Trials / Conditions / Metabolism, Inborn Errors

Metabolism, Inborn Errors

18 registered clinical trials studyying Metabolism, Inborn Errors — 2 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	A Phase 3 Study of NTLA-2001 in ATTRv-PN NCT06672237	Intellia Therapeutics	Phase 3
Recruiting	Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria NCT06337864	Rigshospitalet, Denmark	N/A
Completed	Cardiac Manifestation and Inherited Metabolic Diseases NCT04999566	University of Lausanne	—
Completed	Genetic Etiology in Patients With Cerebral Palsy NCT05123768	University Medical Centre Ljubljana	—
Unknown	COVID-19 and Hereditary Metabolic Diseases NCT04645498	University Hospital, Lille	—
Withdrawn	Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia NCT03810690	ModernaTX, Inc.	Phase 1 / Phase 2
Withdrawn	Biomarker for Hurler Disease (BioHurler) NCT02298712	CENTOGENE GmbH Rostock	—
Completed	North Carolina Newborn Exome Sequencing for Universal Screening NCT02826694	University of North Carolina, Chapel Hill	N/A
Unknown	Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy NCT02635269	Rigshospitalet, Denmark	N/A
Terminated	Natural History Study of Children With Metachromatic Leukodystrophy NCT01963650	Shire	—
Completed	An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patie NCT00694109	Kastle Therapeutics, LLC	Phase 3
Completed	Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemi NCT00607373	Kastle Therapeutics, LLC	Phase 3
Completed	Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia NCT00477594	Kastle Therapeutics, LLC	Phase 2
Completed	Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration NCT00362180	Kastle Therapeutics, LLC	Phase 2
Completed	Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia NCT00099996	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3
Completed	Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marr NCT00010361	Fred Hutchinson Cancer Center	N/A
Completed	Study of Phosphatidylcholine in a Patient With Methionine Adenosyltransferase Deficiency NCT00006061	National Center for Research Resources (NCRR)	N/A
Completed	Diagnostic and Screening Study of Genetic Disorders NCT00006057	National Center for Research Resources (NCRR)	—