NCT06075537

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

• Status: Enrolling By Invitation
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 99 (estimated)
• Sponsor: Denali Therapeutics Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous (IV) enzyme replacement therapy (ERT) for Hunter syndrome (MPS II). Participants who complete at least through the Week 49 visit in Study DNLI-E-0002 and do not discontinue study intervention early and participants who complete Study DNLI-E-0007 will be enrolled in this OLE. All participants will receive DNL310 for up to 5 years from the time of entry in this OLE. Participants, site staff, and the Sponsor will remain blinded to the original treatment assignment for participants entering this OLE from Study DNLI-E-0007.

Conditions

• Mucopolysaccharidosis II

Interventions

Type	Name	Description
DRUG	tividenofusp alfa	Intravenous repeating dose

Timeline

• Start date: 2023-09-20
• Primary completion: 2027-06-01
• Completion: 2027-06-01
• First posted: 2023-10-10
• Last updated: 2026-03-11

Locations

25 sites across 13 countries: United States, Argentina, Belgium, Canada, Czechia, France, Germany, Italy, Netherlands, Spain, Sweden, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study