IRB Study Number 23-703
Status Recruiting
Institutes Taussig Cancer Institute, Pediatric Institute
Description
3.1.1 Primary Objective
The primary objective of this study is to evaluate the ability of a prophylactic ITR to prevent the development of high titers of anti-idursulfase binding antibodies and NAbs in treatment naïve patients.
3.1.2 Secondary Objectives
The secondary objective of this study is to evaluate the relationship between the development of immune tolerance and iduronate-2-sulfatase (IDS) gene mutations and clinical outcomes
• To assess uGAG levels normalized to urine creatinine and normalized uGAG levels divided by the upper limit of normal for age (uGAG/ULN)
• To assess liver volume.
Inclusion Criteria
The patient is male.
The patient is ELAPRASE-naïve at study entry.
The patient must have a documented diagnosis of MPS II. The following combination will be accepted as diagnostic of MPS II:
a. The patient has a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory’s normal range). The patient 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).
b. The patient has a documented mutation in the IDS gene; additionally, patients must have a severe mutation (eg, large deletion or complex gene rearrangement), which is predicted to lead to development of a persistent anti-idursulfase antibody response.
The patient will be <6 years of age at enrollment.
The patient has a negative test result for serum anti-idursulfase antibodies.
The patient’s parent(s) or legally authorized representative(s) must have voluntarily signed an Institutional Review Board (IRB)-/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient’s parent(s) or legally authorized representative(s) and the patient’s assent, as relevant, must be obtained.
The patient’s parent(s) or legally authorized representative(s) can understand and has the ability and willingness to fully comply with study procedures and requirements.
The patient’s parent(s) or legally authorized representative(s) has the ability to voluntarily provide written, signed, and dated informed consent/assent as applicable to participate in the study.
Exclusion Criteria
The patient has received treatment with any investigational drug within the 30 days prior to study entry.
The patient has received or is receiving treatment with idursulfase-IT.
The patient has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time.
The patient has received blood product transfusions within 90 days prior to screening.
The patient is unable to comply with the protocol as determined by the investigator.
The patient 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.
The patient has current or recurrent disease that could affect the action, absorption, or disposition of the investigational product, or clinical or laboratory assessments.
The patient has current or relevant history of physical or psychiatric illness, or any medical disorder that may require treatment or make the patient unlikely to fully complete the study, or any condition that presents undue risk from the investigational product or procedures.
The patient 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.) See Section 6.6 (Prior and Concomitant Therapy) for a list of prohibited and restricted medications.
Within 30 days prior to the first dose of investigational product, the patient has been enrolled in a clinical study (including vaccine studies) that, in the investigator’s opinion, may impact this Takeda-sponsored study.
