Long-Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Patients With Hunter Syndrome That Have Completed Study HGT-HIT-094

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02412787
Recruitment Status : Enrolling by invitation
First Posted : April 9, 2015
Last Update Posted : March 29, 2017
Information provided by (Responsible Party):

Brief Summary:
This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not received idursulfase IT treatment in Study HGT-HIT-094.

Condition or disease Intervention/treatment Phase
Hunter Syndrome Drug: Idursulfase-IT Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 49 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment
Study Start Date : April 2015
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : September 2022

Arm Intervention/treatment
Experimental: Idursulfase-IT
All patients in this study will be treated with intrathecal idursulfase-IT in conjunction with Elaprase therapy. Patients who complete Visit Week 52 assessments of Study HGT HIT 094 and who meet the eligibility criteria and for whom informed consent is provided will be enrolled in this extension study. All patients will receive idursulfase-IT at the same dose and frequency (10 mg once every 28 days) as selected for the HGT-HIT-094 study. Patients who are younger than 3 years of age will continue to receive an adjusted dose.
Drug: Idursulfase-IT
Patients will receive idursulfase-IT treatment once every 28 days at a dose of 10 mg. Idursulfase will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD).
Other Name: HGT-2310

Primary Outcome Measures :
  1. Change in physical and neurological examination to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  2. Change in height and weight [ Time Frame: Baseline to month 60 ]
  3. Change in head circumference [ Time Frame: Baseline to month 60 ]
  4. Change in auditory capacity for hearing assessment [ Time Frame: Baseline to month 60 ]
  5. Number of participants with adverse events as measured by type, severity, and relationship to treatment [ Time Frame: Baseline to month 60 ]
  6. Change in vital signs [ Time Frame: Baseline to month 60 ]
  7. Change in 12-lead ECG recordings [ Time Frame: Baseline to month 60 ]
  8. Change in clinical laboratory testings as measured by serum chemistry, hematology, and urinalysis to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  9. Change in cerebrospinal fluid laboratory standard chemistry to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  10. Change in cerebrospinal fluid laboratory parameters to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  11. Change in anti-idursulfase antibodies in CSF and serum [ Time Frame: Baseline to month 60 ]
  12. Change in urine GAG and creatinine [ Time Frame: Baseline to month 60 ]
  13. Change in serum albumin [ Time Frame: Baseline to month 60 ]

Secondary Outcome Measures :
  1. Change in standard scores in cluster areas of the DAS-II / BAS-II [ Time Frame: Baseline to month 60 ]
  2. Change in standard scores of the VABS-II domains [ Time Frame: Baseline to month 60 ]
  3. Change in T-scores for the subtests of the DAS-II [ Time Frame: Baseline to month 60 ]
  4. Change in V-scale scores of the VABS-II subdomains [ Time Frame: Baseline to month 60 ]
  5. Change in observed maladaptive levels of the VABS-II Maladaptive Behavior Index and its subscales [ Time Frame: Baseline to month 60 ]
  6. Change in brain structure volume as measured by magnetic resonance imagine (MRI) [ Time Frame: Baseline to month 60 ]

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patients must have completed Visit Week 52 assessments in Study HGT-HIT-094
  2. 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
  3. The patient has continued to receive Elaprase on a regular basis in Study HGT-HIT-094

Exclusion Criteria:

  1. The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with idursulfase-IT, including, but not limited to,uncontrolled seizure disorder, bleeding disorder, and clinically relevant hypertension
  2. The patient has a known hypersensitivity to any of the components of idursulfase-IT
  3. The patient has clinically relevant intracranial hypertension
  4. The patient is enrolled in another clinical study, other than HGT-HIT-094, that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study
  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 compromised airways 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:

    1. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
    2. 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
    3. The patient's drug therapy requires substances known to be incompatible with the materials of construction
    4. The patient has a known or suspected local or general infection
    5. The patient is at risk of abnormal bleeding due to a medical condition or therapy
    6. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    7. The patient has a functioning CSF shunt device
    8. The patient has shown an intolerance to an implanted device

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02412787

United States, California
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
United States, Illinois
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, North Carolina
University of North Carolina
Chapel Hill, North Carolina, United States, 27514
Women's and Children's Hospital
Adelaide, Australia, 5006
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Hôpital Femme Mère Enfants
Bron, France, 69677
Instituto Nacional de Pediatría
Coyoacan, Ciudad De México, Mexico, 04530
Hospital Infantil Universitario Niño Jesus
Madrid, Spain, 28009
United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Study Director: David Alexanderian, DO Shire

Responsible Party: Shire Identifier: NCT02412787     History of Changes
Other Study ID Numbers: SHP609-302
2014-004143-13 ( EudraCT Number )
First Posted: April 9, 2015    Key Record Dates
Last Update Posted: March 29, 2017
Last Verified: March 2017

Additional relevant MeSH terms:
Mucopolysaccharidosis II
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases