A Phase 1, Double-Masked, Randomized, Placebo-Controlled, Single Ascending Dose Crossover Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Normal Healthy Participants

Design Therapeutics, Inc.1 site in 1 country36 target enrollmentFebruary 2025

ConditionsFriedreich Ataxia

InterventionsDT-216P2 Saline

DrugsDT-216P2 Saline

Overview

Phase: Phase 1
Intervention: DT-216P2
Conditions: Friedreich Ataxia
Sponsor: Design Therapeutics, Inc.
Enrollment: 36
Locations: 1
Primary Endpoint: Frequency of treatment emergent adverse events (TEAEs)
Status: Not yet recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants. Approximately 36 participants will be enrolled into this study.

Registry

clinicaltrials.gov

Start Date

February 2025

End Date

December 2025

Last Updated

last year

Study Type

Interventional

Study Design

Crossover

Sex

All

Investigators

Sponsor

Design Therapeutics, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Participants must be 18-45 years of age inclusive, at the time of signing the informed consent.
•Participants who are overtly healthy as determined by medical evaluation including medical history, physical examination, laboratory tests, and ECG.
•Agree to abstain from strenuous physical activity outside of daily norm, until end of study.
•Body mass index between 16 and 32 kg/m2 (inclusive) 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.
•Is not willing to comply with the contraceptive requirements during the study period, as per protocol.

Arms & Interventions

DT-216P2

Intervention: DT-216P2

Placebo

Intervention: Saline

Outcomes

Primary Outcomes

Frequency of treatment emergent adverse events (TEAEs)

Time Frame: From first dose to end of study, Day 30 post first dose administration.

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).

Secondary Outcomes

Maximum Plasma Concentration (Cmax) of DT-216P2(Pre-dose and up to 240 hours post first dose for both SC and IV infusion.)
Area Under the Concentration-time Curve (AUC) of DT-216P2(Pre-dose and up to 240 hours post first dose for both SC and IV infusion.)
Time to Maximum Plasma Concentration (Tmax) of DT-216P2(Pre-dose and up to 240 hours post first dose for both SC and IV infusion.)