Update: April 27, 2021
Last year Sobi decided to halt development of this ERT program. The patients enrolled in the trial were provided access to drug for the full 2 years of the planned trial period. All patients have now completed the trial and transitioned off of treatment. Patient families observed improvements in their children’s quality of life while on this enzyme replacement and are devastated by the loss of access to this drug. Data from the clinical trial has not been released publicly by the company yet. This clinical program is available for divestment to other interested industry partners.
March 9, 2020
The aims of the present study is to assess the dose related safety, tolerability, PK and PD of SOBI003, a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
This is an open-label, non-controlled, parallel, sequential ascending multiple-dose, multicenter study to assess the dose related safety, tolerability, PK and PD of SOBI003 in pediatric MPS IIIA patients. Patients between 1 and 6 years of age who have not received previous treatment for MPS IIIA with an ERT, gene- or stem cell therapy will be eligible to participate in the study. The study is planned to consist of 3 dose cohorts, each comprising 3 patients. Treatment initiations will be staggered within each cohort in order to be able to observe, interpret and treat possible adverse reactions. SOBI003 is administered as weekly i.v. infusions over a period of 24 weeks. Upon completion of the 24-week treatment period with satisfactory tolerability, the patient is offered to receive continued SOBI003 treatment by participation in an extension study.
The trial has finished enrollment (an estimated 9 patients) and is no longer recruiting. There are four trial locations globally, including two in the United States (California and North Carolina). The others are in Germany and Turkey.
Read this clinical trial’s information on ClinicalTrials.gov, which includes enrollment criteria, trial locations, and study contact information.