Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

Completed · Phase 2/Phase 3

Conditions studied: Hunter Syndrome

In brief

Study HGT-HIT-094 is a multicenter study designed 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.

Key facts

Study ID

NCT02055118

Run by

Shire

People needed

58

Starts

2014-03-24

Expected to finish

2017-09-28

Last updated by the study team

2021-06-11

Who can join

Age: any, up to 18. Sex: male. Healthy volunteers: not accepted.

You may not qualify if…

• Patients who meet any of the following criteria are not eligible to be randomized into the pivotal study or enrolled in the separate substudy:
• The patient has clinically significant non-Hunter syndrome-related CNS involvement (such as Fragile-X syndrome) which is judged by the Investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
• The patient has a large chromosomal deletion or complex rearrangement that includes a deletion of the FMR1 and/or FMR2 genes.
• The patient has a significant medical or psychiatric comorbidity(ies) that might affect study data or confound the integrity of study results.
• The patient has contra-indications for performance of lumbar puncture such as musculoskeletal/spinal abnormalities or risk of abnormal bleeding.
• The patient has a history of complications from previous lumbar punctures or technical challenges in conducting lumbar punctures such that the potential risks would exceed possible benefits for the patient.
• The patient has an opening CSF pressure upon lumbar puncture that exceeds 30.0 cm H2O.
• The patient has experienced infusion-related anaphylactoid event(s) or has evidence of consistent severe adverse events related to treatment with Elaprase which, in the Investigator's opinion, may pose an unnecessary risk to the patient.
• The patient has received a cord blood or bone marrow transplant at any time or has received blood product transfusions within 90 days prior to Screening.
• The patient has a history of poorly controlled seizure disorder.
• The patient is unable to comply with the protocol (eg, has significant hearing or vision impairment, a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, known clinically significant psychiatric/behavioral instability, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator.
• The patient is enrolled in another clinical study that involves clinical investigation 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.
• 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.
• The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use (IFU), including but not limited to the presence of a CSF shunt device in the patient.

Where it is running

• Children's Hospital and Research Center at Oakland — Oakland, California, United States
• Ann & Robert H Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of North Carolina — Chapel Hill, North Carolina, United States
• Women's and Children's Hospital, 72 King William Road — North Adelaide, Australia
• The Hospital for Sick Children — Toronto, Ontario, Canada
• Hôpital Femme Mère Enfant — Bron, France
• Instituto Nacional de Pediatría — Coyoacán, Mexico City, Mexico
• Hospital Infantil Universitario Niño Jesus — Madrid, Spain
• Royal Manchester Children's Hospital — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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