RecruitingPhase 2Phase 3NCT05371613

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)

A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

Sponsor

Denali Therapeutics Inc.

Enrollment

63 participants

Start Date

Jul 21, 2022

Study Type

INTERVENTIONAL

Conditions

Mucopolysaccharidosis II

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

Min Age: 2 YearsMax Age: 25 Years

Plain Language Summary

Simplified for easier understanding

This study is comparing a new enzyme therapy drug called Tividenofusp Alfa (DNL310) to the existing standard drug idursulfase in children and young adults with Hunter syndrome (Mucopolysaccharidosis Type II, or MPS II). MPS II is a rare genetic disorder where the body cannot break down certain sugars, leading to buildup that damages organs and the brain. You may be eligible if... - You are between 2 and 25 years old - You have a confirmed diagnosis of MPS II - You have either never received enzyme replacement therapy (ERT), or have been on stable ERT for at least 4 months You may NOT be eligible if... - You have a documented genetic mutation causing developmental delay unrelated to MPS II - You have previously received IDS gene therapy or stem cell therapy - You received CNS-targeted MPS ERT within the last 6 months - You have contraindications to lumbar punctures or MRI - You are currently enrolled in another investigational drug study Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGtividenofusp alfa

Intravenous repeating dose

DRUGidursulfase

Intravenous repeating dose

Locations(32)

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Ann and Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

UNC Children's Research Institute

Chapel Hill, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

The University of Texas Medical School at Houston

Houston, Texas, United States

University of Utah, PPDS

Salt Lake City, Utah, United States

Sanatorio Mater Dei

Buenos Aires, Argentina

Women's and Children's Hospital

North Adelaide, Australia

UZ Antwerpen

Antwerp, Antwerpen, Belgium

Universitair Ziekenhuis Brussel

Jette, Brussels Capital, Belgium

Hospital de Clínicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Brazil

Instituto Fernandes Figueira

Rio de Janeiro, Brazil

University of Alberta - Faculty of Medicine & Dentistry

Edmonton, Alberta, Canada

Hospital for Sick Children

Toronto, Ontario, Canada

McGill University Health Center

Montreal, Quebec, Canada

Vseobecna Fakultni Nemocnice V Praze

Prague, Czechia

Hôpital Jeanne de Flandre

Lille, France

SphinCS

Höchheim, Hochheim, Germany

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, Netherlands

Hospital Universitario Vall d'Hebron

Barcelona, Barcelona, Spain

Hospital Infantil Universitario Niño Jesus

Madrid, Madrid, Spain

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, United Kingdom

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, United Kingdom

Royal Free Hospital

London, United Kingdom

Salford Royal Hospital

Salford, United Kingdom

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NCT05371613

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