Pmm2-CDGPhase 2NCT06657859
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
A Phase 2 study testing GLM101 in people with Pmm2-CDG and Phosphomannomutase 2 Deficiency, run by Glycomine, Inc. at 15 sites in 8 countries.
Trial summary
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and learn about the long term effect of GLM101. Participants will complete weekly infusions of GLM101 at the same dose level received in previous trials. Read more about this study
- Who is it for?
- Ages 2 years and older
- 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
- 90 participants
- Where
- 15 sitesin 8 countries
- Study dates
- 30 Sep 2024to Oct 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Is willing and able to provide informed consent/assent, directly or through a legally authorized representative.
- Has successfully completed the Treatment Period with GLM101 in a previous clinical study.
- At least 2 years of age, at the time of signing the informed consent form (ICF).
- 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 including from a prior parent trial is permitted;
- Male or female participant has appropriate measures in place to prevent pregnancy:
- 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)) or becomes of childbearing potential during the study, 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: True abstinence: 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 (such as calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
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- 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 (or becomes sexually active during the study) 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 comply with this protocol.
Who cannot join
- Participants who meet any of the following criteria will be excluded from participation in the study:
- Has any other condition that would, in the opinion of the Investigator, potentially compromise the safety or compliance of the participant or preclude the participant's successful completion of the study.
- 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.
- If not enrolling directly from a parent study (i.e., more than 28 days from Final Treatment visit in a parent study to date of consent), has an active infection requiring parenteral antibiotics, antivirals, or antifungals or treatment with systemic steroids within 7 days prior to Screening;
- 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) considered clinically significant;
- Has a history of liver transplant;
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- Has a history of drug or alcohol use disorder within the 12 months prior to Screening;
- If not enrolling directly from a parent study (i.e., if more than 28 days from Final Treatment visit in a parent study to date of consent), has had a major surgical procedure within 30 days prior to Screening;
- Has 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, and breastfeeding.
- Is currently participating in another interventional clinical study or has completed another clinical study with an investigational drug or device (other than GLM101) within 30 days or 5 half-lives before GLM101 infusion.
- Has a hypersensitivity to anti-histamine pre-medication.
- Has a history of a severe allergic reaction to any drug or excipients of GLM101 (as listed in the GLM101 IB);
- If not enrolling directly from a parent study (i.e., if more than 28 days from Final Treatment visit in a parent study to date of consent), has serology positive for hepatitis B surface antigen or hepatitis C antibody during Screening;
- 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;
- Weight exceeds 120 kg.
- Persons who have been committed to an institution by virtue of an order issued either by the judicial or the administrative authorities.
- Participant is unwilling or unable to comply with scheduled visits, study drug administration plan, laboratory tests, other study procedures, and study restrictions.
Trial locations and contacts
15 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
15 sites in 8 countries
United States4 sites
University of Minnesota
Minneapolis, Minnesota, 55455
The Icahn School of Medicine at Mount Sinai
New York, New York, 10029
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
Seattle Children's Hospital
Seattle, Washington, 98105
Spain3 sites
Hospital Sant Joan de Déu
Esplugues de Llobregat, 08950
Hospital Universitario 12 de Octubre - Unidad Pediatrica de Investigacion y Ensayos Clinicos (UPIC)
Madrid, 28041
Hospital Universitario 12 de Octubre
Madrid, 28041
Italy2 sites
Azienda Ospedaliero Universitaria Policlinico G. Rodolico-San Marco - Presidio Ospedaliero G. Rodolico
Catania, 95124
Azienda Ospedaliero Universitaria Pisana
Pisa, 56126
United Kingdom2 sites
Birmingham Children's Hospital
Birmingham, B4 6NH
Great Ormond Street Hospital for Children
London, WC1N 3JH
Portugal1 site
Unidade Local de Saúde de Santo António, E.P.E
Porto, 4099-001
About this study
Official title: A Phase 2, Open-Label Extension Study to Assess the Safety and Efficacy of GLM101 Administered Intravenously to Participants With PMM2-CDG
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and learn about the long term effect of GLM101. Participants will complete weekly infusions of GLM101 at the same dose level received in previous trials.
Read the detailed description
This is a phase 2 open-label clinical study of GLM101 in patients with PMM2-CDG who have previously participated in a study of GLM101. This study is designed to monitor long-term safety and treatment effect of GLM101 and provide continued access to study treatment. Participants will receive 30 mg/kg. Dose levels may be adjusted to lower doses or higher doses based on available data that demonstrates a change to be safe and favorable. Among other assessments, participants will be asked to complete questionnaires to evaluate changes in ataxia and quality of life.
- Conditions
- Pmm2-CDG, Phosphomannomutase 2 Deficiency
- Being tested
- GLM101
- Keywords
- GLM101, Pmm2, CDG, CDG 1a
- 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 |
|---|---|---|
| 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 long-term safety
Number of participants with treatment related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Measured: From enrollment to end of treatment up to 4 years
Secondary outcomes (4)
Evaluate changes in ataxia using International Cooperative Ataxia Rating Scale (ICARS)
The scale is scored out of 100 with 19 items and 4 subscales of postural and gait disturbances, limb ataxia, dysarthria, and oculomotor disorders. Higher scores indicate higher levels of impairment.
Measured: From enrollment, at 3 months, 6 months and annually to end of treatment up to 4 years
Maximum observed plasma concentration (Cmax)
Assessment of the pharmacokinetics (PK) of GLM101
Measured: From enrollment to end of treatment up to 4 years
Time to maximum observed plasma concentration (Tmax)
Assessment pharmacokinetics (PK) of GLM101
Measured: From enrollment to end of treatment up to 4 years
Area under the plasma concentration vs. time curve (AUC)
Assessment of the pharmacokinetics (PK) of GLM101
Measured: From enrollment to end of treatment up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2024
- Study start30 Sep 2024
- Primary completionSep 2029 (estimated)
- Study completionOct 2029 (estimated)
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
- Not applicable
- Intervention model
- Single group
- Model description
- This is an open-label extension study.
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Glycomine, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief Medical Officer, Study director, Glycomine, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06657859
- Sponsor's protocol ID
- GLM101-007
- Status last verified
- Jul 2026
- First submitted
- 22 Oct 2024
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label Extension Study to Assess the Safety and Efficacy of GLM101 Administered Intravenously to Participants With PMM2-CDG
- ClinicalTrials.gov ID
- NCT06657859
- Acronym / sponsor ID
- GLM101-007
- Conditions
- Pmm2-CDG, Phosphomannomutase 2 Deficiency
- Keywords
- GLM101, Pmm2, CDG, CDG 1a
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Glycomine, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Sep 2024 (actual)
- Primary completion
- Sep 2029 (estimated)
- Study completion
- Oct 2029 (estimated)
- First posted
- 26 Oct 2024 (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.
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and learn about the long term effect of GLM101. Participants will complete weekly infusions of GLM101 at the same dose level received in previous trials.
Detailed description
This is a phase 2 open-label clinical study of GLM101 in patients with PMM2-CDG who have previously participated in a study of GLM101. This study is designed to monitor long-term safety and treatment effect of GLM101 and provide continued access to study treatment. Participants will receive 30 mg/kg. Dose levels may be adjusted to lower doses or higher doses based on available data that demonstrates a change to be safe and favorable. Among other assessments, participants will be asked to complete questionnaires to evaluate changes in ataxia and quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an open-label extension study.
| Arm | Type | Receives |
|---|---|---|
| 30 mg/kg GLM101 | Experimental | Drug: GLM101GLM101 IV infusions, given weekly |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Is willing and able to provide informed consent/assent, directly or through a legally authorized representative.
- Has successfully completed the Treatment Period with GLM101 in a previous clinical study.
- At least 2 years of age, at the time of signing the informed consent form (ICF).
- 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 including from a prior parent trial is permitted;
- Male or female participant has appropriate measures in place to prevent pregnancy:
Key exclusion criteria (20 listed)
- Participants who meet any of the following criteria will be excluded from participation in the study:
- Has any other condition that would, in the opinion of the Investigator, potentially compromise the safety or compliance of the participant or preclude the participant's successful completion of the study.
- 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.
- If not enrolling directly from a parent study (i.e., more than 28 days from Final Treatment visit in a parent study to date of consent), has an active infection requiring parenteral antibiotics, antivirals, or antifungals or treatment with systemic steroids within 7 days prior to Screening;
- 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) considered clinically significant;
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 | 30 mg/kg GLM101 |
Primary outcome
Evaluate long-term safety
Time frame: From enrollment to end of treatment up to 4 years
Number of participants with treatment related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
4 secondary outcomes
Evaluate changes in ataxia using International Cooperative Ataxia Rating Scale (ICARS)
Time frame: From enrollment, at 3 months, 6 months and annually to end of treatment up to 4 years
Maximum observed plasma concentration (Cmax)
Time frame: From enrollment to end of treatment up to 4 years
Time to maximum observed plasma concentration (Tmax)
Time frame: From enrollment to end of treatment up to 4 years
Area under the plasma concentration vs. time curve (AUC)
Time frame: From enrollment to end of treatment up to 4 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2026.
Related trials for Pmm2-CDG
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
RecruitingObservational study
Clinical and Basic Investigations Into Congenital Disorders of Glycosylation
Congenital Disorders of Glycosylation
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.
Chief Medical Officer study director
Glycomine, Inc.
15 sites in 8 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 15 sites in 8 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 10 inclusion and 20 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: enrolling by invitation, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Spain, Italy, United Kingdom, Czechia and 3 more countries. 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?
This study is still under way. Results are not available in the source record yet.
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 long-term safety (time frame: From enrollment to end of treatment up to 4 years). 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 23 Oct 2026.