given weekly Receives: Drug: GLM101 |
| 20 mg/kg GLM101 | Experimental | GLM101 IV infusions, given weekly Receives: Drug: GLM101 |
| 30 mg/kg GLM101 | Experimental | GLM101 IV infusions, given weekly Receives: Drug: GLM101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate changes from baseline in ataxia
Changes in ICARS (International Co-operative Ataxia Rating Scale)
Measured: 12 weeks and 24 weeks
Secondary outcomes (4)
Number of participants with treatment-emergent adverse events assessed by severity and frequency
Evaluate Safety and tolerability of multiple doses of GLM101. Collection and assessment of adverse events, adverse event of special interest, serious adverse events, deaths, discontinuations, clinical laboratory tests, vital signs and physical exam.
Measured: 12 weeks and 24 weeks
Maximum observed plasma concentration (Cmax)
Assessment of the pharmacokinetics (PK) of GLM101
Measured: over 24 weeks
Time to maximum observed plasma concentration (Tmax)
Assessment pharmacokinetics (PK) of GLM101
Measured: over 24 weeks
Area under the plasma concentration vs. time curve (AUC)
Assessment of the pharmacokinetics (PK) of GLM101
Measured: over 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2022
- Study start29 Nov 2022
- Primary completion4 Nov 2025
- Study completion18 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pmm2-CDGStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- Participants will randomized to receive either 10 mg/kg GLM101 or 20 mg/kg GLM101. Subsequently, if safety and tolerability are demonstrated at 10 and 20 mg/kg, 30 mg/kg groups will be added.
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Glycomine, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05549219
- Sponsor's protocol ID
- GLM101-002
- Status last verified
- Dec 2025
- First submitted
- 24 Aug 2022
- Last updated
- 17 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Open-Label, 24-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 Administered Intravenously to Adult, Adolescent, and Pediatric Participants With PMM2-CDG
- ClinicalTrials.gov ID
- NCT05549219
- Acronym / sponsor ID
- GLM101-002
- Conditions
- Pmm2-CDG, Phosphomannomutase 2 Deficiency
- Keywords
- Pmm2-CDG, Pmm2, CDG, GLM101
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Glycomine, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Nov 2022 (actual)
- Primary completion
- 4 Nov 2025 (actual)
- Study completion
- 18 Nov 2025 (actual)
- First posted
- 22 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2, randomized, open-label, 24-week treatment study to evaluate the potential pharmacodynamic (PD) activity, safety, tolerability, and pharmacokinetics (PK) of GLM101 in adult, adolescent, and pediatric, patients with a confirmed diagnosis of PMM2-CDG. The planned doses of GLM101 to be investigated are 10, 20, and 30 mg/kg. The study will consist of a Screening Period, a 24-week (6-month) Treatment Period, and a 30-day (1-month) Follow-Up Period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Participants will randomized to receive either 10 mg/kg GLM101 or 20 mg/kg GLM101. Subsequently, if safety and tolerability are demonstrated at 10 and 20 mg/kg, 30 mg/kg groups will be added.
| Arm | Type | Receives |
|---|---|---|
| 10 mg/kg GLM101 | Experimental | Drug: GLM101GLM101 IV infusions, given weekly |
| 20 mg/kg GLM101 | Experimental | Drug: GLM101GLM101 IV infusions, given weekly |
| 30 mg/kg GLM101 | Experimental | Drug: GLM101GLM101 IV infusions, given weekly |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Is male or female, 18 to 65 years of age, inclusive, at Screening (Cohorts 1-3, 7), 12-17 years of age, inclusive, at Screening (Cohort 4) or 2-11 years of age, inclusive, at Screening (Cohorts 5 and 6);
- Molecularly confirmed 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 bi-allelic variants AND phosphomannomutase-2 (PMM2) enzyme activity consistent with a diagnosis of PMM2-CDG. Historical diagnosis with lab report(s) on file is permitted;
- If the participant is a female of childbearing potential (i.e. fertile, following menarche and until becoming post-menopausal 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 of GLM101; 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 subject. 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 pre-pubertal, surgically sterile, or must have an ovarian dysfunction confirmed by a follicle stimulating hormone (FSH) >40 IU/L and absence of menses for 12 months without an alternative medical cause;
- If the participant is a sexually active male with female partners, the sexually mature, nonsterile male participant agrees to use a medically acceptable method of contraception (abstinence, the partner taking a hormonal contraceptive in conjunction with a male condom, or use by the partner of an intrauterine device with a male condom) and agrees to continue using this method for 50 days after the last infusion of GLM101. Males are considered surgically sterile if they have undergone bilateral orchiectomy or vasectomy at least 3 months prior to Screening;
Key exclusion criteria (18 listed)
- 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 bi-allelic variants AND the defined CDG enzyme activity consistent with a diagnosis of the CDG other than PMM2 CDG.
- Has an active infection requiring parenteral antibiotics, antivirals, or antifungals or treatment with systemic steroids within 7 days prior to Screening;
- Has confirmed active coronavirus disease-2019 (COVID-19) or tests positive for severe acute respiratory syndrome coronavirus 2 (SARS CoV 2) at Screening or check in to the clinical site;
- ALT or AST >3× ULN OR total bilirubin >2× ULN or INR >1.5
- Has a history of a severe allergic reaction to any drug or excipients of GLM101 (as listed in the GLM101 Investigator's Brochure);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GLM101GLM101 IV Infusion | Drug | 10 mg/kg GLM101, 20 mg/kg GLM101, 30 mg/kg GLM101 |
Primary outcome
Evaluate changes from baseline in ataxia
Time frame: 12 weeks and 24 weeks
Changes in ICARS (International Co-operative Ataxia Rating Scale)
4 secondary outcomes
Number of participants with treatment-emergent adverse events assessed by severity and frequency
Time frame: 12 weeks and 24 weeks
Maximum observed plasma concentration (Cmax)
Time frame: over 24 weeks
Time to maximum observed plasma concentration (Tmax)
Time frame: over 24 weeks
Area under the plasma concentration vs. time curve (AUC)
Time frame: over 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2025.
- Individual participant dataSharing planned: No
Related trials for Pmm2-CDG
Enrolling by invitationPhase 2
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
Pmm2-CDG, Phosphomannomutase 2 Deficiency
Active, not recruitingPhase 2/3
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
Phosphomannomutase 2 Deficiency, PMM2-CDG
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GLM101 in people with Pmm2-CDG and Phosphomannomutase 2 Deficiency, run by Glycomine, Inc. at 4 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate changes from baseline in ataxia (time frame: 12 weeks and 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2025; AzTrials last refreshed its copy on 26 Oct 2026.