drugset / Trial / NCT05549219
24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG
RandomizedSequentialOpen-labelTreatment
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, adolescent, and pediatric, patients with a confirmed diagnosis of PMM2-CDG. The planned doses of GLM101 to be investigated are 10, 20, and 30 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.
Timeline
- Start
- 2022-11-29
- Primary completion
- 2025-11-04
- Completion
- 2025-11-18
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | GLM101 | Unknown | 10 mg/kg | Intravenous |
| Subject | GLM101 | Unknown | 20 mg/kg | Intravenous |
| Subject | GLM101 | Unknown | 30 mg/kg | Intravenous |