Hurler syndrome is the severe form of mucopolysaccharidosis type I, a lysosomal storage disorder caused by alpha-L-iduronidase deficiency. It leads to progressive multisystem manifestations including skeletal abnormalities, organomegaly, cardiorespiratory complications, and neurocognitive impairment. Early diagnosis is critical because disease-modifying options are time-sensitive.
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 |
|---|---|---|---|
| NCT03639844 |
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study |
NO_LONGER_AVAILABLE | |
| NCT03580083 |
RGX-111 Gene Therapy in Patients With MPS I |
SUSPENDED | PHASE1/PHASE2 |
| NCT03513328 |
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation |
COMPLETED | PHASE1/PHASE2 |
| NCT02171104 |
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT01917708 |
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases |
COMPLETED | PHASE1 |
| NCT01873911 |
Neurobehavioral Phenotypes in MPS III |
COMPLETED | |
| NCT01572636 |
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome |
TERMINATED | |
| NCT01173016 |
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler |
COMPLETED | PHASE1 |
| NCT01043640 |
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders |
COMPLETED | PHASE2 |
| NCT00638547 |
Intrathecal Enzyme Replacement for Hurler Syndrome |
COMPLETED | PHASE1 |
