Phase 1 Mucopolysaccharidosis II Clinical Trials
2 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–2 of 2 trials
Recruiting
Phase 1
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Batten DiseaseAdrenoleukodystrophyPelizaeus-Merzbacher Disease+9 more
Joanne Kurtzberg, MD40 enrolled1 locationNCT02254863
Recruiting
Phase 1Phase 2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
JCR Pharmaceuticals Co., Ltd.10 enrolled3 locationsNCT06488924