ClinicalTrials.gov record 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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusEnrolling by invitationThe study enrols only people the study team invites, for example patients already at a particular clinic. It is not open to the public.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 2/3A study that tests whether a treatment works while continuing to check safety, in a larger group than a phase 2 study alone. It combines the aims of phase 2 and phase 3 in one protocol.

AgeUp to 18 years old

SexAll

LocationArgentina, Belgium, Canada, Czechia, France, Germany, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom, United States

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label extension enrolls people with Mucopolysaccharidosis II (MPS II) who completed Study DNLI-E-0002 or DNLI-E-0007. All participants receive intravenous tividenofusp alfa (DNL310) and may remain on treatment for up to 5 years. The study collects long-term safety, tolerability, and clinical and biomarker measurements; entrants from DNLI-E-0007 will keep their original treatment assignment blinded.

What it is studying. The study tests the long-term safety, tolerability, and efficacy of intravenous tividenofusp alfa (DNL310), an investigational enzyme replacement therapy described as CNS-penetrant, in people with MPS II.

What the researchers hope to learn. Collect long-term data on safety and tolerability (including infusion-related reactions and treatment-emergent adverse events); monitor biomarkers such as urine glycosaminoglycans and cerebrospinal fluid heparan sulfate; and track clinical measures including adaptive behavior, motor function, and liver and spleen volume over a 5-year period.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: tividenofusp alfa

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive repeated intravenous infusions of tividenofusp alfa (DNL310) and be followed for up to 5 years. Study visits will include safety monitoring (reporting and tracking adverse events and infusion-related reactions), urine testing, MRI scans to assess liver and spleen volume, developmental and functional tests (for example Vineland-3, Bayley Scales, Six-Minute Walk Test), and caregiver global-impression questionnaires. Some assessments include cerebrospinal fluid testing (which requires a spinal tap/lumbar puncture) to measure heparan sulfate. Participants entering from Study DNLI-E-0007 will receive DNL310 in this open-label extension while remaining blinded to their original treatment assignment from that parent study.

How the study is structured. An open-label extension where all participants receive repeated intravenous doses of DNL310 and are followed for up to 5 years. The study is non-randomized with parallel cohorts defined by age and MPS II subtype; there is no masking in this extension, though original treatment assignments for some entrants from DNLI-E-0007 remain blinded to participants, site staff, and the sponsor.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The registry says participants are enrolled by invitation. Who decides who is invited, and how?
The listed sites are in Argentina, Belgium, Canada, Czechia, France, Germany, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Hunter syndrome

Clinical trials10 trials listed for Hunter syndromeEvery trial the registry lists for Hunter syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Hunter syndrome researchLatest monthly update: September 2026. Includes: Brain-targeted enzyme. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Sep 20, 2023
Primary completion: June 2027 (estimated)
Study completion: June 2027 (estimated)
Target enrollment: 99 participants (estimated)
Last updated on registry: Jun 11, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Denali Therapeutics Inc.

Participating sites (29)

Filter by country:

UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609 · United States
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611 · United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601 · United States
UNC Children's Research Institute
Chapel Hill, North Carolina, 27599 · United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229 · United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104 · United States
The University of Texas Medical School at Houston
Houston, Texas, 77030 · United States
Huntsman Cancer Hospital
Salt Lake City, Utah, 84112 · United States
Sanatorio Mater Dei
Buenos Aires · Argentina
Universitair Ziekenhuis Antwerpen
Edegem, Antwerpen, 2650 · Belgium

Terms used on this page · explanations by MedicalUpdates.ai

open-label extension: A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
investigational: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Sponsor: The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
blinded: Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
Cohort: A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
Key Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Key Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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