Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | PHASE2 | ENROLLING BY_INVITATION | 99 | — | — | Sep 20, 2023 | Jun 1, 2027 | May 12, 2026 | 27 | United States, Argentina +11 |
| 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) | PHASE2 | RECRUITING | 63 | — | — | Jul 21, 2022 | Dec 1, 2027 | Aug 5, 2025 | 32 | United States, Argentina +13 |
| NCT04251026 | A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome | PHASE1 | ACTIVE NOT_RECRUITING | 47 | — | — | Jul 16, 2020 | Feb 1, 2031 | Aug 7, 2025 | 7 | United States, Canada +2 |
The intensity of IRRs will be assessed following each infusion of DNL310 using the categories of Mild, Moderate and Severe. IRRs will be summarized overall as well as stratified by intensity.
| Arm | Type | Description |
|---|---|---|
| Cohort A2 | EXPERIMENTAL | Participants with nMPS II, aged ≥5 to ≤10 years |
| Cohort B2 | EXPERIMENTAL | Participants with nMPS II or nnMPS II, aged ≥1 to ≤18 years |
| Cohort C2 | EXPERIMENTAL | Participants with nMPS II, aged \<4 years |
| Cohort D2 | EXPERIMENTAL | Participants with nMPS II or nnMPS II, aged ≤18 years with preexisting hepatomegaly who have never taken standard-of-care ERT |
| Cohort E2 | EXPERIMENTAL | Participants with nMPS II, aged ≥6 years; participants with nnMPS II, aged \<6 or ≥17 years; or participants with nMPS II, aged ≥1 to ≤18 years, with a history of prior HSCT or gene therapy and have completed at least 48 weeks in Study DNLI-E-0001 |
| Cohort A7 | EXPERIMENTAL | Participants with nMPS II, aged ≥2 to \<6 years |
| Cohort B7 | EXPERIMENTAL | Participants with nnMPS II, aged ≥6 to \<17 years |
| Cohort A: Participants with nMPS II | EXPERIMENTAL | - |
| Cohort B: Participants with nnMPS II | EXPERIMENTAL | - |
| Open-label Treatment Phase | EXPERIMENTAL | Participants who meet pre-specified criteria may receive DNL310 or idursulfase |
| Cohort A | EXPERIMENTAL | Dose escalation followed by a consistent dose level in participants with neuronopathic MPS II |
| Cohort B | EXPERIMENTAL | A consistent dose level in participants with non-neuronopathic MPS II, neuronopathic MPS II, or unknown phenotype followed by dose escalation in some participants. |
| Cohort C | EXPERIMENTAL | A consistent dose level in participants with neuronopathic MPS II |
| Cohort D | EXPERIMENTAL | A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II |
| Cohort E | EXPERIMENTAL | A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II |
| Name | Type | Description |
|---|---|---|
| tividenofusp alfa | DRUG | Intravenous repeating dose |
| idursulfase | DRUG | Intravenous repeating dose |
Key Inclusion Criteria: * For participants from Study DNLI-E-0002 only: Completed at least through the Week 49 visit in Study DNLI-E-0002 and did not discontinue study intervention early * For participants from Study DNLI-E-0007 only: Completed the treatment period of 96 weeks in Cohort A for nMPS ...