Phase 1 Mucopolysaccharidosis I Clinical Trials

3 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 1–3 of 3 trials

Recruiting

Phase 1

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Mucopolysaccharidosis IH/SMucopolysaccharidosis IS

Immusoft of CA, Inc.11 enrolled2 locationsNCT05682144

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