Extension of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase (IT)in Conjunction With Elaprase in Pediatric Patients With Hunter Syndrome and Cognitive Impairment
This trial is active, not recruiting.
|Phase||phase 1/phase 2|
|Start date||August 2010|
|End date||July 2021|
|Trial size||20 participants|
|Trial identifier||NCT01506141, 2011-000212-25, HGT-HIT-046|
Elaprase, a large molecular protein, is not expected to cross the blood brain barrier when administered intravenously. A revised formulation of idursulfase, idursulfase-IT, that differs from that of the intravenous (IV) formulation, Elaprase, has been developed to be suitable for delivery into the cerebrospinal fluid (CSF) via intrathecal administration.
This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric patients with Hunter syndrome and cognitive impairment who are receiving intrathecal idursulfase-IT and intravenous Elaprase enzyme replacement therapy.
|United States||No locations recruiting|
|Other countries||No locations recruiting|
|Chicago, IL||Ann & Robert H Lurie Childrens Hospital of Chicago||no longer recruiting|
|Chapel Hill, NC||University of North Carolina at Chapel Hill||no longer recruiting|
|Portland, OR||Legacy Emanuel Hospital||no longer recruiting|
|Pittsburgh, PA||Children's Hospital of Pittsburgh of UPMC||no longer recruiting|
|Nashville, TN||Vanderbilt Children's Hospital||no longer recruiting|
|Salt Lake City, UT||University of Utah Hospital||no longer recruiting|
|Seattle, WA||Seattle Children's Hospital||no longer recruiting|
|Vancouver, Canada||British Columbia Children's Hospital||no longer recruiting|
|Birmingham, United Kingdom||Birmingham Children's Hospital||no longer recruiting|
|Intervention model||single group assignment|
Safety of intrathecal idursulfase-IT administration
time frame: 30 Months
Pharmacokinetic (PK) parameters of idursulfase-IT administered in conjunction with Elaprase in CSF and blood.
time frame: PK in blood at time 0, 1, 2, 3, 4, 6, 8, 12, 24 30 and 36 hours every 12 months upon initiation of study, CSF at time immediately prior to each monthly dose out to 30 months.
Change from baseline in CSF biomarkers.
time frame: 30 months
Change from baseline in urinary GAGs and GAG-degradation products
time frame: 30 months
Male participants from 3 years up to 18 years old.
- Patient must have completed all study requirements and end of study assessments for study HGT-HIT-045 prior to enrolling in Study HGT-HIT-046 and must have no safety or medical issues that contraindicate participation.
- The patient'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 patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained.
- That patient 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.
- Patient has received treatment with any investigational drug (other than idursulfase-IT) or device within 30 days prior to study entry.
- Patient 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.
- Patient has experienced an adverse reaction to study drug in Study HGT-HIT-045 that contraindicates further treatment with intrathecal idursulfase-IT.
- Patient has a known hypersensitivity to any of the components of idursulfase-IT.
- For patients who were previously untreated with intrathecal idursulfase-IT in Study HGT-HIT-045, the patient has an opening CSF pressure upon lumbar puncture that exceeds 30.0 cm H2O
|Official title||An Open Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive Impairment|
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