Brain Diseases, Metabolic, Inborn Clinical Trials
2 recruiting trials for Brain Diseases, Metabolic, Inborn. Eligibility criteria explained in plain English.
2
Total Trials
2
Recruiting Now
0
Phase 3 Trials
2
Sponsors
Recruiting Trials
RECRUITINGPhase 1NCT02254863
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of...
Sponsor: Joanne Kurtzberg, MDEnrolling: 401 location
RECRUITINGNCT06337864
Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
The overall aim of this study is to evaluate LNAA treatment as a potential alternative to conventional dietary treatment for PKU. This study investigates the effects of LNAA...
Sponsor: Rigshospitalet, DenmarkEnrolling: 302 locations