A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants with Hunter Syndrome

Clinicaltrials.gov ID# 04251026

This is a multicenter, open-label clinical study to assess safety, PK, PD of DNL310.  DNL310 is an investigational central nervous system (CNS) penetrant enzyme replacement therapy (ERT) designed to treat peripheral and CNS manifestations of mucopolysaccharidosis type II (MPS II; Hunter syndrome). It is given as a weekly intravenous infusion.  Eligible participants are aged 2 to 18 years, have a confirmed diagnosis of MPS II, and if receiving IV iduronate 2-sulfatase ERT, must have tolerated a minimum of 4 months of therapy prior to screening.   Exclusion criteria include: 1) unstable medical condition, significant medical or psychological comorbidities that would interfere with safe participation; 2) history of IDS gene therapy or stem cell therapy; 3) history of CNS targeted MPS II ERT within 3months (if >5yo), within 6 months (if <5yo); 4) history of brain surgery, ventriculoperitoneal shunt placement or malfunction in the past 30 days; 5) clinically significant stroke, status epilepticus, head trauma with loss of consciousness or CNS disease not related to MPS II within 1 year; 65) contraindication for lumbar punctures; 7) significant thrombocytopenia or coagulopathy

Recruitment status: 4 sites in USA are actively recruiting, 1 site in Netherlands is recruiting.  BCH is not actively recruiting as yet.

PI:  Dr. Walla Al-Hertani

Contact information: 

Dr. Walla Al-Hertani  -- Walla.Al-Hertani@childrens.harvard.edu

Lora Pixley, study coordinator – Lora.Pixley@childrens.harvard.edu