Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Shire

Status and phase: Completed; Phase 3; Phase 2

Conditions: Hunter Syndrome

Treatments: Drug: Idursulfase-IT; Drug: Elaprase

Study type: Interventional

Funder types: Industry

Identifiers

NCT02412787

SHP609-302

2014-004143-13 (EudraCT Number)

Details and patient eligibility

About

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 receive idursulfase IT treatment in Study HGT-HIT-094.

Enrollment: 56 patients

Sex: Male

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants must have completed Visit Week 52 assessments in Study HGT-HIT-094 (NCT02055118).
• 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 consent/assent, as relevant, must be obtained.
• The participant has continued to receive Elaprase on a regular basis in Study HGT-HIT-094 (NCT02055118).

Exclusion criteria

• The participant 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.

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

• The participant has clinically relevant intracranial hypertension.

• The participant is enrolled in another clinical study, other than HGT-HIT-094 (NCT02055118), 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.

• The participant 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 participant has a condition that is contraindicated as described in the SOPH-A-PORT® Mini S, Implantable Access Port, Spinal, Mini Unattached, with Guidewire (SOPH-A-PORT Mini S) intrathecal drug delivery device (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 1 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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56 participants in 1 patient group

Idursulfase-IT

Experimental group

Description:

Participants received 10 milligrams (mg) of idursulfase-IT intrathecally via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once every 28 days along with standard-of-care therapy with Elaprase, for 470 weeks (for participants who began intrathecal \[IT\] treatment in this study) and 480 weeks (for participants who began IT treatment in HGT-HIT-094 and continued to receive IT treatment in this study). Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (\>8 months to 30 months of age) or 10 mg (\>30 months to 3years of age) of idursulfase-IT.

Treatment:

Drug: Elaprase

Drug: Idursulfase-IT