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New enzyme therapy aims to reach the brain in MPS II

NCT ID NCT05371613

Disease control Sponsor: Denali Therapeutics Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 09, 2026 · Last updated Sep 10, 2026 · Updated 1 time

Summary

Researchers are testing tividenofusp alfa (DNL310), an experimental enzyme replacement therapy, against the standard treatment idursulfase in children and young adults with mucopolysaccharidosis type II (MPS II). The study includes participants with and without brain involvement, aged 2 to 26. The main goal is to see if DNL310 can lower harmful substances in the spinal fluid and improve adaptive behavior.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
tividenofusp alfa (DNL310), an experimental enzyme replacement therapy designed to reach the brain
What this could lead to
If it works, this could offer a treatment that reaches the brain and better controls MPS II symptoms, especially thinking and behavior problems.
What could go wrong
The trial is small and early, so results may not hold up in larger studies. Enzyme therapies can cause infusion reactions, and the brain-penetrating version is still experimental.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

65 people

The number who actually took part.

Started

Jul 2022

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 25 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<26 years (Cohort B) * Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II) * Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening Key Exclusion Criteria: * Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay * Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy * Received any CNS-targeted MPS ERT within 6 months prior to screening * Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI) * Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 2 Sudden Infant Death

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mucopolysaccharidosis ii

Contacts and locations

Locations

  • Ann and Robert H Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia

    Udine, 33100, Italy

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, United Kingdom

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Cukurova University Medical Faculty Balcali Hospital

    Adana, 1330, Turkey (Türkiye)

  • Drottning Silvias Barn Och Ungdomssjukhus

    Gothenburg, 416 85, Sweden

  • Erasmus Medical Center - Sophia Children's Hospital

    Rotterdam, Rotterdam, 3000, Netherlands

  • Gazi Universitesi Tip Fakultesi

    Çankaya, 06500, Turkey (Türkiye)

  • Great Ormond Street Hospital for Children

    London, London, WC1N 3JH, United Kingdom

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Hospital Infantil Universitario Niño Jesus

    Madrid, Madrid, 28009, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, Barcelona, 08035, Spain

  • Hospital de Clínicas de Porto Alegre (HCPA) - PPDS

    Porto Alegre, Brazil

  • Hospital for Sick Children

    Toronto, Ontario, M5G1X8, Canada

  • Hôpital Jeanne de Flandre

    Lille, 59000, France

  • McGill University Health Center

    Montreal, Quebec, H4A3J1, Canada

  • Medizinische Universität Lausitz - Carl Thiem

    Cottbus, Germany

  • Royal Free Hospital

    London, NW3 2QG, United Kingdom

  • Salford Royal Hospital

    Salford, M6 8HD, United Kingdom

  • Sanatorio Mater Dei

    Buenos Aires, Argentina

  • SphinCS

    Höchheim, Hochheim, 65239, Germany

  • The University of Texas Medical School at Houston

    Houston, Texas, 77030, United States

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • UNC Children's Research Institute

    Chapel Hill, North Carolina, 27514, United States

  • UZ Antwerpen

    Antwerp, Antwerpen, 2650, Belgium

  • Universitair Ziekenhuis Brussel

    Jette, Brussels Capital, 1090, Belgium

  • University of Alberta - Faculty of Medicine & Dentistry

    Edmonton, Alberta, Canada

  • University of Utah, PPDS

    Salt Lake City, Utah, 84132, United States

  • Vseobecna Fakultni Nemocnice V Praze

    Prague, 128 08, Czechia

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