Overview

This trial is active, not recruiting.

Condition hunter syndrome
Treatment idursulfase-it
Phase phase 1/phase 2
Sponsor Shire
Start date August 2010
End date July 2021
Trial size 20 participants
Trial identifier NCT01506141, 2011-000212-25, HGT-HIT-046

Summary

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

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Idursulfase-IT administered once monthly at the dose used in study HGT-HIT-045 via intrathecal drug delivery device (IDDD)
idursulfase-it
Idursulfase-IT administered once monthly at the dose used in study HGT-HIT-045 via intrathecal drug delivery device (IDDD)

Primary Outcomes

Measure
Safety of intrathecal idursulfase-IT administration
time frame: 30 Months

Secondary Outcomes

Measure
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

Eligibility Criteria

Male participants from 3 years up to 18 years old.

Inclusion Criteria: - 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. Exclusion Criteria: - 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

Additional Information

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
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Shire.