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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02412787




Registration number
NCT02412787
Ethics application status
Date submitted
1/04/2015
Date registered
9/04/2015
Date last updated
19/06/2025

Titles & IDs
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
Secondary ID [1] 0 0
2014-004143-13
Secondary ID [2] 0 0
SHP609-302
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hunter Syndrome 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Idursulfase-IT
Treatment: Drugs - Elaprase

Experimental: Idursulfase-IT - Participants received 10 milligrams (mg) of idursulfase-IT intrathecally via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once every 28 days along with standard-of-care therapy with Elaprase, for 470 weeks (for participants who began intrathecal \[IT\] treatment in this study) and 480 weeks (for participants who began IT treatment in HGT-HIT-094 and continued to receive IT treatment in this study). Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (\>8 months to 30 months of age) or 10 mg (\>30 months to 3years of age) of idursulfase-IT.


Treatment: Drugs: Idursulfase-IT
Participants received 10 mg of idursulfase-IT intrathecally via IDDD or LP once every 28 days. Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (\>8 months to 30 months of age) and 10 mg (\>30 months to 3 years of age).

Treatment: Drugs: Elaprase
Participants received intravenous (IV) Elaprase infusions at a minimum of 48 hours after IT administration of idursulfase-IT.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events (AEs)
Timepoint [1] 0 0
Up to 9 years
Primary outcome [2] 0 0
Number of Participants With Clinically Significant Changes in Vital Signs
Timepoint [2] 0 0
Up to 9 years
Primary outcome [3] 0 0
Number of Participants With Clinically Significant Changes in Laboratory Parameters
Timepoint [3] 0 0
Up to 9 years
Primary outcome [4] 0 0
Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings
Timepoint [4] 0 0
Up to 9 years
Primary outcome [5] 0 0
Maximum Observed Serum Concentration (Cmax) of Idursulfase
Timepoint [5] 0 0
Predose and at 30 min, 60 min, 120 min, 4 hours, 6 hours, 8 hours, 12 hours, 24 hours, 30 hours and 36 hours post-dose at Week 100 in relation to HGT-HIT-094 (Week 48 of this study)
Primary outcome [6] 0 0
Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67
Timepoint [6] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Primary outcome [7] 0 0
Percent Change From Baseline in the Concentration of GAG in Urine at Month 67
Timepoint [7] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Primary outcome [8] 0 0
Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF
Timepoint [8] 0 0
Up to 9 years
Primary outcome [9] 0 0
Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum
Timepoint [9] 0 0
Up to 9 years
Secondary outcome [1] 0 0
Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Standard Cluster Scores at Month 67
Timepoint [1] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Secondary outcome [2] 0 0
Change From Baseline in Standard Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Domains at Month 67
Timepoint [2] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Secondary outcome [3] 0 0
Change From Baseline in Standard Composite Scores of the VABS-II Domains at Month 67
Timepoint [3] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Secondary outcome [4] 0 0
Change From Baseline in Age Equivalents Score of the Differential Ability Scales, Second Edition (DAS-II) at Month 61
Timepoint [4] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study.
Secondary outcome [5] 0 0
Change From Baseline in Developmental Quotients (DQ) of the DAS-II at Month 61
Timepoint [5] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study.
Secondary outcome [6] 0 0
Change From Baseline in T-scores of the Core Subtests DAS-II at Month 61
Timepoint [6] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study.
Secondary outcome [7] 0 0
Change From Baseline in Age Equivalents Score of the VABS-II Sub Domains at Month 67
Timepoint [7] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Secondary outcome [8] 0 0
Change From Baseline in DQ of the VABS-II Sub Domains at Month 67
Timepoint [8] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Secondary outcome [9] 0 0
Change From Baseline in v-Scores of the VABS-II Sub Domains at Month 67
Timepoint [9] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Secondary outcome [10] 0 0
Change From Baseline in v-Scale Scores of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 67
Timepoint [10] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study.
Secondary outcome [11] 0 0
Number of Participants With Observed Maladaptive Levels of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 61
Timepoint [11] 0 0
For participants who began IT treatment in HGT-HIT-094: At Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: At Month 49 of this study.
Secondary outcome [12] 0 0
Change From Baseline in Brain Structure Volume as Measured by Magnetic Resonance Imaging (MRI)
Timepoint [12] 0 0
For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 120 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 108 of this study.

Eligibility
Key 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).
Minimum age
No limit
Maximum age
18 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women's and Children's Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
5006 - Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
North Carolina
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario
Country [5] 0 0
France
State/province [5] 0 0
Bron
Country [6] 0 0
Mexico
State/province [6] 0 0
Ciudad De México
Country [7] 0 0
Spain
State/province [7] 0 0
Madrid
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Shire
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Takeda
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.