A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
Status:
Not yet recruiting
Trial end date:
2026-09-09
Target enrollment:
Participant gender:
Summary
The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing
regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies
in treatment-naïve participants with Hunter syndrome.
In this open label, single arm study, all participants will receive ELAPRASE treatment and a
prophylactic ITR.
Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will
start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week
cycle of: Rituximab (intravenously [IV], weekly for 4 weeks); Methotrexate (oral, 3 times per
week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle).
Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18
regarding the need for administering another 5-week cycle of the ITR.
Participants will be in the study for approximately 112 weeks (including 6 weeks for
screening, up to 104 weeks for treatment, and 2 weeks for follow-up).