MPS II (mucopolysaccharidosis type II, Hunter syndrome) is an X-linked lysosomal storage disorder caused by iduronate-2-sulfatase deficiency, resulting in progressive glycosaminoglycan accumulation and multisystem involvement.
A class action lawsuit was filed against REGENXBIO covering the period from February 9, 2022 to January 27, 2026, following FDA clinical holds on gene therapies RGX-111 and RGX-121 after a CNS tumor was discovered in a trial participant.
The FDA placed clinical holds on Regenxbio's RGX-111 and RGX-121 gene therapies following a tumor case in an MPS I patient. The company's stock fell nearly 18% on the news.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07344376 |
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) |
COMPLETED | PHASE3 |
| NCT07236606 |
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) |
SUSPENDED | PHASE3 |
| NCT04976231 |
MPS II Immunophenotyping |
TERMINATED | |
| NCT04532047 |
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) |
RECRUITING | PHASE1 |
| NCT03333200 |
Longitudinal Study of Neurodegenerative Disorders |
RECRUITING | |
| NCT03041324 |
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II |
TERMINATED | PHASE1/PHASE2 |
| NCT00607386 |
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy |
COMPLETED | PHASE4 |
