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A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

NCT05494593 · Status: WITHDRAWN · Phase: PHASE4 · Type: INTERVENTIONAL

Last updated 2025-12-09

No results posted yet for this study

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).

Conditions

Interventions

DRUG

Intravenous Immunoglobulin (IVIG)

Participants will receive 500 mg/kg of IVIG every 4 weeks in 5-week cycle.

DRUG

Rituximab

Participants will receive 375 milligram per square meter per dose (mg/m\^2/dose) of intravenous rituximab weekly for 4 weeks in 5-week cycle.

DRUG

Methotrexate

Participants will receive 0.4 mg/kg of methotrexate by mouth (PO) 3 times per week for 5 weeks in each cycle.

DRUG

ELAPRASE

Participants will receive 0.5 milligram per kilogram (mg/kg) of body weight of ELAPRASE, intravenous, infusion for 104 weeks.

Sponsors & Collaborators

  • Takeda Development Center Americas, Inc.

    collaborator INDUSTRY

  • Takeda

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
6 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-28
Primary Completion
2025-08-29
Completion
2025-08-29
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases
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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05494593 on ClinicalTrials.gov