Phase 4
Completed N=28
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
Hunter Syndrome · Mucopolysaccharidosis II · MPS II
Source: ClinicalTrials.gov NCT00607386 ↗
Enrolled (actual)
28
Serious AEs
46.4%
Results posted
Nov 2013
Primary outcomePrimary: Safety Evaluation — 28; 0; 0; 16 participants
Summary
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety Evaluation |
28; 0; 0; 16; 13; 2 | — |
| SECONDARY Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels |
738.3; -368.0; -400.3; -402.4 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) |
1333; 1032 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax) |
163; 167 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to the Final Time Point With a Concentration of at Least Lower Limit of Quantitation (AUClast) |
196526; 174869 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf) |
224343; 201130 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2) |
160; 109 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf) |
153; 127 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Clearance (CL) |
2.4; 4.7 | — |
| SECONDARY Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss) |
394; 551 | — |
Eligibility Criteria
Inclusion Criteria
- The patient has a diagnosis of Hunter syndrome based upon biochemical criteria either documented in their medical history or established at Screening:
- 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 normal range of measuring laboratory)
AND
- A normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory).
- The patient is 5 years of age and under.
- The patient is male.
- The patient's parent(s), or patient's legal guardian must have voluntarily signed an Institutional Review Board approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient's parent(s), or the patient's legal guardian.
Exclusion Criteria
- The patient has received treatment with another investigational therapy within 30 days prior to enrollment.
- The patient has clinically relevant medical condition(s) making implementation of the protocol difficult.
- The patient has previously received idursulfase.
- The patient has known hypersensitivity to any of the components of idursulfase.
- The patient has had a tracheostomy.
Data sourced from ClinicalTrials.gov (NCT00607386). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.