Clinical trial NCT05795361: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome
Official title: Post-trial Access Program: Idursulfase-IT (HGT-2310) in Conjunction With Intravenous Elaprase® in Pediatric and Adult Patients With Hunter Syndrome and Cognitive Impairment
Study type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Hunter Syndrome
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2023-04-03
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no

Brief summary: As the HGT-HIT-046 (NCT01506141) and SHP609-302 (NCT02412787) studies will be closed, this post-trial access (PTA) program provides TAK-609 to participants in these studies for whom the benefit:risk ratio of continued treatment with idursulfase-IT remains positive.


Eligibility criteria: Inclusion Criteria:

1. Participants will have completed the treatment period of the HGT-HIT-046 (NCT01506141) or SHP609-302 (NCT02412787) study prior to the first dose on this program.
2. Participant and/or a parent(s)/legal guardian is informed of the nature of this compassionate post-trial access program and can provide written informed consent for themselves or the child to participate (with assent from the child when appropriate prior to treatment).

Exclusion Criteria:

1. Participant has a condition that in the opinion of the treating physician may compromise their safety.
2. Participant has a known hypersensitivity to idursulfase-IT or its components.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Childrens Hospital
state: Arizona
city: Oakland
country: United States
facility: The Regents of the University of California
state: California
city: Wilmington
country: United States
facility: The Nemours Foundation
state: Delaware
city: Miami
country: United States
facility: Jackson Memorial Hospital University of Miami
state: Florida
city: Chicago
country: United States
facility: Ann and Robert H Lurie Childrens Hospital of Chicago
state: Illinois
city: Washington
country: United States
facility: Washington University
state: Missouri
city: Omaha
country: United States
facility: Board of Regents of the University of Nebraska
state: Nebraska
city: Hackensack
country: United States
facility: Joseph M. Sanzari Children's Hospital
state: New Jersey
city: New York
country: United States
facility: NYU Langone Medical Center
state: New York
city: Chapel Hill
country: United States
facility: The University of North Carolina
state: North Carolina
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Portland
country: United States
facility: Randall Children's Hospital at Legacy Emanuel
state: Oregon
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Childrens Hospital of Pittsburgh
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Children's Hospital
state: Tennessee
city: Salt Lake City
country: United States
facility: Division of Medical Genetics, University of Utah
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital - PIN
state: Washington
city: South Brisbane
country: Australia
facility: Queensland Childrens Hospital
state: Queensland
city: Coyoacan
country: Mexico
facility: Instituto Nacional de Pediatria
state: Ciudad De Mexico
city: Valladolid
country: Mexico
facility: H.C.U. de Valladolid
city: Cordoba
country: Spain
facility: Hospital Universitario Reina Sofia
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital - PPDS
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: