RGX-111 Gene Therapy in Patients With MPS I

  • End date
    Jul 15, 2023
  • participants needed
  • sponsor
    Regenxbio Inc.
Updated on 15 July 2021


RGX-111 is a gene therapy which is intended to deliver a functional copy of the -L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.


Mucopolysaccharidosis type I (MPS I) is a rare recessive genetic disease caused by a deficiency of -L-iduronidase (IDUA) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of patients with MPS I. While currently available therapies, enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), provide clinical benefit over untreated disease progression, they still possess significant limitations. ERT does not cross the blood-brain barrier and, therefore, does not treat the central nervous system (CNS) effects of the disease, and HSCT has clinically relevant morbidity and mortality and is not able to completely treat the CNS effects. RGX-111 is designed to deliver a functioning gene enabling the production of IDUA in the brain. This is a Phase I/II, first-in-human, multicenter, open-label, dose escalation study of RGX-111. Two, one time doses of RGX-111 will be studied in approximately 5 subjects who have MPS I. Safety will be the primary focus for the initial 24 weeks after treatment (primary study period) whereupon, subjects will continue to be assessed (safety and efficacy) for up to a total of 104 weeks following treatment with RGX-111.

Condition Hurler's Syndrome, mucopolysaccharidosis type i
Treatment RGX-111
Clinical Study IdentifierNCT03580083
SponsorRegenxbio Inc.
Last Modified on15 July 2021


Yes No Not Sure

Inclusion Criteria

Has documented evidence of CNS involvement due to MPS I or documented diagnosis of severe MPS I
Subjects who have had HSCT may be enrolled in the study if the PI, medical monitor, and sponsor agree that he/she can safely and successfully participate in the study
Patient or Patient's legal guardian must be willing and able to provide written, signed informed consent

Exclusion Criteria

Has contraindications for intracisternal and intracerebroventricular injection or lumbar puncture
Has contraindications for immunosuppressive therapy
Has neurocognitive deficit not attributable to MPS I or diagnosis of a neuropsychiatric condition
Received intrathecal (IT) laronidase at any time and experienced a significant AE considered related to IT administration
Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 upper limit of normal (ULN) or total bilirubin >1.5 ULN at screening, unless the subject has a previously known history of Gilbert's syndrome and a fractionated bilirubin that shows conjugated bilirubin <35% of total bilirubin
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