Intrathecal Enzyme Replacement Therapy Sanfilippo A
Shire Human Genetics
Dr. Simon Jones
Dr. Frits Wijberg
A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan N-Sulfatase (rhHNS) Intrathecal Administration Via an Intrathecal Drug Delivery Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Shire Human Genetic Therapies (Shire HGT) is developing a sulfamidase enzyme replacement therapy (ERT)rhHNS for patients with MPS IIIA. rhHNS is being administered into the cerebrospinal fluid (CSF) via a surgically implanted intrathecal drug delivery device (IDDD).
This study is a multicenter, multiple-dose, dose escalation study designed to evaluate the safety, tolerability, and clinical activity of up to 3 dose levels (2 doses [10 and 45mg] monthly and 1 dose [45mg] every other week for 6 months) of rhHNS administered via an IDDD in patients with Sanfilippo syndrome Type A ages greater than or equal to 3 years of age.
The phase I/II clinical trial is planning to enroll 15 patients, beginning June 2010. The study is expected to be completed March 2012, and the duration of the study for each patient is nine months.
The Phase I / II clinical study is being conducted at two sites:
- Emma Children’s Hospital, Academic Medical Center in The Netherlands by Dr. Frits Wijberg
- St. Mary’s Hospital in Manchester, UK under the direction of Drs. Simon Jones and Ed Wraith.