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 States15 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 range3 Years – 18 Years

SexMALE

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
✕. The participant has shown an intolerance to an implanted 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

Number of Participants With Anti-idursulfase Antibodies in CSF

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

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 range3 Years – 18 Years

SexMALE

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
✕. The participant has shown an intolerance to an implanted 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

Number of Participants With Anti-idursulfase Antibodies in CSF

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