Clinical Trials

Date: 2017-12-19

Type of information: Results

phase: 2-3

Announcement: results

Company: Shire (UK - USA)

Product: SHP609 (idursulfase-IT)

Action mechanism:

enzyme replacement therapy. SHP609 is an investigational formulation of idursulfase administered intrathecally for a new potential indication for the treatment of pediatric patients with Hunter syndrome (mucopolysaccharidosis II or MPS II) and cognitive impairment.
SHP609 is being investigated and developed for use with Shire’s current treatment for Hunter syndrome, Elaprase® (idursulfase) which is administered intravenously and does not cross the blood-brain barrier in clinically relevant amounts.

Disease: mucopolysaccharidosis II (MPS II, Hunter Syndrome)

Therapeutic area: Rare diseases - Genetic diseases

Country: Australia, Canada, France, Mexico, Spain, UK, USA

Trial details:

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®. The trial had an enrollment of 48 patients with Hunter syndrome and cognitive impairment who continued to receive and tolerate therapy with intravenous idursulfase.
Cognition was assessed by the Differential Ability Scale, Second Edition (DAS-II). One of the key secondary endpoints measured adaptive ability based on the Vineland Adaptive Behavioral Scales, Second Edition (VABS-II).8 VABS-II assesses the development of personal and social skills, including talking, walking and motor skills, as well as interpersonal relationships and coping skills.9 The safety profile observed was generally consistent with that seen in previous studies. Idursulfase-IT has been well studied with up to 67 months of patient exposure data. Of 15 patients in the Phase I/II trial of SHP609, all had ?1 Treatment-Emergent Adverse Events (TEAE) and all had ?1 drug-related TEAE. Of 54 serious TEAE types, 2 were believed to be causally related to idursulfase-IT: pyrexia (71 events, 3.3%) and vomiting (63 events, 2.9%). (NCT02055118)

Latest news:

• On December 19, 2017, Shire announced top-line results from its Phase II/III clinical trial evaluating SHP609, previously known as HGT-2310. The study did not meet either its primary or its key secondary endpoint. The primary endpoint evaluated the difference in cognition between the SHP609-treated and control groups, as measured by change from baseline in General Conceptual Ability (GCA) scores in children with Hunter syndrome after 12 months of treatment. The key secondary endpoint evaluated the difference between the SHP609-treated and control groups as measured by the change from baseline in Adaptive Behavior Composite (ABC) score.