Friedreich Ataxia
Conditions
Brief summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
Interventions
DRUGDT-216
DT-216 will be administered by intravenous (IV) injection
DRUGPlacebo
Matching Placebo will be administered by intravenous (IV) injection
Sponsors
Design Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
No
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 concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study. * Has clinically significant abnormal laboratory results. * Has significant cardiac disease. * Received an investigational drug within 3 months of screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety Measure | Up to approximately 60 days | Frequency of treatment emergent adverse events (TEAEs) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetic parameters | Up to approximately 60 days | Maximum Plasma Concentration (Cmax) of DT-216 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Pharmacodynamic parameters | Up to approximately 60 days | Frataxin expression |
Countries
United States
Outcome results
None listed