This study is not accepting new patients

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

a study on Hunter Syndrome Mucopolysaccharidosis

Summary

Eligibility
for males (full criteria)
Location
at Oakland, California and other locations
Dates
study started
Principal Investigator
by Paul Harmatz
Headshot of Paul Harmatz
Paul Harmatz

Description

Summary

As the HGT-HIT-046 (NCT01506141) and SHP609-302 (NCT02412787) studies are completed and 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.

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

Keywords

Hunter Syndrome, Mucopolysaccharidosis II, Idursulfase-IT

Eligibility

You can join if…

Open to males

  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).

You CAN'T join if...

  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.

Locations

  • The Regents of the University of California not accepting new patients
    Oakland California 94609 United States
  • Phoenix Childrens Hospital not accepting new patients
    Phoenix Arizona 85015 United States

Lead Scientist at UCSF

  • Paul Harmatz
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.
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