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



Status:Active, not recruiting
Conditions:Cognitive Studies, Metabolic
Therapuetic Areas:Pharmacology / Toxicology, Psychiatry / Psychology
Healthy:No
Age Range:3 - 18
Updated:2/8/2019
Start Date:August 1, 2010
End Date:July 1, 2021

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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

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.


Inclusion Criteria:

- Participant 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 participant'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 participant's parent(s) or legally authorized guardian(s)
and the participant's assent, as relevant, must be obtained.

- The participant 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:

- The participant 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.

- The participant 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.

- The participant has experienced an adverse reaction to study drug in Study HGT-HIT-045
that contraindicates further treatment with intrathecal idursulfase-IT.

- The participant has a known hypersensitivity to any of the components of
idursulfase-IT.

- The participant 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.

- The participant has a condition that is contraindicated as described in the
SOPH-A-PORT Mini S IDDD Instructions for Use, including:

1. The participant has had, or may have, an allergic reaction to the materials of
construction of the SOPH-A-PORT Mini S device

2. 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

3. The participant's drug therapy requires substances known to be incompatible with
the materials of construction

4. The participant has a known or suspected local or general infection

5. The participant is at risk of abnormal bleeding due to a medical condition or
therapy

6. The participant has one or more spinal abnormalities that could complicate safe
implantation or fixation

7. The participant has a functioning CSF shunt device

8. The participant has shown an intolerance to an implanted device

- The participant has an opening CSF pressure upon lumbar puncture that exceeds 30.0
centimeter (cm) water (H2O).
We found this trial at
8
sites
Portland, Oregon 97227
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Chapel Hill, North Carolina 27599
(919) 962-2211
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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Chapel Hill, NC
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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Pittsburgh, PA
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Chicago, IL
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2200 Children's Way
Nashville, Tennessee 37232
(615) 936-1000
Vanderbilt Children's Hospital Monroe Carell Jr. Children's Hospital at Vanderbilt is one of the nation's...
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Nashville, TN
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Salt Lake City, Utah 84132
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Salt Lake City, UT
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Seattle, WA
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Vancouver, British Columbia
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Vancouver,
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