GLM101 for Pmm2-CDG

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
University of Minnesota, Minneapolis, MN
Pmm2-CDG+1 More
GLM101 - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This is a Phase 2, randomized, open-label, 12-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 12-week (3-month) Treatment Period, and a 30-day (1-month) Follow-Up Period.

Eligible Conditions

  • Pmm2-CDG

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Pmm2-CDG

Study Objectives

1 Primary · 6 Secondary · Reporting Duration: 12 weeks

12 weeks
Evaluate changes in coagulation and antithrombosis factors
Number of participants who experience clinically significant change in pharmacodynamic activity by evaluating changes in blood chemistry
Number of participants who experience clinically significant change in pharmacodynamic activity by evaluating changes in hematology assessments
Number of participants with treatment-emergent adverse events assessed by severity and frequency
4 weeks
Area under the plasma concentration vs. time curve (AUC)
Maximum observed plasma concentration (Cmax)
Time to maximum observed plasma concentration (Tmax)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Pmm2-CDG

Trial Design

2 Treatment Groups

20 mg/kg GLM101
1 of 2
10 mg/kg GLM101
1 of 2
Experimental Treatment

6 Total Participants · 2 Treatment Groups

Primary Treatment: GLM101 · No Placebo Group · Phase 2

20 mg/kg GLM101
Drug
Experimental Group · 1 Intervention: GLM101 · Intervention Types: Drug
10 mg/kg GLM101
Drug
Experimental Group · 1 Intervention: GLM101 · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 12 weeks
Closest Location: University of Minnesota · Minneapolis, MN
Photo of University of Minnesota  1Photo of University of Minnesota  2Photo of University of Minnesota 3
1993First Recorded Clinical Trial
1 TrialsResearching Pmm2-CDG
830 CompletedClinical Trials

Who is running the clinical trial?

Glycomine, Inc.Lead Sponsor
1 Previous Clinical Trials
120 Total Patients Enrolled
Horacio Plotkin, MDStudy DirectorGlycomine, Inc.

Eligibility Criteria

Age 18+ · All Participants · 8 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are a male or female, 18 to 65 years of age, inclusive, at Screening.
You have been diagnosed with PMM2-CDG with genetic test confirmation.
You are a female of childbearing potential, aged 18 years or older, and you are not pregnant, using a medically accepted method of contraception, and you agree to continue using this method for 30 days after the last infusion of GLM101.
You are female, aged 18 years or older, and surgically sterile.
You are willing and able to provide informed consent/assent, directly or through his/her legally authorized representative.
You have antithrombin III (ATIII) levels below 80%.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.