Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
DT-216 · 2 trials · 1 indication
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 ...
DT-216 is an investigational small molecule being developed for the treatment of Friedreich Ataxia, a rare inherited disease that affects the nervous system and heart. It is currently in Phase 1 clinical development and has not been approved by the FDA.
DT-216 is being developed by Design Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol DSGN. The company is conducting clinical trials to evaluate the safety and efficacy of DT-216 in patients with Friedreich Ataxia.
DT-216 is in Phase 1 clinical development. Two Phase 1 trials have been completed, with a total of 71 participants enrolled. The drug is still investigational and has not received regulatory approval.
DT-216 has been studied in two completed Phase 1 trials: NCT05285540, which evaluated the drug in 39 adult patients with Friedreich Ataxia, and NCT05573698, which assessed multiple ascending doses in 32 adult patients. Both trials were conducted in the United States.
No alternative names for DT-216 have been reported. It is identified solely by its code name DT-216 in clinical trials and by the developer, Design Therapeutics.