Treatment of Hunter syndrome and cognitive impairment MedDRA version: 20.1 Level: PT Classification code 10056889 Term: Mucopolysaccharidosis II System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Idursulfase(12S)-IT
Product Code: 12S-IT
Pharmaceutical Form: Solution for injection
INN or Proposed INN: idursulfase
CAS Number: 50936-59-9
Current Sponsor code: 12S-IT
Other descriptiv
Sponsors
Shire HGT Inc
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1 Patients must have completed all study requirements and EOS asessments for Study HGT-HIT-045 prior to enrolling in Study HGT-HIT-046 and must have no safety or medical issues that contraindicate participation. 2 The patient’s parent(s) or legally authorized representative(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 representative(s) and the patient’s assent, as relevant, must be obtained. 3 The 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. Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 1 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1 The patient 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. 2 The 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. 3 The patient has experienced an adverse reaction to study drug in Study HGT-HIT-045 that contraindicates further treatment with intrathecal idursulfase-IT. 4 The patient has a known hypersensitivity to any of the components of idursulfase-IT. 5. The patient 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. 6. The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use, including: a. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device b. The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator c. The patient’s drug therapy requires substances known to be incompatible with the materials of construction d. The patient has a known or suspected local or general infection e. The patient is at risk of abnormal bleeding due to a medical condition or therapy f. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation g. The patient has a functioning CSF shunt device h. The patient 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: 1. The patient has an opening CSF pressure upon lumbar puncture that exceeds 30.0 cm H2O.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To collect long-term safety data in pediatric patients with Hunter syndrome and cognitive impairment who are receiving intrathecal idursulfase-IT and intravenous (IV) Elaprase® enzyme replacement therapy ;Secondary Objective: - To determine the serum pharmacokinetic (PK) profile of idursulfase when administered as intrathecal idursulfase-IT and in conjunction with Elaprase - To determine the effect of intrathecal idursulfase-IT, given in conjunction with Elaprase, on CSF biomarkers (eg, glycosaminoglycan [GAG], including heparan sulfate [HS]/dermatan sulfate [DS] - To determine the effects of intrathecal idursulfase-IT, given in conjunction with Elaprase, on urinary GAGs ;Primary end point(s): Safety of intrathecal idursulfase-IT administration. Safety will be measured by AEs (by type and severity), changes in clinical laboratory testing (serum chemistry including liver function tests, hematology, urinalysis), 12-lead ECG, CSF chemistries (contingent on sample availability; cell counts, glucose, and protein), and anti-idursulfase antibodies and antibodies having enzyme neutralizing activity in CSF and serum. ;Timepoint(s) of evaluation of this end point: Pre-Treatment timepoints at monthly visits as defined in the protocol. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Serum idursulfase concentration-time profiles and serum PK parameters of idursulfase, administered as intrathecal idursulfase-IT and in conjunction with Elaprase - Change from baseline in CSF biomarkers (eg, GAG [HS/DS] - Change from baseline in urinary GAGs ;Timepoint(s) of evaluation of this end point: PK sampling in blood - several timepoints up to 36 hours following IT injection at week 3 and 23 (initial treatment phase) and at months 19, 31 and 43 (extended treatment phase). | — |
Countries
Canada, United Kingdom, United States
Contacts
Public ContactAlkisti Rouvas
Shire HGT
Outcome results
None listed