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New hope for hunter syndrome: Long-Term drug trial launches

NCT ID NCT06075537

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at the long-term safety and effects of an experimental drug called DNL310 for people with Hunter syndrome (MPS II), a rare genetic disorder. About 99 participants who completed earlier studies will receive the drug for up to 5 years. Researchers will monitor side effects and measure changes in disease markers and development.

Why investors are watching

Denali Therapeutics is running an open-label extension study of tividenofusp alfa, an enzyme replacement therapy for Hunter syndrome, a rare genetic disease. This trial tests long-term safety and effectiveness over up to five years in patients who completed earlier studies. For a small company, this readout matters because it could support regulatory approval and define the drug's commercial value.

If it works: If the long-term data show the drug remains safe and controls symptoms, Denali could gain a stronger case for approval and a durable treatment option for a rare disease. That could make the company more attractive to partners or buyers.

If it fails: Long-term trials can reveal new safety problems or show that the drug stops working over time. If the results disappoint, Denali may face delays or fail to get approval, which would hurt the company's prospects.

AI-written from the trial record. Speculative, and not investment advice.

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

About 99 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2023

Expected to finish

Jun 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

Up to 18 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: * 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

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    29 sites in 13 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • ASST di Cremona

    Cremona, Italy

  • 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, 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

  • Drottning Silvias Barn Och Ungdomssjukhus

    Gothenburg, Sweden

  • Erasmus Medical Center - Sophia Children's Hospital

    Rotterdam, 3015 GD, Netherlands

  • Gazi Universitesi Tip Fakultes

    Ankara, Turkey (Türkiye)

  • Great Ormond Street Hospital

    London, WC1N 3JH, United Kingdom

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Hopital Jeanne De Flandre - Metabolic Diseases Unit

    Lille, France

  • Hospit U. Vall d'Hebron - PPDS

    Barcelona, Spain

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • Huntsman Cancer Hospital

    Salt Lake City, Utah, 84112, United States

  • McGill University Health Center

    Montreal, Quebec, H4A3J1, Canada

  • Royal Free Hospital

    London, NW3 2QG, United Kingdom

  • Royal Manchester Children's Hospital

    Manchester, United Kingdom

  • Sanatorio Mater Dei

    Buenos Aires, Argentina

  • SpinCS GmbH

    Höchheim, 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, 27599, United States

  • UZ Brussel

    Jette, Belgium

  • Universitair Ziekenhuis Antwerpen

    Edegem, Antwerpen, 2650, Belgium

  • University Medical Faculty Balcali Hospital

    Adana, Turkey (Türkiye)

  • University of Alberta - Faculty of Medicine & Dentistry

    Edmonton, Alberta, Canada

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, Germany

  • Vseobecna Fakultni Nemocnice V Praze

    Prague, 120 00, Czechia

More trials for these conditions

Other studies related to the condition(s) this trial covers.