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Trial record 1 of 4 for:    Pmm2-CDG
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24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Adults With PMM2-CDG

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05549219
Recruitment Status : Recruiting
First Posted : September 22, 2022
Last Update Posted : May 24, 2023
Sponsor:
Information provided by (Responsible Party):
Glycomine, Inc.

Study Description
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Brief 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 patients with a confirmed diagnosis of PMM2-CDG. The planned doses of GLM101 to be investigated are 10 and 20 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.

Condition or disease Intervention/treatment Phase
Pmm2-CDG Drug: GLM101 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Participants will assigned either 10mg/kg GLM101 or 20mg/kg GLM101 in a 1:1 ratio
Masking: None (Open Label)
Primary Purpose: Treatment
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 Participants With PMM2-CDG
Actual Study Start Date : November 29, 2022
Estimated Primary Completion Date : August 2023
Estimated Study Completion Date : September 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 10 mg/kg GLM101
GLM101 IV infusions, given weekly
 Drug: GLM101
GLM101 IV Infusion
Experimental: 20 mg/kg GLM101
GLM101 IV infusions, given weekly
 Drug: GLM101
GLM101 IV Infusion


Outcome Measures
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Primary Outcome Measures :
  1. Evaluate changes in coagulation and antithrombosis factors [ Time Frame: 12 weeks and 24 weeks ]
    Changes in AT-III activity level and factor XI activity


Secondary Outcome Measures :
  1. Number of participants who experience clinically significant change in pharmacodynamic activity by evaluating changes in hematology assessments [ Time Frame: 12 weeks and 24 weeks ]
    Evaluation of Pharmacodynamic activity

  2. Number of participants who experience clinically significant change in pharmacodynamic activity by evaluating changes in blood chemistry [ Time Frame: 12 weeks and 24 weeks ]
    Evaluation of Pharmacodynamic activity

  3. Number of participants with treatment-emergent adverse events assessed by severity and frequency [ Time Frame: 12 weeks and 24 weeks ]
    Safety and tolerability of multiple doses of GLM101

  4. Maximum observed plasma concentration (Cmax) [ Time Frame: 12 weeks and 24 weeks ]
    Assessment of the pharmacokinetics (PK) of GLM101

  5. Time to maximum observed plasma concentration (Tmax) [ Time Frame: 12 weeks and 24 weeks ]
    Assessment pharmacokinetics (PK) of GLM101

  6. Area under the plasma concentration vs. time curve (AUC) [ Time Frame: 12 weeks and 24 weeks ]
    Assessment of the pharmacokinetics (PK) of GLM101


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Is a male or female, 18 to 65 years of age, inclusive, at Screening;
  • Has been diagnosed with PMM2-CDG with genetic test confirmation;
  • Has antithrombin III (ATIII) levels below 80%;
  • If the participant is a female of childbearing potential, she must not be pregnant (confirmed by a negative serum pregnancy test), is using a medically accepted method of contraception (abstinence, a hormonal contraceptive in conjunction with a barrier method, double-barrier method, or use of an intrauterine device), and must agree to continue using this method for 30 days after the last infusion of GLM101;
  • 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 > 40 IU/L;
  • 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 barrier method, double-barrier method, or use by the partner of an intrauterine device) and agrees to continue using this method for 30 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 30 days after the last infusion of GLM101;
  • Is willing and able to provide informed consent/assent, directly or through his/her legally authorized representative.

Exclusion Criteria:

  • Diagnosis of congenital disorder of glycosylation (CDG) other than PMM2;
  • 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) infection or tests positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or check in to clinical site;
  • 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;
  • Has a history of liver transplant;
  • Has a history of drug or alcohol use disorder within 12 months from 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;
  • 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 Principal Investigator's and Medical Monitor's discretion;
  • 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, except for acetazolamide. Participants may be enrolled and continue treatment with acetazolamide only if they are on a stable dose for at least 30 days prior to dosing with GLM101, and the dose remains unchanged for the duration of the study.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05549219


Contacts
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Contact: Director Clinical Operations 650-264-7560 info@glycomine.com

Locations
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United States, Florida
Clinical Research of West Florida Recruiting
Tampa, Florida, United States, 33606
Contact: Courtney Bryan    813-870-1292    CBryan@crwf.com   
Principal Investigator: Lon Lynn, DO         
United States, Minnesota
University of Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55454
Contact: Sara Kramer       krame455@umn.edu   
Principal Investigator: Kyriakie Sarafoglou, MD         
Mayo Clinic Not yet recruiting
Rochester, Minnesota, United States, 55905
Contact: Kaitlin Schwartz    507-293-9114    Schwartz.Kaitlin@mayo.edu   
Principal Investigator: Eva Morava, MD PhD         
Australia, South Australia
Royal Adelaide Hospital Not yet recruiting
Adelaide, South Australia, Australia, 5000
Contact: Crystal Eldridge       Crystal.Eldridge@sa.gov.au   
Principal Investigator: Drago Bratkovic, MD         
Australia, Victoria
Royal Melbourne Hospital Not yet recruiting
Parkville, Victoria, Australia, 3050
Contact: Sarah Donoghue, MD       sarah.donoghue@mh.org.au   
Spain
Hospital Sant Joan de Déu Not yet recruiting
Barcelona, Spain, 08950
Contact: Mercedes Serrano, MD       mercedes.serrano@sjd.es   
Sponsors and Collaborators
Glycomine, Inc.
More Information
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Responsible Party: Glycomine, Inc.
ClinicalTrials.gov Identifier: NCT05549219    
Other Study ID Numbers: GLM101-002
First Posted: September 22, 2022    Key Record Dates
Last Update Posted: May 24, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Glycomine, Inc.:
Pmm2-CDG
Pmm2
Additional relevant MeSH terms:
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Congenital Disorders of Glycosylation
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases