|
Main
|
|
Note: This record shows only the 20 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
13 May 2013 |
|
Main ID: |
NCT01506141 |
|
Date of registration:
|
15/12/2011 |
|
Primary sponsor: |
|
|
Public title:
|
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
|
|
Scientific 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 |
|
Date of first enrolment:
|
August 2010 |
|
Target sample size:
|
20 |
|
Recruitment status: |
Active, not recruiting |
|
URL:
|
http://clinicaltrials.gov/show/NCT01506141 |
|
Study type:
|
Interventional |
|
Study design:
|
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
|
|
|
Countries of recruitment
|
|
United Kingdom
|
United States
| | | | | | |
|
Contacts
|
|
Name:
|
Joseph Muenzer, MD, PhD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University of North Carolina, Chapel Hill |
| | |
|
Key inclusion & exclusion criteria
|
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
Age minimum:
3 Years
Age maximum:
18 Years
Gender:
Male
|
|
Health Condition(s) or Problem(s) studied
|
|
Hunter Syndrome
|
|
Intervention(s)
|
|
Drug: Idursulfase-IT
|
|
Primary Outcome(s)
|
|
Safety of intrathecal idursulfase-IT administration
[Time Frame: 30 Months]
|
|
Secondary Outcome(s)
|
|
Change from baseline in CSF biomarkers.
[Time Frame: 30 months]
|
|
Change from baseline in urinary GAGs and GAG-degradation products
[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.]
|
|
Secondary ID(s)
|
|
HGT-HIT-046
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|