Last updated 9 days ago
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)
54 patients around the world
Available in Spain, United States, Argentina
Denali Therapeutics Inc.
1Research sites
54Patients around the world
This study is for people with
Rare diseases
Mucopolysaccharidoses
Requirements for the patient
To 26 Years
All Gender
Medical requirements
- Participants aged ≥2 to <6 years (Cohort A) or ≥6 to <17 years (Cohort B)Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)Be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase for a minimum of 4 months prior to screening
- Have a documented mutation of other genes or genetic diagnosis accounting for developmental delayPreviously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapyReceived any CNS-targeted MPS ERT within 6 months prior to screeningHave a contraindication for lumbar punctures and/or magnetic resonance imagings (MRIs)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
Sites
Hospital Italiano de Buenos Aires - CABA, Buenos Aires
Recruiting
StudyCOMPASS
SponsorDenali Therapeutics Inc.
Study typeInterventional
Conditions
Mucopolysaccharidoses
Requirements
To 26 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT05371613
