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

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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Friedreich Ataxia
• Intervention / Treatment: Drug: DT-216P2; Drug: Saline

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Uyen Nguyen, BS; Phone Number: +1 858-293-4902; Email: uyen@designtx.com
• Study Contact Backup: Name: Matthias Kurth, MD, PhD; Phone Number: +1 619-987-7861; Email: mkurth@designtx.com

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • Nucleus Network, Level 5, 89 Commercial Road
      • Contact: Ofer Gronen, MD; Phone Number: +61 1300 715 787; Email: o.gonen@nucleusnetwork.com.au
      • Contact: Ofer Gronen, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Saline; Normal saline solution will be used as placebo control.
Experimental: DT-216P2	Drug: DT-216P2; DT-216P2 will be administered as subcutaneous injection, subcutaneous infusion and intravenous infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of treatment emergent adverse events (TEAEs)	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).	From first dose to end of study, Day 30 post first dose administration.

Secondary Outcome Measures

Outcome Measure	Time Frame
Maximum Plasma Concentration (Cmax) 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.
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.

Collaborators and Investigators

• Sponsor: Design Therapeutics, Inc.
• Investigators: Principal Investigator: Ofer Gronen, MD, Nucleus Network

Study record dates

Study Major Dates

• Study Start (Estimated): February 1, 2025
• Primary Completion (Estimated): September 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: January 6, 2025
• First Submitted That Met QC Criteria: January 11, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 20, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolic Diseases; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Spinal Cord Diseases; Mitochondrial Diseases; Cerebellar Diseases; Spinocerebellar Degenerations; Friedreich Ataxia
• Other Study ID Numbers: DTX-216P2-011

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No