RECRUITINGPhase 2 / Phase 3INTERVENTIONAL
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
About This Trial
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.
Who May Be Eligible (Plain English)
Key Who May Qualify:
- 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 Who Should NOT Join This Trial:
- 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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
DRUG
tividenofusp alfa
Intravenous repeating dose
DRUG
idursulfase
Intravenous repeating dose
Locations (20)
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