Clinical Trials Logo
  1. Home
  2. Mucopolysaccharidosis II
  3. NCT05371613

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

Mucopolysaccharidosis II Clinical Trial

Official title:
A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05371613
Study type Interventional
Source Denali Therapeutics Inc.
Contact Clinical Trials at Denali Therapeutics
Email clinical-trials@dnli.com
Status Recruiting
Phase Phase 2/Phase 3
Start date July 21, 2022
Completion date December 2025
View Details
See also
  Status Clinical Trial Phase
Withdrawn NCT05238324 - Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Phase 1
Completed NCT03529786 - Mucopolysaccharidosis Type II Natural History
Recruiting NCT02254863 - UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Phase 1
Terminated NCT01675674 - Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics N/A
Enrolling by invitation NCT06075537 - 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 Phase 2/Phase 3
Recruiting NCT05422482 - A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ Phase 1
Completed NCT00069641 - Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Phase 2/Phase 3
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Active, not recruiting NCT04348136 - An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II Phase 2/Phase 3
Completed NCT04007536 - A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
Completed NCT00004454 - Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) Phase 1/Phase 2
Active, not recruiting NCT04628871 - Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Terminated NCT00748969 - Clinical Trial of Growth Hormone in MPS I, II, and VI Phase 2/Phase 3
Recruiting NCT05619900 - Registry of Patients Diagnosed With Lysosomal Storage Diseases
Completed NCT01301898 - To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients Phase 1/Phase 2
Terminated NCT03041324 - Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II Phase 1/Phase 2
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed NCT03128593 - A Study of JR-141 in Patients With Mucopolysaccharidosis Type II Phase 1/Phase 2
Withdrawn NCT04591834 - Mucopolysaccharidosis Type II Observational
Enrolling by invitation NCT04597385 - Long-term Follow-Up for RGX-121