An open, single-arm, multicenter extension study to assess the safety, tolerability and efficacy of long-term SOBI003 treatment in pediatric MPS IIIA patients
Study ID: STU-2019-0796
This is an open, single-arm, multicenter extension study to assess the safety, tolerability and efficacy of long-term SoBi003 treatment in pediatric MPS iiia patients. The study is an extension of the First in Human (FiH) SoBi003-001 study, allowing continuous treatment of SoBi003 for up to 2 years. Study patients who complete Week 24 of the FiH study (SoBi003-001) will be invited to continue to Week 25 in the extension study.
SoBi003 is being developed for treatment of MPS iiia. Scientific advice on the nonclinical and chemistry, manufacturing and control (CMC) development has been obtained from the FDa on 16 February 2016 and from eMa in 28 June 2016. a pre-inD meeting was held with the FDa on 21 June 2017 to obtain agreement on the conduct of the FiH study (SoBi003-001) with SoBi003. The FiH study (SoBi003-001) is designed to assess the safety, tolerability, PK, PD and immunogenicity of SoBi003 at 3 dose levels to provide knowledge needed to optimize the further evaluation of SoBi003 in patients with MPS iiia. Patients completing the 24-week, open-label treatment period of the FiH study will be offered enrolment into the extension study SoBi003-002. Study SoBi003-002 is designed to assess the safety and tolerability of long-term SoBi003 treatment and to evaluate the efficacy of SoBi003 in improving neurocognitive function and other measures indicative of a favorable outcome of SoBi003 treatment. The follow-up period of the extension study is 80 weeks, giving a total SoBi003 treatment duration of 104 weeks to allow for a comparison with data from a recent nH study (22).