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Clinical Trials

Status Study Identifier Study Name Disorders / CDG Type(s) Therapeutic approach Eligibility Study Type Industry / Institution(s) Location Contact Persons Direct Link
Upcoming Trial A Multicenter, Open-Label, Withdrawal and Treatment Trial Assessing the Efficacy, Safety, and Tolerability of 2 Doses of CERC-801 in Subjects with Phosphoglucomutase-1 Deficiency Related Congenital Disorders of Glycosylation (PGM1-CDG) PGM1-CDG Drug: CERC-801 D-galactose supplement; Administration: Oral Diagnosis of PGM1-CDG Interventional Phase 2 Mayo Clinic/CHOP

United States

Eva Morava-Kozicz, MD, PhD
Dr. Andrew Edmondson, MD, PhD
Active, Recruiting NCT02089789 Clinical and basic investigations into known and suspected Congenital Disorders of Glycosylation CDG None People age 1-80 with CDG or suspected to have a CDG Observational National Institutes of Health Clinical Center

Maryland, MD
United States

Lynne A Wolfe
C.R.N.P.
+1 (301) 443-8577
lynne.wolfe@nih.gov
Completed NCT03250728 Role of the endothelium in stroke-like episode among CDG Patients (PECDG) CDG None Patients older than 6 years with CDG molecularly diagnosed Interventional Collection of blood by peripheral blood puncture Hopital Necker enfants malades

Paris
France

Pascale De Lonlay, MD, PhD
Active, Not Recruiting NCT04201067 Large-Scale Metabolomic Profiling for the Diagnosis of Inborn Errors of Metabolism ALG13-CDG
PGM1-CDG
SLC35A2-CDG
None CDG patients all ages that have agreed to share samples/data Observational and retrospective Mayo Clinic

Rochester, MN
United States

Eva Morava-Kozicz, MD, PhD
+1 507-266-2967
morava-kozicz.eva@mayo.edu

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