A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Launched by DENALI THERAPEUTICS INC. · May 9, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called tividenofusp alfa (DNL310) to see if it is effective and safe for children and young adults with a condition known as Mucopolysaccharidosis Type II (MPS II). MPS II can affect the nervous system and lead to various health issues. The trial is comparing tividenofusp alfa to a standard treatment called idursulfase. It is designed for participants aged 2 to 25 years who have a confirmed diagnosis of MPS II and have not received certain previous treatments.

If you or your child are eligible, you will be randomly assigned to receive either the new treatment or the standard one, and you will not know which one you are getting. The study is currently recruiting participants, and there may be opportunities for those in the trial to receive open-label treatment with either medication after the initial phase. It’s important to note that participants must meet specific health criteria and not have certain other medical conditions. This trial aims to gather more information to help improve treatment options for MPS II.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<26 years (Cohort B)
• • Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
• • Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening
• Key Exclusion Criteria:
• • Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
• • Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy
• • Received any CNS-targeted MPS ERT within 6 months prior to screening
• • Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI)
• • Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study