GLM101 for Phosphomannomutase 2 Deficiency

Recruiting at 12 trial locations

Overseen By Director Clinical Operations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Glycomine, Inc.

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults. Researchers will compare participants receiving GLM101 to those receiving a placebo to see if GLM101 improves symptoms of PMM2-CDG. The study includes two treatment parts: a 24-week double blind placebo-controlled treatment period (Part A), and a 24-week open-label phase where every participant will receive GLM101(Part B).

Research Team

Chief Medical Officer

Principal Investigator

Glycomine, Inc.

Eligibility Criteria

This trial is for individuals, including children (aged ≥4), with PMM2-CDG—a rare genetic disorder. Participants must have a confirmed molecular diagnosis and be able to complete specific assessments. Women of childbearing potential must use contraception and not be pregnant; men must agree to use contraception.

Inclusion Criteria

I can give my consent or have someone legally authorized to do so on my behalf.

I have a caregiver willing to help with questionnaires and consent.

My ICARS score is between 20 and 80.

Treatment Details

Interventions

• GLM101 (Monoclonal Antibodies)

Trial Overview The study tests GLM101's safety and effectiveness in improving PMM2-CDG symptoms over a 24-week double-blind phase comparing it with placebo, followed by another 24 weeks where all participants receive GLM101 openly.

Participant Groups

Group I: GLM101- 30 mg/kg weekly administered IV in Part A (double-blind) and Part B (open-label) Experimental Treatment 2 Interventions

Group II: Placebo weekly admin. IV in Part A (double-blind); 30 mg/kg weekly admin. IV in Part B (open-label) Placebo Group 2 Interventions

Who Is Running the Clinical Trial?

Glycomine, Inc.

Lead Sponsor

Trials

4

Recruited

250+