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

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

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

NCT02412787•Shire

View on ClinicalTrials.gov

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

Age

0 - 18 years

Sex

MALE

Healthy Volunteers

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.
+4 more criteria

Locations (9)

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

Key Dates

Start Date

April 14, 2015

Primary Completion Date

April 18, 2024

Completion Date

April 18, 2024

Last Updated

June 19, 2025

Enrollment

ACTUAL participants

Conditions

Hunter Syndrome

Interventions

Idursulfase-IT

DRUG

Elaprase

DRUG

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NCT05422482

Mucopolysaccharidosis IIHunter Syndrome

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AVAILABLE

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

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Hunter Syndrome

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 19, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

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