Health Condition 1: G118- Other hereditary ataxias
Conditions
Interventions
Intervention1: DT-216P2 Drug Product (DT-216 for Injection): Increasing doses will be administered weekly intravenously (into a vein) or subcutaneously (under the skin) for up to 6 months.
Control Int
Sponsors
Design Therapeutics, Inc
Eligibility
Inclusion criteria
Inclusion criteria: Participants must be 18 to 65 years of age inclusive, at the at the time of signing the informed consent Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansion in the frataxin gene Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening Body mass index (BMI)35 kg/m2 at screening If of child-bearing potential, must agree to defined contraception for 30 days (female) or 90 days (male) Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form and, in the protocol
Exclusion criteria
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. 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 (TEAEs)Timepoint: Throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| PK parameters: o Area under the curve (AUClast, AUCinf) o Maximum concentration (Cmax) o Time to Cmax (Tmax) o Half-life (t ) Timepoint: Days 1-36 | — |
Countries
India, United States of America
Contacts
Public ContactDr Neeraj Prasad
JSS Medical Research Asia Pacific Pvt. Ltd.
Outcome results
None listed