2 terminated, 1 active not recruiting, 1 completed, 1 enrolling by invitation. 4 Phase 2, 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 1, Completed 1, Enrolling by invitation 1, Terminated 1 |
| Phase 3 | 3 | Terminated 2, Active not recruiting 1 |
Follow PMM2-CDG: new trials by email Literature Review of PMM2-CDG trials Research Report on PMM2-CDG
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG GLM101 (Part A, Double-blind), Placebo (Part A, Double-blind), GLM101 (Part B, Open-label) - Phosphomannomutase 2 Deficiency, PMM2-CDG | Phase 2 / Phase 3 | Active not recruiting | Glycomine, Inc. | 50 | 2025-07-09 | 2026-08-14 |
| Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients GLM101 - Pmm2-CDG, Phosphomannomutase 2 Deficiency | Phase 2 | Enrolling by invitation | Glycomine, Inc. | 90 | 2024-09-30 | 2026-10-23 |
| 24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG GLM101 - Pmm2-CDG, Phosphomannomutase 2 Deficiency | Phase 2 | Completed | Glycomine, Inc. | 27 | 2022-11-29 | 2025-12-17 |
| Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG Epalrestat, Placebo - Pmm2-CDG, Phosphomannomutase 2 Deficiency | Phase 3 | Terminated | Maggie's Pearl, LLC | 42 | 2022-11-10 | 2025-09-25 |
| Acetazolamide Efficacy in Ataxia in PMM2-CDG Placebo, Acetazolamide - Pmm2-CDG, CDG1A | Phase 2 / Phase 3 | Terminated | Mayo Clinic | 25 | 2021-03-17 | 2025-03-20 |
Congenital disorder of glycosylation type 1A (5) · Ataxia (1)
Dyskinesias (1) · Nervous System Diseases (1) · Neurologic Manifestations (1) · Pathological Conditions, Signs and Symptoms (1) · Signs and Symptoms (1)
Acetazolamide (1) · Double-Blind Method (1) · Gelatin (1) · Lactose (1) · epalrestat (1)
Glycomine, Inc. (3) · Maggie's Pearl, LLC (1) · Mayo Clinic (1)
United States (5) · Spain (3) · United Kingdom (3) · Czechia (2) · France (2) · Germany (2) · Italy (2) · Portugal (2) · Belgium (1) · Poland (1)