WITHDRAWN

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

An Open-label, Multicenter, Phase 4 Study to Assess the Effects of a Prophylactic Immune Tolerizing Regimen in MPS II Treatment-Naïve Patients Planned to Receive ELAPRASE Who Are at Risk of Developing Persistent Neutralizing Antibodies

Quick Facts

Study Start:2023-02-28
Study Completion:2025-08-29
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:WITHDRAWN

Study ID

NCT05494593

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 6 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Participant is male.
  2. * Participant is ELAPRASE-naïve at study entry.
  3. * Participant must have a documented diagnosis of MPS II. The following combination will be accepted as diagnostic of MPS II:
  4. * Participant has a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of less than or equal to (\<=) 10 percent (%) of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory's normal range). The participant has a normal enzyme activity level of at least 1 other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory's normal range).
  5. * Participant has a documented mutation in the IDS gene; additionally, participants must have a severe mutation (example, large deletion or complex gene rearrangement), which is predicted to lead to development of a persistent anti-idursulfase antibody response.
  6. * Participant will be less than (\<) 6 years of age at enrollment.
  7. * Participant has a negative test result for serum anti-idursulfase antibodies.
  1. * Participant has received treatment with any investigational drug within the 30 days prior to study entry.
  2. * Participant has received or is receiving treatment with idursulfase-IT.
  3. * Participant has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time.
  4. * Participant has received blood product transfusions within 90 days prior to screening.
  5. * Participant is unable to comply with the protocol as determined by the investigator.
  6. * Participant has known or suspected intolerance or hypersensitivity to the investigational product(s), closely related compounds, or any of the stated ingredients, including the prophylactic ITR.
  7. * Participant has current or recurrent disease that could affect the action, absorption, or disposition of the investigational product, or clinical or laboratory assessments.
  8. * Participant has current or relevant history of physical or psychiatric illness, or any medical disorder that may require treatment or make the participant unlikely to fully complete the study, or any condition that presents undue risk from the investigational product or procedures.
  9. * Participant has current use of any medication (including over-the-counter, herbal, or homeopathic preparations) that could affect (improve or worsen) the condition being studied, or could affect the action, absorption, or disposition of the investigational product(s), or clinical or laboratory assessment (Current use is defined as use within 30 days).
  10. * Within 30 days prior to the first dose of investigational product, the participant has been enrolled in a clinical study (including vaccine studies) that, in the investigator's opinion, may impact this study.

Contacts and Locations

Principal Investigator

Study Director
STUDY_DIRECTOR
Takeda

Study Locations (Sites)

Phoenix Childrens Hospital
Phoenix, Arizona, 85016
United States
Children's Hospital and Research Center at Oakland
Oakland, California, 94609
United States
Rady Childrens Hospital San Diego - PIN
San Diego, California, 92123
United States
The Lundquist Institute for BioMedical Innovation at Harbor-UCLA Medical Center
Torrance, California, 90502
United States
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611
United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
United States
NewYork-Presbyterian Morgan Stanley Children's Hospital
New York, New York, 10032
United States
The Cleveland Clinic Foundation
Twinsburg, Ohio, 44087
United States

Collaborators and Investigators

Sponsor: Takeda

  • Study Director, STUDY_DIRECTOR, Takeda

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-02-28
Study Completion Date2025-08-29

Study Record Updates

Study Start Date2023-02-28
Study Completion Date2025-08-29

Terms related to this study

Additional Relevant MeSH Terms

  • Mucopolysaccharidosis (MPS)
  • Hunter Syndrome