MedDataX Logo

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

NCT ID: NCT06075537

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2/PHASE3

Total Enrollment

99 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-09-20

Study Completion Date

2027-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Mucopolysaccharidosis II

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Hunter Syndrome MPS II nMPS II nnMPS II

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Cohort A2

Participants with nMPS II, aged ≥5 to ≤10 years

Group Type EXPERIMENTAL

tividenofusp alfa

Intervention Type DRUG

Intravenous repeating dose

Cohort B2

Participants with nMPS II or nnMPS II, aged ≥1 to ≤18 years

Group Type EXPERIMENTAL

tividenofusp alfa

Intervention Type DRUG

Intravenous repeating dose

Cohort C2

Participants with nMPS II, aged \<4 years

Group Type EXPERIMENTAL

tividenofusp alfa

Intervention Type DRUG

Intravenous repeating dose

Cohort D2

Participants with nMPS II or nnMPS II, aged ≤18 years with preexisting hepatomegaly who have never taken standard-of-care ERT

Group Type EXPERIMENTAL

tividenofusp alfa

Intervention Type DRUG

Intravenous repeating dose

Cohort E2

Participants with nMPS II, aged ≥6 years; participants with nnMPS II, aged \<6 or ≥17 years; or participants with nMPS II, aged ≥1 to ≤18 years, with a history of prior HSCT or gene therapy and have completed at least 48 weeks in Study DNLI-E-0001

Group Type EXPERIMENTAL

tividenofusp alfa

Intervention Type DRUG

Intravenous repeating dose

Cohort A7

Participants with nMPS II, aged ≥2 to \<6 years

Group Type EXPERIMENTAL

tividenofusp alfa

Intervention Type DRUG

Intravenous repeating dose

Cohort B7

Participants with nnMPS II, aged ≥6 to \<17 years

Group Type EXPERIMENTAL

tividenofusp alfa

Intervention Type DRUG

Intravenous repeating dose

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

tividenofusp alfa

Intravenous repeating dose

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

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
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Denali Therapeutics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Jose Alcantara Rodriguez, PharmD

Role: STUDY_DIRECTOR

Denali Therapeutics

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Site Status

UNC Children's Research Institute

Chapel Hill, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

The University of Texas Medical School at Houston

Houston, Texas, United States

Site Status

Huntsman Cancer Hospital

Salt Lake City, Utah, United States

Site Status

Sanatorio Mater Dei

Buenos Aires, , Argentina

Site Status

Universitair Ziekenhuis Antwerpen

Edegem, Antwerpen, Belgium

Site Status

UZ Brussel

Jette, , Belgium

Site Status

University of Alberta - Faculty of Medicine & Dentistry

Edmonton, Alberta, Canada

Site Status

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

McGill University Health Center

Montreal, Quebec, Canada

Site Status

Vseobecna Fakultni Nemocnice V Praze

Prague, , Czechia

Site Status

Hopital Jeanne De Flandre - Metabolic Diseases Unit

Lille, , France

Site Status

Universitätsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Site Status

SpinCS GmbH

Höchheim, , Germany

Site Status

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

Udine, , Italy

Site Status

Erasmus Medical Center - Sophia Children's Hospital

Rotterdam, , Netherlands

Site Status

Hospit U. Vall d'Hebron - PPDS

Barcelona, , Spain

Site Status

Drottning Silvias Barn Och Ungdomssjukhus

Gothenburg, , Sweden

Site Status

University Medical Faculty Balcali Hospital

Adana, , Turkey (Türkiye)

Site Status

Gazi Universitesi Tip Fakultes

Ankara, , Turkey (Türkiye)

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Argentina Belgium Canada Czechia France Germany Italy Netherlands Spain Sweden Turkey (Türkiye)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2023-503837-23

Identifier Type: OTHER

Identifier Source: secondary_id

DNLI-E-0008

Identifier Type: -

Identifier Source: org_study_id