CompletedPhase 1/2

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

United States, Canada, United Kingdom15 participantsStarted 2010-08-01

Plain-language summary

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.

Who can participate

Age range

3 Years – 18 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. 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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Timeframe: From start of study drug administration up to follow-up (up to 165 months)

Number of Participants With Clinically Significant Changes or Apparent Difference Across Treatment Groups in Laboratory Parameters

Timeframe: From start of study drug administration up to follow-up (up to 165 months)

Number of Participants With Clinically Significant Changes or Apparent Difference Across Treatment Groups in 12-lead Electrocardiogram (ECG) Findings

Timeframe: From start of study drug administration up to follow-up (up to 165 months)

CSF Chemistries: Change From Baseline in CSF Total Cell Count

Timeframe: Baseline, Month 163

CSF Chemistries: Change From Baseline in CSF Glucose

Timeframe: Baseline, Month 163

CSF Chemistries: Change From Baseline in CSF Protein

Timeframe: Baseline, Month 163

Trial details

NCT IDNCT01506141

SponsorTakeda

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-04-30

Results submitted2025-04-29

View on ClinicalTrials.gov More Hunter Syndrome trials

Who can participate

Age range

3 Years – 18 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. 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

What they're measuring

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Timeframe: From start of study drug administration up to follow-up (up to 165 months)

Number of Participants With Clinically Significant Changes or Apparent Difference Across Treatment Groups in Laboratory Parameters

Timeframe: From start of study drug administration up to follow-up (up to 165 months)

Number of Participants With Clinically Significant Changes or Apparent Difference Across Treatment Groups in 12-lead Electrocardiogram (ECG) Findings

Timeframe: From start of study drug administration up to follow-up (up to 165 months)

CSF Chemistries: Change From Baseline in CSF Total Cell Count

Timeframe: Baseline, Month 163

CSF Chemistries: Change From Baseline in CSF Glucose

Timeframe: Baseline, Month 163

CSF Chemistries: Change From Baseline in CSF Protein

Timeframe: Baseline, Month 163