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

Part of paid clinical trials in Oakland, California.

Sponsor
Denali Therapeutics Inc.
Study ID
NCT06075537
Phase
PHASE2/PHASE3
Status
Enrolling By Invitation

Conditions

  • Mucopolysaccharidosis II

Eligibility Criteria

Sex
ALL
Age
N/A - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • tividenofusp alfa — DRUG
    Intravenous repeating dose

Study Details

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.

Key Dates

Start date
Sep 20, 2023
Status verified
Jun 2026
Primary completion
Jun 30, 2027
Completion
Jun 30, 2027

Study Design

Enrollment
99 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort A2
    Participants with nMPS II, aged ≥5 to ≤10 years
  • Experimental: Cohort B2
    Participants with nMPS II or nnMPS II, aged ≥1 to ≤18 years
  • Experimental: Cohort C2
    Participants with nMPS II, aged \<4 years
  • Experimental: Cohort D2
    Participants with nMPS II or nnMPS II, aged ≤18 years with preexisting hepatomegaly who have never taken standard-of-care ERT
  • Experimental: Cohort E2
    Participants with nMPS II, aged ≥6 years; participants with nnMPS II, aged \<6 or ≥17 years; or participants with nMPS II, aged ≥1 to ≤18 years, with a history of prior HSCT or gene therapy and have completed at least 48 weeks in Study DNLI-E-0001
  • Experimental: Cohort A7
    Participants with nMPS II, aged ≥2 to \<6 years
  • Experimental: Cohort B7
    Participants with nnMPS II, aged ≥6 to \<17 years

Primary Outcome Measure

Incidence and intensity of treatment-emergent adverse events (TEAEs) [ Time Frame: 5 years ]

Locations (8)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609-
Ann and Robert H Lurie Children's Hospital of ChicagoChicagoIllinois60611-
Hackensack University Medical CenterHackensackNew Jersey07601-
UNC Children's Research InstituteChapel HillNorth Carolina27599-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
The University of Texas Medical School at HoustonHoustonTexas77030-
Huntsman Cancer HospitalSalt Lake CityUtah84112-

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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