Status:
ENROLLING_BY_INVITATION
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
Lead Sponsor:
Denali Therapeutics Inc.
Conditions:
Mucopolysaccharidosis II
Eligibility:
All Genders
Up to 18 years
Phase:
PHASE2
PHASE3
Brief 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 (C...
Eligibility Criteria
Inclusion
- Key
- For participants from Study DNLI-E-0002 only: Completed at least through the Week 49 visit in Study DNLI-E-0002 and did not discontinue study intervention early
- For participants from Study DNLI-E-0007 only: Completed the treatment period of 96 weeks in Cohort A for nMPS II participants and 48 weeks in Cohort B for nnMPS II participants
- Key
Exclusion
- Unstable or poorly controlled medical condition(s) or significant medical or psychological comorbidity or comorbidities that, in the opinion of the investigator, would interfere with safe participation in the trial or interpretation of study assessments
Key Trial Info
Start Date :
September 20 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2027
Estimated Enrollment :
99 Patients enrolled
Trial Details
Trial ID
NCT06075537
Start Date
September 20 2023
End Date
June 1 2027
Last Update
December 24 2025
Active Locations (22)
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1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
2
UNC Children's Research Institute
Chapel Hill, North Carolina, United States, 27599
3
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104