An 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

Takeda

Status and phase: Completed; Phase 2; Phase 1

Conditions: Hunter Syndrome

Treatments: Drug: Elaprase; Drug: Idursulfase-IT

Study type: Interventional

Funder types: Industry

Identifiers

NCT01506141

HGT-HIT-046

2011-000212-25 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 15 patients

Sex: Male

Ages: 3 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Eligibility Criteria

Inclusion Criteria:

• Participant must have completed all study requirements and End of study (EOS) assessments for study HGT-HIT-045 (NCT00920647) 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 (NCT00920647) 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

     An additional exclusion criterion for patients who were previously untreated with intrathecal idursulfase-IT in Study HGT-HIT-045 (NCT00920647):

• The participant has an opening CSF pressure upon lumbar puncture that exceeds 30.0 centimeter (cm) water (H2O).

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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15 participants in 3 patient groups

Idursulfase-IT 1 milligram (mg)

Experimental group

Description:

Participants will receive 1 mg idursulfase-IT intrathecally via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once monthly and standard-of-care (SoC) therapy of Elaprase intravenous (IV) infusions.

Treatment:

Drug: Idursulfase-IT

Drug: Elaprase

Idursulfase-IT 10 mg

Experimental group

Description:

Participants will receive 10 mg idursulfase-IT intrathecally via IDDD or LP once monthly and SoC therapy of Elaprase IV infusions.

Treatment:

Drug: Idursulfase-IT

Drug: Elaprase

Idursulfase-IT 30 mg

Experimental group

Description:

Participants will receive 30 mg idursulfase-IT intrathecally via IDDD or LP once monthly and SoC therapy of Elaprase IV infusions.

Treatment:

Drug: Idursulfase-IT

Drug: Elaprase