Friedreich Ataxia
Conditions
Brief summary
The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
Interventions
DRUGDT-216P2
Active
Sponsors
Design Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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 screening; weight should be \<= 100 kg at screening. * Male and/or female using protocol defined and regulatory approved contraception. * Capable of giving signed informed consent.
Exclusion criteria
* Any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study protocol. * Any clinically significant nonmedical conditions and psychiatric disorders that could put the participant at higher risk for participation in the study, influence the participant's ability to participate in the study, or interfere with interpretation of the participant's study results, in the opinion of the investigator. * Received an investigational agent within the last 30 days or 5 half-lives, whichever longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment. Exception: Potential participants who are currently on Omaveloxolone must be on stable doses for at least 3 months. * Is not willing to comply with the contraceptive requirements during the study period, as per protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency of treatment-emergent adverse events (TEAE) | Up to Week 12 |
Secondary
| Measure | Time frame |
|---|---|
| Area under the curve (AUClast, AUCinf) | Up to Week 12 |
| Maximum concentration (Cmax) | Up to Week 12 |
| Time to Cmax (Tmax) | Up to Week 12 |
| Half-life (t1/2) | Up to Week 12 |
| Frataxin expression at baseline and after treatment will be measured | Up to Week 12 |
Countries
Australia
Contacts
Primary ContactVarlli Scott
Outcome results
None listed