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)

Part of paid clinical trials in Oakland, California.

Sponsor
Denali Therapeutics Inc.
Study ID
NCT05371613
Phase
PHASE2/PHASE3
Status
Recruiting
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Conditions

  • Mucopolysaccharidosis II

Eligibility Criteria

Sex
ALL
Age
2 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

  • tividenofusp alfa — DRUG
    Intravenous repeating dose
  • idursulfase — DRUG
    Intravenous repeating dose

Study Details

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 nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.

Key Dates

Start date
Jul 21, 2022
Status verified
Aug 2025
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
63 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort A: Participants with nMPS II
  • Experimental: Cohort B: Participants with nnMPS II
  • Experimental: Open-label Treatment Phase
    Participants who meet pre-specified criteria may receive DNL310 or idursulfase

Primary Outcome Measure

Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A) [ Time Frame: 24 weeks ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609
Marissa Evans
[email protected]
Paul Harmatz, MD (PRINCIPAL_INVESTIGATOR)
Ann and Robert H Lurie Children's Hospital of ChicagoChicagoIllinois60611
Lindsey Lang
[email protected]
312-227-6712
Barbara Burton, MD (PRINCIPAL_INVESTIGATOR)
Hackensack University Medical CenterHackensackNew Jersey07601
Susan Mathus
[email protected]
Helio Pedro, MD (PRINCIPAL_INVESTIGATOR)
UNC Children's Research InstituteChapel HillNorth Carolina27514
Lindsay Torrice
[email protected]
Joseph Muenzer, MD (PRINCIPAL_INVESTIGATOR)
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229
Laurie Bailey
[email protected]
Nancy Leslie, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Genevieve Nesom
[email protected]
Can Ficicioglu, MD (PRINCIPAL_INVESTIGATOR)
The University of Texas Medical School at HoustonHoustonTexas77030
Marilyn Garcia
[email protected]
Paul Hillman, MD (PRINCIPAL_INVESTIGATOR)
University of Utah, PPDSSalt Lake CityUtah84132
Carrie Bailey
[email protected]
David Viskochil, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Oakland, CA

By research site
UCSF Benioff Children's Hospital Oakland· Oakland, CAAnn and Robert H Lurie Children's Hospital of Chicago· Chicago, ILHackensack University Medical Center· Hackensack, NJUNC Children's Research Institute· Chapel Hill, NCCincinnati Children's Hospital Medical Center· Cincinnati, OHChildren's Hospital of Philadelphia· Philadelphia, PA

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