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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05549219




Registration number
NCT05549219
Ethics application status
Date submitted
24/08/2022
Date registered
22/09/2022

Titles & IDs
Public title
24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG
Scientific 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
Secondary ID [1] 0 0
GLM101-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pmm2-CDG 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GLM101

Experimental: 10 mg/kg GLM101 - GLM101 IV infusions, given weekly

Experimental: 20 mg/kg GLM101 - GLM101 IV infusions, given weekly

Experimental: 30 mg/kg GLM101 - GLM101 IV infusions, given weekly


Treatment: Drugs: GLM101
GLM101 IV Infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Evaluate changes in coagulation and antithrombosis factors
Timepoint [1] 0 0
12 weeks and 24 weeks
Primary outcome [2] 0 0
Evaluate changes from baseline in ataxia
Timepoint [2] 0 0
12 weeks and 24 weeks
Secondary outcome [1] 0 0
Significant change in pharmacodynamic activity
Timepoint [1] 0 0
12 weeks and 24 weeks
Secondary outcome [2] 0 0
Number of participants with treatment-emergent adverse events assessed by severity and frequency
Timepoint [2] 0 0
12 weeks and 24 weeks
Secondary outcome [3] 0 0
Maximum observed plasma concentration (Cmax)
Timepoint [3] 0 0
12 weeks and 24 weeks
Secondary outcome [4] 0 0
Time to maximum observed plasma concentration (Tmax)
Timepoint [4] 0 0
12 weeks and 24 weeks
Secondary outcome [5] 0 0
Area under the plasma concentration vs. time curve (AUC)
Timepoint [5] 0 0
12 weeks and 24 weeks

Eligibility
Key inclusion criteria
* Is a male or female, 18 to 65 years of age, inclusive, at Screening; 12-17 years of age inclusive at Screening or 2-11 years of age, inclusive at Screening
* Has been diagnosed with PMM2-CDG with genetic test confirmation;
* 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.
Minimum age
2 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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) 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 >3x ULN and total bilirubin >2x ULN or INR >1.5 (abnormal biological values of liver function)
* 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 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.
* Weight exceeds 75 kg

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/11/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2025
Actual
Sample size
Target
44
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
Spain
State/province [3] 0 0
Barcelona

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Glycomine, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Director Clinical Operations
Address 0 0
Country 0 0
Phone 0 0
650-264-7560
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05549219