A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants

Phase 1

Not yet recruiting

• Conditions: Friedreich Ataxia
• Interventions: Drug: DT-216P2; Drug: Saline

• Registration Number: NCT06772870
• Lead Sponsor: Design Therapeutics, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-01-06
• Study First Submitted QC: 2025-01-11
• Study First Posted: 2025-01-14
• Status Verified: 2025-02
• Study Start: 2025-02
• Last Update Submitted: 2025-02-20
• Last Update Posted: 2025-02-21
• Primary Completion: 2025-09
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 36

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
DT-216P2	DT-216P2	-
Placebo	Saline	-

Primary Outcome Measures

Name	Time	Method
Frequency of treatment emergent adverse events (TEAEs)	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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Nucleus Network, Level 5, 89 Commercial Road; 🇦🇺 Melbourne, Victoria, Australia