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NCT05371613 · ClinicalTrials.gov registry record · Phase 2
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 study of Mucopolysaccharidosis II, sponsored by Denali Therapeutics.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 63
- Enrollment target
- 20
- Study locations
NCT05371613: Recruiting Phase 2 study of Mucopolysaccharidosis II, sponsored by Denali Therapeutics.
NCT05371613 is a Phase 2 study of Mucopolysaccharidosis II that is actively recruiting participants, run by Denali Therapeutics. The registered enrollment target is 63 participants, roughly in line with the 65-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target. The trial reports 20 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05371613, a Phase 2 study of Mucopolysaccharidosis II, is actively recruiting participants, sponsored by Denali Therapeutics.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 63 participants
- Enrollment target
- 20
- Study locations
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.
Conditions Studied
Interventions
- DRUG tividenofusp alfa
- DRUG idursulfase
Study Locations (20)
Other
- Sanatorio Mater Dei - Buenos Aires
- Women's and Children's Hospital - North Adelaide
- Hospital de Clínicas de Porto Alegre (HCPA) - PPDS - Porto Alegre
- Instituto Fernandes Figueira - Rio de Janeiro
- Vseobecna Fakultni Nemocnice V Praze - Prague
- Hôpital Jeanne de Flandre - Lille
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Illinois
- Ann and Robert H Lurie Children's Hospital of Chicago - Chicago
New Jersey
- Hackensack University Medical Center - Hackensack
North Carolina
- UNC Children's Research Institute - Chapel Hill
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Texas
- The University of Texas Medical School at Houston - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 63 participants |
| Start Date | 2022-07-21 |
| Est. Completion | 2027-12 |
| Phase | Phase 2 |
What NCT05371613 shows while recruiting
NCT05371613 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 63 participants, a relatively small participant target, roughly in line with the 65-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target.
The record links to 1 condition, with Mucopolysaccharidosis II appearing as the primary indexed condition, and to 2 interventions - of which tividenofusp alfa is the first listed.
NCT05371613 names 20 study sites across 15 states, led by Other, California, Illinois.
Frequently Asked Questions
What is clinical trial NCT05371613 about?
NCT05371613 is a clinical study titled "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)". 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 (MP...
What is the current status of trial NCT05371613?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 63 participants. The study started on 2022-07-21. Estimated completion is 2027-12.
What conditions does trial NCT05371613 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis II.
What interventions are being tested in trial NCT05371613?
The interventions under investigation include: tividenofusp alfa (DRUG), idursulfase (DRUG).
Who is sponsoring clinical trial NCT05371613?
This trial is sponsored by Denali Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05371613 being conducted?
This trial has 20 study locations across California, Illinois, New Jersey, North Carolina, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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