Status:
RECRUITING
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)
Lead Sponsor:
Denali Therapeutics Inc.
Conditions:
Mucopolysaccharidosis II
Eligibility:
All Genders
2-25 years
Phase:
PHASE2
PHASE3
Brief Summary
This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central n...
Eligibility Criteria
Inclusion
- Key
- 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
- 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
Key Trial Info
Start Date :
July 21 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2027
Estimated Enrollment :
63 Patients enrolled
Trial Details
Trial ID
NCT05371613
Start Date
July 21 2022
End Date
December 1 2027
Last Update
August 5 2025
Active Locations (32)
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1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
2
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
3
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
4
UNC Children's Research Institute
Chapel Hill, North Carolina, United States, 27514