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DT-216P2 · 2 trials · 1 indication
To evaluate the safety and tolerability of single ascending doses of DT-216P2 in normal healthy participants by frequency of treatment-emergent adverse events (TEAEs).
| Arm | Type | Description |
|---|---|---|
| DT-216P2 | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| DT-216P2 | DRUG | Active |
| Saline | DRUG | Normal saline solution will be used as placebo control. |
Inclusion Criteria: * Participants must be 18-65 years of age inclusive, at the time of signing the informed consent. * Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene. * Stage 5.5 or less on the FSA at screening. * BMI between 16 and 32 kg/m2 at sc...
DT-216P2 is an investigational small molecule being developed for the treatment of Friedreich Ataxia, a rare inherited disease that causes progressive nervous system damage. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
DT-216P2 targets the FXN gene, which encodes the protein frataxin. In Friedreich Ataxia, mutations in FXN lead to reduced frataxin levels. By targeting FXN, DT-216P2 aims to address the underlying genetic cause of the disease.
DT-216P2 is being developed by Design Therapeutics, Inc., a biopharmaceutical company publicly traded under the ticker symbol DSGN on the NASDAQ stock exchange.
DT-216P2 is currently in Phase 1 clinical trials. It is an investigational drug still in early-stage clinical development and has not received FDA approval. Two Phase 1 studies are listed as active, with one recruiting and one not yet recruiting participants.
DT-216P2 is being studied in two Phase 1 trials. NCT06874010 is a multiple ascending dose study in patients with Friedreich Ataxia, currently recruiting in Australia. NCT06772870 is a single ascending dose study in healthy participants, also in Australia, which has not yet started recruiting.
No alternative names for DT-216P2 have been disclosed. It is identified solely by its compound designation DT-216P2 in clinical trial registries and by its developer, Design Therapeutics.