Phase 3
N=56
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
Hunter Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02412787 ↗Enrolled (actual)
56
Serious AEs
85.7%
Results posted
Jun 2025
Primary outcome: Primary: Number of Participants With Adverse Events (AEs) — 56 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Idursulfase-IT (Drug); Elaprase (Drug)
- Age
- Pediatric, Adult
- Sex
- Male
- Sponsor
- Shire
- Primary completion
- Apr 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs) |
56 | — |
| PRIMARY Number of Participants With Clinically Significant Changes in Vital Signs |
— | — |
| PRIMARY Number of Participants With Clinically Significant Changes in Laboratory Parameters |
— | — |
| PRIMARY Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings |
— | — |
| PRIMARY Maximum Observed Serum Concentration (Cmax) of Idursulfase |
128.19; 104.89; 120.23 | — |
| PRIMARY Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67 |
-71.15 | — |
| PRIMARY Percent Change From Baseline in the Concentration of GAG in Urine at Month 67 |
-32.44 | — |
| PRIMARY Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF |
25 | — |
| PRIMARY Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum |
46 | — |
| SECONDARY Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Standard Cluster Scores at Month 67 |
-6.0; -23.7; -20.5; 0.3; -5.5 | — |
| SECONDARY Change From Baseline in Standard Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Domains at Month 67 |
-13.5; -9.5; -14.5; 11.5; -13.5 | — |
| SECONDARY Change From Baseline in Standard Composite Scores of the VABS-II Domains at Month 67 |
-13.5 | — |
| SECONDARY Change From Baseline in Age Equivalents Score of the Differential Ability Scales, Second Edition (DAS-II) at Month 61 |
1.50; 2.63; 3.13; 1.38; 3.38; 1.13 | — |
| SECONDARY Change From Baseline in Developmental Quotients (DQ) of the DAS-II at Month 61 |
-27.45; -7.75; -10.95; -22.25; -9.20; -34.95 | — |
| SECONDARY Change From Baseline in T-scores of the Core Subtests DAS-II at Month 61 |
-14.5; -6.5; -8.5; -10.0; 2.5; -14.0 | — |
| SECONDARY Change From Baseline in Age Equivalents Score of the VABS-II Sub Domains at Month 67 |
2.13; 2.54; 1.58; 4.50; 3.42; 1.83 | — |
| SECONDARY Change From Baseline in DQ of the VABS-II Sub Domains at Month 67 |
-7.30; -6.65; -40.15; 9.50; -22.60; -27.15 | — |
| SECONDARY Change From Baseline in v-Scores of the VABS-II Sub Domains at Month 67 |
-0.5; -1.0; -8.0; 1.0; -2.0; -5.0 | — |
| SECONDARY Change From Baseline in v-Scale Scores of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 67 |
-3.0; -4.0; -1.0 | — |
| SECONDARY Number of Participants With Observed Maladaptive Levels of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 61 |
7; 1; 6; 2; 2; 6 | — |
| SECONDARY Change From Baseline in Brain Structure Volume as Measured by Magnetic Resonance Imaging (MRI) |
-19.813; -38.573; 98.180; -136.750; 58.480 | — |
Summary
This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not receive idursulfase IT treatment in Study HGT-HIT-094.
Eligibility Criteria
Inclusion Criteria
- Participants must have completed Visit Week 52 assessments in Study HGT-HIT-094 (NCT02055118).
- The participant's parent(s) or legally authorized guardian(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 participant's parent(s) or legally authorized guardian(s) and the participant's consent/assent, as relevant, must be obtained.
- The participant has continued to receive Elaprase on a regular basis in Study HGT-HIT-094 (NCT02055118).
Exclusion Criteria
- The participant has experienced, in the opinion of the investigator, a safety or medical issue that contraindicates treatment with idursulfase-IT, including, but not limited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevant hypertension.
- The participant has a known hypersensitivity to any of the components of idursulfase-IT.
- The participant has clinically relevant intracranial hypertension.
- The participant is enrolled in another clinical study, other than HGT-HIT-094 (NCT02055118), that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study.
- The participant has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to compromised airways or other conditions.
- The participant has a condition that is contraindicated as described in the SOPH-A-PORT® Mini S, Implantable Access Port, Spinal, Mini Unattached, with Guidewire (SOPH-A-PORT Mini S) intrathecal drug delivery device (IDDD) Instructions for Use, including:
- The participant has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device.
- The participant's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the investigator.
- The participant's drug therapy requires substances known to be incompatible with the materials of construction.
- The participant has a known or suspected local or general infection.
- The participant is at risk of abnormal bleeding due to a medical condition or therapy.
- The participant has 1 or more spinal abnormalities that could complicate safe implantation or fixation.
- The participant has a functioning CSF shunt device.
- The participant has shown an intolerance to an implanted device.
Data sourced from ClinicalTrials.gov (NCT02412787). 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.