An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

Completed · Phase 1/Phase 2

Conditions studied: Hunter Syndrome

In brief

This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV) Elaprase enzyme replacement therapy.

Key facts

Study ID

NCT01506141

Run by

Takeda

People needed

15

Starts

2010-08-01

Expected to finish

2024-04-30

Last updated by the study team

2025-08-06

Who can join

Age: 3 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Participant must have completed all study requirements and End of study (EOS) assessments for study HGT-HIT-045 (NCT00920647) prior to enrolling in Study HGT-HIT-046 and must have no safety or medical issues that contraindicate participation.
• 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 assent, as relevant, must be obtained.
• The participant has received and tolerated a minimum of 12 months of treatment with weekly IV infusions of Elaprase and has received 80% of the total planned infusions within the last 6 months.

You may not qualify if…

• The participant is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device) other than the PORT-A-CATH IDDD within 30 days prior to study enrollment or at any time during the study.
• The participant is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the investigator.
• The participant has experienced an adverse reaction to study drug in Study HGT-HIT-045 (NCT00920647) that contraindicates further treatment with intrathecal idursulfase-IT.
• The participant has a known hypersensitivity to any of the components of idursulfase-IT.
• The participant has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions.
• The participant has a condition that is contraindicated as described in the SOPH-A-PORT Mini S 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 one 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
• An additional exclusion criterion for patients who were previously untreated with intrathecal idursulfase-IT in Study HGT-HIT-045 (NCT00920647):
• The participant has an opening CSF pressure upon lumbar puncture that exceeds 30.0 centimeter (cm) water (H2O).

Where it is running

• Ann & Robert H Lurie Childrens Hospital of Chicago — Chicago, Illinois, United States
• University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
• Legacy Emanuel Hospital — Portland, Oregon, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Vanderbilt Children's Hospital — Nashville, Tennessee, United States
• University of Utah Hospital — Salt Lake City, Utah, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• British Columbia Children's Hospital — Vancouver, British Columbia, Canada
• Birmingham Children's Hospital — Birmingham, United Kingdom

Full record on ClinicalTrials.gov

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