PMM2-CDG: Clinical Trials

3 trials for PMM2-CDG, newest first

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2 terminated, 1 active not recruiting. 3 Phase 3, 2 Phase 2.

By phase and status

PhaseTrialsStatus
Phase 33Terminated 2, Active not recruiting 1
Phase 22Active not recruiting 1, Terminated 1

Follow PMM2-CDG: new trials by email Literature Review of PMM2-CDG trials Research Report on PMM2-CDG

Trials (filtered) newest first · largest first

All 3 trials

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
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 3Active not recruitingGlycomine, Inc.502025-07-092026-08-14
Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG
Epalrestat, Placebo - Pmm2-CDG, Phosphomannomutase 2 Deficiency
Phase 3TerminatedMaggie's Pearl, LLC422022-11-102025-09-25
Acetazolamide Efficacy in Ataxia in PMM2-CDG
Placebo, Acetazolamide - Pmm2-CDG, CDG1A
Phase 2 / Phase 3TerminatedMayo Clinic252021-03-172025-03-20

Narrower conditions

Congenital disorder of glycosylation type 1A (3)  ·  Ataxia (1)

Broader conditions

Dyskinesias (1)  ·  Nervous System Diseases (1)  ·  Neurologic Manifestations (1)  ·  Pathological Conditions, Signs and Symptoms (1)  ·  Signs and Symptoms (1)

Interventions

Acetazolamide (1)  ·  Double-Blind Method (1)  ·  Gelatin (1)  ·  Lactose (1)  ·  epalrestat (1)

Sponsors

Glycomine, Inc. (1)  ·  Maggie's Pearl, LLC (1)  ·  Mayo Clinic (1)

Countries

United States (3)  ·  Belgium (1)  ·  Czechia (1)  ·  France (1)  ·  Germany (1)  ·  Italy (1)  ·  Poland (1)  ·  Portugal (1)  ·  Spain (1)  ·  United Kingdom (1)

Top investigators

Eva Morava-Kozicz, MD, PhD (2)