Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)
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|ClinicalTrials.gov Identifier: NCT02055118|
Recruitment Status : Completed
First Posted : February 4, 2014
Last Update Posted : November 8, 2017
|Condition or disease||Intervention/treatment||Phase|
|Hunter Syndrome||Biological: idursulfase-IT Other: No IT treatment||Phase 2 Phase 3|
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.
Mucopolysaccharidosis II (MPS II) is a rare, X-linked, inherited disease that affects males nearly exclusively. The disease is caused by the absence of, or deficiency in, the activity of the lysosomal enzyme, iduronate-2-sulfatase (I2S) which acts to cleave O-linked sulfate moieties from the glycosaminoglycan (GAG) molecules dermatan sulfate and heparan sulfate.
Study HGT-HIT-094 is a controlled, randomized, two-arm, open-label, assessor-blinded, multicenter study to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter syndrome and cognitive impairment who have previously received and tolerated a minimum of 4 months of therapy with Elaprase.
Pediatric patients under 3 years of age will be enrolled into a separate substudy to evaluate the safety and efficacy of idursulfase-IT. The separate substudy is open label and single arm. Patients who are enrolled in the substudy will receive idursulfase-IT treatment and follow the same schedule of study visits.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||48 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Controlled, Randomized, Two-arm, Open-label, Assessor-blinded, Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment|
|Study Start Date :||February 1, 2014|
|Actual Primary Completion Date :||September 28, 2017|
|Actual Study Completion Date :||September 28, 2017|
10 mg administered via IT using IDDD (intrathecal drug delivery device) once a month for 52 weeks.
Patients will receive weekly standard of care treatment with IV Elaprase only.
Other: No IT treatment
Standard of Care
Other Name: Non-treatment Control
- Change from baseline in the GCA score after 12 months of treatment at visit week 52, as obtained by DAS-II testing [ Time Frame: Baseline to week 52 ]
- Change from baseline in the adaptive behavior composite (ABC) score after 12 months of treatment at visit week 52, as obtained by VABS-II testing [ Time Frame: Baseline to week 52 ]
- Change from baseline in the GCA score at visit weeks 16, 28 and 40 as obtained by DAS-II testing [ Time Frame: Baseline to week 40 ]
- Change from baseline in the ABC score at visit weeks 16, 28 and 40 as obtained by VABS-II testing [ Time Frame: Baseline to week 40 ]
- Change from baseline in standard scores at visit weeks 16, 28, 40 and 52 in cluster areas of the DAS-II [ Time Frame: Baseline to week 52 ]
- Change from baseline in standard scores at visit weeks 16, 28, 40 and 52 of VABS-II domains [ Time Frame: Baseline to week 52 ]
- Change from baseline in age equivalents at visit weeks 16, 28, 40 and 52 developmental quotients, and T-scores for the subtests of the DAS-II [ Time Frame: Baseline to week 52 ]
- Change from baseline to visit weeks 16, 28, 40 and 52 in age equivalents, developmental quotients, and V-scale scores of the VABS-II subdomains [ Time Frame: Baseline to week 52 ]
- Change from baseline to visit weeks 16, 28, 40 and 52 in the V-scale scores and observed maladaptive levels of the VABS-II Maladaptive Behavior Index and its subscales [ Time Frame: Baseline to week 52 ]
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 ClinicalTrials.gov identifier (NCT number): NCT02055118
|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, 27599|
|Women's and Children's Hospital, 72 King William Road|
|North Adelaide, Australia, 5006|
|The Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|Hôpital Femme Mère Enfant|
|Bron, France, 69677|
|Instituto Nacional de Pediatría|
|Coyoacan, Ciudad De México, Mexico, 04530|
|Hospital Infantil Universitario Niño Jesus|
|Madrid, Spain, 28009|
|Royal Manchester Children's Hospital|
|Manchester, United Kingdom, M13 9WL|
|Study Director:||Shire Study Physician||Shire|