Trial · NCT05371613
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)
NCT05371613, Phase 2, Phase 3, Active not recruiting, Denali Therapeutics Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. 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). Trials, record NCT05371613. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05371613
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
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)
- NCT number
- NCT05371613
- Status
- Active not recruiting
- Phases
- Phase 2, Phase 3
- Enrollment
- 65 participants, actual
- Lead sponsor
- Denali Therapeutics Inc.
- Conditions
- Mucopolysaccharidosis II
- Interventions
- tividenofusp alfa, idursulfase
- First posted
- 2022-05-12
- Last updated
- 2026-09-09
- Other registries' IDs
- eudract:2021-005200-35
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Ratio to baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A) | 24 weeks | |
| Primary | Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A) | 96 weeks | |
| Secondary | Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only) | 96 weeks | |
| Secondary | Change from baseline in the Vineland-3 Adaptive Behavior Composite (ABC) score (Cohort A only) | 96 weeks | |
| Secondary | Ratio to baseline in serum neurofilament light chain (NfL) (Cohort A only) | 96 weeks | |
| Secondary | Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only) | 48 weeks | |
| Secondary | Ratio to baseline in the sum of urine HS and dermatan sulfate (DS) concentrations (normalized to creatinine) (Cohorts A and B) | up to 48 weeks | |
| Secondary | Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) (Cohorts A and B) | 48 weeks | |
| Secondary | Spleen volume within the normal range (normal vs abnormal) as measured by MRI (Cohorts A and B) | 48 weeks | |
| Secondary | Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B) | 48 weeks |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05371613
GET https://conductscience.org/api/trials/v1/NCT05371613/registered