Overview

12-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Adults With PMM2-CDG

Status:
Not yet recruiting
Trial end date:
2023-07-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Glycomine, Inc.