Phase 3
Completed N=94
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Hunter Syndrome · Mucopolysaccharidosis II (MPS II)
Source: ClinicalTrials.gov NCT00630747 ↗
Enrolled (actual)
94
Serious AEs
40.4%
Results posted
Mar 2014
Primary outcomePrimary: Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105 — -0.056 percent predicted FVC
Summary
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study. The primary objective of this extension study was to collect long-term safety and clinical outcome data in Mucopolysaccharidosis II (MPS II), also known as Hunter Syndrome, from the Phase 2/Phase 3 Study TKT024. All patients enrolling into this study received weekly active treatment with idursulfase, the primary dosing regimen investigated in Study TKT024.
Hunter Syndrome is an X-linked recessive lysosomal storage disease caused by a deficiency of iduronate-2-sulfatase, an enzyme required to catabolize glycosaminoglycans (GAGS) in cells. As a result, GAGs accumulate in the lysosomes leading to cellular engorgement, organomegaly, tissue destruction, and organ system dysfunction. Hunter Syndrome is a rare disease with an estimated incidence of 1 in 162,000 live births.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105 |
-0.056 | — |
| PRIMARY Change From Baseline in Mean Distance Walked in the 6-minute Walk Test (6MWT) at Week 105 |
23.0 | — |
| SECONDARY Change From Baseline in Mean Passive Joint Range of Motion (JROM) at Week 105 |
0.63 | — |
| SECONDARY Change From Baseline in Mean Combined Liver and Spleen Volume at Week 105 |
-325.5 | — |
| SECONDARY Change From Baseline in Mean Normalized Urine Glycosaminoglycans (GAG) Levels at Week 105 |
-238.25 | — |
| SECONDARY Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 105 |
3.28 | — |
Eligibility Criteria
Inclusion Criteria
- Patient must have completed the double-blind phase of Study TKT024, defined as completing the Week 53 final evaluations.
- Patient, patient's parent(s), or legally authorized representative 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 with the patient.
Exclusion Criteria
- Patient has received treatment with an investigational therapy other than iduronate-2-sulfatase in Study TKT024 within the past 60 days.
- Patient is unable to comply with the protocol (e.g., due to a medical condition such as cervical cord compression or uncooperative attitude) or is unlikely to complete the study, as determined by the investigator.
- Patient has experienced an adverse reaction to study drug in Study TKT024, which contraindicates further treatment with idursulfase.
- Patient with known hypersensitivity to any of the components of idursulfase.
Data sourced from ClinicalTrials.gov (NCT00630747). 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.