Active — Not RecruitingPhase 1/2

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

United States, Canada47 participantsStarted 2020-07-16

Plain-language summary

This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.

Who can participate

Age range18 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Key Inclusion Criteria: * Confirmed diagnosis of MPS II * Cohort A: Participants aged ≥5 to ≤10 years with neuronopathic MPS II * Cohort B: Participants aged ≥1 to ≤18 years with non-neuronopathic MPS II, neuronopathic MPS II, or unknown phenotype * Cohort C: Participants aged \<4 years with neuronopathic MPS II (this cohort can include participants ≥4 to ≤18 years of age if participant is a blood relative of a participant \<4 years of age) * Cohort D: Participants aged ≤18 years with non-neuronopathic MPS II or neuronopathic MPS II with preexisting hepatomegaly who have never taken standard-of-care ERT * Cohort E: neuronopathic MPS II participants aged ≥6 years at screening, non-neuronopathic MPS II participants \<6 or ≥17 years at screening, and neuronopathic MPS II participants ≥1 to ≤18 years at screening with a history of prior haematopoietic stem cell transplantation or gene therapy who have completed at least 48 weeks in Study DNLI-E-0001 * For participants receiving intravenous iduronate 2-sulfatase (IDS) ERT, tolerated a minimum of 4 months of therapy during the period immediately prior to screening. Key Exclusion Criteria: * 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 * Use of any CNS-targeted MPS II ERT within 3 months before study start for participants a…

What they're measuring

Incidence and severity of treatment-emergent adverse events (TEAEs)

Timeframe: 24 weeks, 104 weeks, and 357 weeks

Change from baseline in urine total glycosaminoglycan (GAG) concentrations

Timeframe: 24 weeks, 104 weeks, and 357 weeks

Incidence and severity of infusion-related reactions (IRRs)

Timeframe: 24 weeks, 104 weeks, and 357 weeks

Change from baseline in concomitant medications

Timeframe: 24 weeks, 104 weeks, and 357 weeks

Trial details

NCT IDNCT04251026

SponsorDenali Therapeutics Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2031-02

View on ClinicalTrials.gov More Mucopolysaccharidosis II trials

Plain-language summary

Who can participate

Age range18 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Incidence and severity of treatment-emergent adverse events (TEAEs)

Timeframe: 24 weeks, 104 weeks, and 357 weeks

Change from baseline in urine total glycosaminoglycan (GAG) concentrations

Timeframe: 24 weeks, 104 weeks, and 357 weeks

Incidence and severity of infusion-related reactions (IRRs)

Timeframe: 24 weeks, 104 weeks, and 357 weeks

Change from baseline in concomitant medications

Timeframe: 24 weeks, 104 weeks, and 357 weeks