MPS II Treatment with DNL310 – Phase I/II

Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Subjects with Hunter Syndrome

Protocol Description

The purpose of this multi-center global clinical research study is to see if the new compound DNL310, an enzyme replacement therapy (ERT) that is thought to penetrate the central nervous system (CNS), is safe and tolerable when given to patients with mucopolysaccharidosis type II (MPS II), also called Hunter syndrome. In this inherited genetic condition, a defective iduronate-2-sulfatase gene (IDS) prevents production of the enzyme iduronate-2-sulfatase (I2S). The lack of circulating I2S enzyme can lead to progressive damage to the brain, heart, and other organs. The study drug has been designed to deliver I2S to both the body and brain.

Study Level: Phase I/II

Eligibility Criteria

Subject to exclusion criteria, this study is enrolling male participants ages 2 through 18 years with a confirmed diagnosis of MPS II.
Males: Ages 2 through 18 years

Requirements

During the treatment period, participants will receive weekly intravenous infusions of DNL310 at dose levels specified by the study protocol. Examinations and other assessments will also be done during study visits.
Visits: 17, including screening
Duration: 24 weeks

Status: Open to Enrollment

Source(s) of Support

Denali Therapeutics Inc.

Primary Investigator

Deepa Soundara Rajan, MD

Contact Information

For more information about the study or enrollment, please contact: 
Dawn Kolar, Clinical Research Coordinator 
412-692-8343
kolardr@upmc.edu