Clinical trials: Pmm2-CDG
Phosphomannomutase 2 Deficiency, PMM2-CDG
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
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.
- Trial name
- NCT06892288
- Testing
- GLM101 (Part A, Double-blind), GLM101 (Part B, Open-label)
- Where
- 15 sites in United States, Belgium and 8 more
- Volunteers
- 50 people
- Sponsor
- Glycomine, Inc.
Pmm2-CDG, Phosphomannomutase 2 Deficiency
Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG
This is a prospective, single-center, randomized, double-blind, placebo-controlled study designed to assess the safety, tolerability, and clinical and metabolic improvement of pediatric subjects with PMM2-CDG on oral epalrestat…
- Conditions
- Pmm2-CDG, Phosphomannomutase 2 Deficiency, Phosphomannomutase 2 Congenital Disorder of Glycosylation, Phosphomannomutase II Congenital Disorder of Glycosylation
- Trial name
- NCT04925960
- Testing
- Epalrestat
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Maggie's Pearl, LLC
Congenital Disorders of Glycosylation
Clinical and Basic Investigations Into Congenital Disorders of Glycosylation
The purpose of this research is to study the natural history of congenital disorders of glycosylation and its causes and treatments.
- Trial name
- NCT04199000
- Where
- 12 sites in United States
- Volunteers
- 500 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Pmm2-CDG, Phosphomannomutase 2 Deficiency
24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG
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…
- Trial name
- NCT05549219
- Testing
- GLM101
- Where
- 4 sites in United States, Spain and 1 more
- Volunteers
- 27 people
- Sponsor
- Glycomine, Inc.
Phosphomannomutase 2 Deficiency
Natural History Study Protocol in PMM2-CDG (CDG-Ia)
Clinical and Basic Investigations into Phosphomannomutase deficiency (PMM2-CDG) This is a natural history (observational) protocol designed to collect clinical and biological information in patients with PMM2-CDG (CDG-Ia).
- Trial name
- NCT03173300
- Where
- 11 sites in United States, Belgium and 7 more
- Volunteers
- 139 people
- Sponsor
- Glycomine, Inc.
Pmm2-CDG, CDG1A
Acetazolamide Efficacy in Ataxia in PMM2-CDG
Objective 1 (Primary): To determine the efficacy of acetazolamide in improving ataxia in patients with PMM2-CDG. Objective 2 (Secondary): To evaluate for any adverse events related to longer term acetazolamide administration.
- Trial name
- NCT04679389
- Testing
- Acetazolamide
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Mayo Clinic
Pmm2-CDG, Phosphomannomutase 2 Deficiency
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and learn about the long term effect of GLM101.
- Trial name
- NCT06657859
- Testing
- GLM101
- Where
- 15 sites in United States, Czechia and 6 more
- Volunteers
- 90 people
- Sponsor
- Glycomine, Inc.