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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05573698 | Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia | PHASE1 | COMPLETED | 32 | — | — | Sep 27, 2022 | Aug 25, 2023 | Feb 29, 2024 | 1 | United States |
| NCT05285540 | Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia | PHASE1 | COMPLETED | 39 | — | — | Mar 11, 2022 | Dec 19, 2022 | Mar 28, 2023 | 1 | United States |
Frequency of treatment emergent adverse events (TEAEs)
Frequency of treatment emergent adverse events (TEAEs)
| Arm | Type | Description |
|---|---|---|
| Multiple Dose: DT-216 | EXPERIMENTAL | Participants will be administered multiple doses of DT-216 |
| Multiple Dose: DT-216 matching placebo | PLACEBO_COMPARATOR | Participants will be administered multiple doses of placebo |
| Single Dose: DT-216 | EXPERIMENTAL | DT-216 will be administered once |
| Single Dose: DT-216 matching placebo | EXPERIMENTAL | Placebo will be administered once |
| Name | Type | Description |
|---|---|---|
| DT-216 | DRUG | DT-216 will be administered by intravenous (IV) injection |
| Placebo | DRUG | Matching Placebo will be administered by intravenous (IV) injection |
| DT-216 matching Placebo | DRUG | Placebo will be administered by intravenous (IV) injection |
Inclusion Criteria: * Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions. * Able and willing to sign informed consent form prior to study enrollment. * Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening. Exclusion Criteria: * Has any ...