A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

NCT05371613 · tracked via the Priya Life Science USA tracker

Sponsor

Denali Therapeutics Inc.

Phase

Phase 2/3

Started

2022-07-21

Last updated

2026-09-09

Condition(s) studied

Mucopolysaccharidosis II

Investigational drug(s) / intervention(s)

tividenofusp alfaidursulfase

tividenofusp alfa: Intravenous repeating dose

idursulfase: Intravenous repeating dose

Study summary

This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II).

Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.

Eligibility

Sex

ALL

Min age

2 Years

Max age

25 Years

Healthy volunteers

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

Primary outcome measure(s)

Ratio to baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A) — 24 weeks
Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A) — 96 weeks

Trial sites (30)

Facility	City	Region
UCSF Benioff Children's Hospital Oakland	Oakland	California
Ann and Robert H Lurie Children's Hospital of Chicago	Chicago	Illinois
Hackensack University Medical Center	Hackensack	New Jersey
UNC Children's Research Institute	Chapel Hill	North Carolina
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
The University of Texas Medical School at Houston	Houston	Texas
University of Utah, PPDS	Salt Lake City	Utah
Sanatorio Mater Dei	Buenos Aires	Argentina
UZ Antwerpen	Antwerp	Antwerpen
Universitair Ziekenhuis Brussel	Jette	Brussels Capital
Hospital de Clínicas de Porto Alegre (HCPA) - PPDS	Porto Alegre	Brazil
University of Alberta - Faculty of Medicine & Dentistry	Edmonton	Alberta
Hospital for Sick Children	Toronto	Ontario
McGill University Health Center	Montreal	Quebec
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Hôpital Jeanne de Flandre	Lille	France
SphinCS	Höchheim	Hochheim
Medizinische Universität Lausitz - Carl Thiem	Cottbus	Germany
Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia	Udine	Italy
Erasmus Medical Center - Sophia Children's Hospital	Rotterdam	Rotterdam
Hospital Universitario Vall d'Hebron	Barcelona	Barcelona
Hospital Infantil Universitario Niño Jesus	Madrid	Madrid
Drottning Silvias Barn Och Ungdomssjukhus	Gothenburg	Sweden
Cukurova University Medical Faculty Balcali Hospital	Adana	Turkey (Türkiye)
Gazi Universitesi Tip Fakultesi	Çankaya	Turkey (Türkiye)
Great Ormond Street Hospital for Children	London	London
Birmingham Women's and Children's NHS Foundation Trust	Birmingham	United Kingdom
Royal Free Hospital	London	United Kingdom
Salford Royal Hospital	Salford	United Kingdom

On this site

📄 Elaprase (idursulfase) drug profile →

More Denali Therapeutics Inc. trials in the USA

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome Active, not recruiting

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN) Active, not recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) Active, not recruiting

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease Recruiting

Safety and Pharmacodynamic Effects of BIIB122 in Participants With LRRK2-Associated Parkinson's Disease (LRRK2-PD) Active, not recruiting

Other trials for the same condition

Long-term Follow-Up for RGX-121 Active, not recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome Active, not recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) Active, not recruiting

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects Enrolling by invitation

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 Enrolling by invitation

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05371613 on ClinicalTrials.gov ↗ ← All trials in the USA