Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

• Register: ClinicalTrials.gov
• Last refreshed on: 15 April 2024
• Main ID: NCT02412787
• Date of registration: 01/04/2015
• Prospective Registration: Yes
• Primary sponsor: Shire
• Public title: Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
• Scientific title: An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment
• Date of first enrolment: October 28, 2015
• Target sample size: 56
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02412787
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Argentina, Australia, Canada, Colombia, France, Mexico, Spain, United Kingdom, United States

Contacts

• Name: Study Director
• Affiliation: Takeda

Key inclusion & exclusion 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:

1. The participant has had, or may have, an allergic reaction to the materials of
construction of the SOPH-A-PORT Mini S device.

2. 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.

3. The participant's drug therapy requires substances known to be incompatible with
the materials of construction.

4. The participant has a known or suspected local or general infection.

5. The participant is at risk of abnormal bleeding due to a medical condition or
therapy.

6. The participant has 1 or more spinal abnormalities that could complicate safe
implantation or fixation.

7. The participant has a functioning CSF shunt device.

8. The participant has shown an intolerance to an implanted device.



Age minimum: N/A
Age maximum: 18 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Hunter Syndrome

Intervention(s)

Drug: Elaprase

Drug: Idursulfase-IT

Primary Outcome(s)

Number of Participants With Adverse Events [Time Frame: From start of study drug administration up to 121 months]

Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in Cerebrospinal Fluid (CSF) [Time Frame: Baseline through Month 121]

Maximum Observed Serum Concentration (Cmax) of Idursulfase [Time Frame: Baseline through Month 121]

Number of Participants With Clinically Significant Changes in Vital Signs, Laboratory Parameters, and 12-lead Electrocardiogram (ECG) Findings [Time Frame: From start of study drug administration up to 121 months]

Number of Participants who Report Anti-idursulfase Antibodies in Cerebrospinal Fluid (CSF) [Time Frame: From start of study drug administration up to 121 months]

Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in Urine [Time Frame: Baseline through Month 121]

Maximum Observed Concentration (Cmax) of Idursulfase in Cerebrospinal Fluid (CSF) [Time Frame: Baseline through Month 121]

Number of Participants who Report Anti-idursulfase Antibodies in Serum [Time Frame: From start of study drug administration up to 121 months]

Secondary Outcome(s)

Change From Baseline in Standard Composite Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Domains [Time Frame: Baseline through Month 121]

Change From Baseline in Age Equivalents Score of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Sub Domains [Time Frame: Baseline through Month 121]

Change From Baseline in Standard Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Domains [Time Frame: Baseline through Month 121]

Change From Baseline in Development Quotient (DQ) of the Bayley Scales of Infant Development, Third Edition (BSID-III) Domains [Time Frame: Baseline through Month 121]

Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Standard Scores and Standard Cluster Scores [Time Frame: Baseline through Month 121]

Change From Baseline in T-scores of the Core Subtests Differential Ability Scales, Second Edition (DAS-II) [Time Frame: Baseline through Month 121]

Change From Baseline in Brain Structure Volume as Measured by Magnetic Resonance Imaging (MRI) [Time Frame: Baseline through Month 121]

Change From Baseline in Developmental Quotients (DQ) of the Differential Ability Scales, Second Edition (DAS-II) [Time Frame: Baseline through Month 121]

Change From Baseline in v-Scale Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Maladaptive Behavior Index and its Subscales [Time Frame: Baseline through Month 121]

Change From Baseline in Age Equivalent Scores of the Bayley Scales of Infant Development, Third Edition (BSID-III) Domains [Time Frame: Baseline through Month 121]

Change From Baseline in Age Equivalents Score of the Differential Ability Scales, Second Edition (DAS-II) [Time Frame: Baseline through Month 121]

Change From Baseline in Developmental Quotients (DQ) of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Sub Domains [Time Frame: Baseline through Month 121]

Change From Baseline in Observed Maladaptive Levels of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Maladaptive Behavior Index and its Subscales [Time Frame: Baseline through Month 121]

Change From Baseline in v-Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Sub Domains [Time Frame: Baseline through Month 121]

Secondary ID(s)

SHP609-302

2014-004143-13

Source(s) of Monetary Support

Please refer to primary and secondary sponsors