Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
NCT ID: NCT02412787
Last Updated: 2025-06-19
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2/PHASE3
56 participants
INTERVENTIONAL
2015-04-14
2024-04-18
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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.
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).
Elaprase
Participants received intravenous (IV) Elaprase infusions at a minimum of 48 hours after IT administration of idursulfase-IT.
Interventions
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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).
Elaprase
Participants received intravenous (IV) Elaprase infusions at a minimum of 48 hours after IT administration of idursulfase-IT.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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.
18 Years
MALE
No
Sponsors
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Shire
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Takeda
Locations
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Children's Hospital and Research Center at Oakland
Oakland, California, United States
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Women's and Children's Hospital
Adelaide, , Australia
The Hospital for Sick Children
Toronto, Ontario, Canada
Hôpital Femme Mère Enfants
Bron, , France
Instituto Nacional de Pediatría
Coyoacán, Mexico City, Mexico
Hospital Infantil Universitario Niño Jesus
Madrid, , Spain
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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References
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Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Orphanet J Rare Dis. 2023 Nov 2;18(1):343. doi: 10.1186/s13023-023-02957-2.
Muenzer J, Burton BK, Harmatz P, Gutierrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D; SHP609-302 study group. Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II. Mol Genet Metab. 2022 Sep-Oct;137(1-2):92-103. doi: 10.1016/j.ymgme.2022.07.016. Epub 2022 Aug 2.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain more information on the study, click here/on this link
Other Identifiers
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2014-004143-13
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
SHP609-302
Identifier Type: -
Identifier Source: org_study_id
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