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

Launched by TAKEDA · Aug 8, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a treatment called ELAPRASE for boys with Hunter Syndrome, a condition caused by a deficiency in an enzyme needed to break down certain sugars in the body. The main goal of the study is to see if a special medication plan can help prevent the body from developing antibodies against ELAPRASE, which could make the treatment less effective. Participants in the study will receive ELAPRASE along with a series of other medications over a period of up to 104 weeks, starting one day before the ELAPRASE treatment begins.

To be eligible for this trial, boys must be under 6 years old, have been diagnosed with Hunter Syndrome, and not have received other treatments for this condition before. They should also have a negative test for antibodies against ELAPRASE at the start of the study. During the trial, participants will be monitored regularly to check how well the treatment is working and whether they need additional cycles of the preventive medication plan. Overall, participants and their families can expect close attention and support throughout the study, which will last about 112 weeks in total.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Participant is male.
• • Participant is ELAPRASE-naïve at study entry.
• * Participant must have a documented diagnosis of MPS II. The following combination will be accepted as diagnostic of MPS II:
• • 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).
• • 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.
• • Participant will be less than (\<) 6 years of age at enrollment.
• • Participant has a negative test result for serum anti-idursulfase antibodies.
• Exclusion Criteria:
• • Participant has received treatment with any investigational drug within the 30 days prior to study entry.
• • Participant has received or is receiving treatment with idursulfase-IT.
• • Participant has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time.
• • Participant has received blood product transfusions within 90 days prior to screening.
• • Participant is unable to comply with the protocol as determined by the investigator.
• • 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.
• • Participant has current or recurrent disease that could affect the action, absorption, or disposition of the investigational product, or clinical or laboratory assessments.
• • 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.
• • 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).
• • 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.