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Enrolling by Invitation

NCT Number: NCT06075537

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

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.

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Key information

Conditions

Mucopolysaccharidosis II Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Death Death, Sudden Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Infant Death Intellectual Disability Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Skin and Connective Tissue Diseases Sudden Infant Death X-Linked Intellectual Disability

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Sanatorio Mater Dei, Buenos Aires, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • 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 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

Treatment and study plan

tividenofusp alfa

Drug

Intravenous repeating dose

Primary outcomes

  1. Incidence and intensity of treatment-emergent adverse events (TEAEs)

    Time frame: 5 years

  2. Clinically significant changes in urine total glycosaminoglycan (GAG) concentrations throughout the treatment period

    Time frame: 5 years

  3. Incidence and intensity of infusion-related reactions (IRRs)

    Time frame: 5 years

    The intensity of IRRs will be assessed following each infusion of DNL310 using the categories of Mild, Moderate and Severe. IRRs will be summarized overall as well as stratified by intensity.

Secondary outcomes

  1. Percentage change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration

    Time frame: 5 years

  2. Change from baseline in the Vineland-3 Adaptive Behavior Scale

    Time frame: 5 years

  3. Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) cognitive raw score

    Time frame: 5 years

  4. Change from baseline in distance walked (meters) in the Six-Minute Walk Test (6MWT)

    Time frame: 5 years

  5. Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations

    Time frame: 5 years

  6. Liver volume within the normal range (normal vs abnormal) as measured by MRI

    Time frame: 5 years

  7. Spleen volume within the normal range (normal vs abnormal) as measured by MRI

    Time frame: 5 years

  8. Improvement in the Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II

    Time frame: 5 years

Sponsors and collaborators

Lead sponsor

Denali Therapeutics Inc.

Industry

Registry information

Official study title

An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Important dates

Study start
2023
Primary completion
2027
Study completion
2027
First posted
Oct 10, 2023
Registry last updated
Jun 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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