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NCT Number: NCT06772870

A Single Ascending Dose Study of 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.

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Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Nucleus Network, Level 5, 89 Commercial Road

Melbourne, Victoria, 3004, Australia

Location contact

Ofer Gronen, MD

CONTACT

[email protected]

+61 1300 715 787

Ofer Gronen, MD

CONTACT

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

DT-216P2

Drug

DT-216P2 will be administered as subcutaneous injection, subcutaneous infusion and intravenous infusion.

Saline

Drug

Normal saline solution will be used as placebo control.

Primary outcomes

  1. 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

  1. Maximum Plasma Concentration (Cmax) of DT-216P2

    Time frame: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.

  2. Time to Maximum Plasma Concentration (Tmax) of DT-216P2

    Time frame: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.

  3. Area Under the Concentration-time Curve (AUC) of DT-216P2

    Time frame: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.

Study contacts

Contact information is provided by the study sponsor or research team.

Matthias Kurth, MD, PhD

CONTACT

[email protected]

+1 619-987-7861

Uyen Nguyen, BS

CONTACT

[email protected]

+1 858-293-4902

Sponsors and collaborators

Lead sponsor

Design Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2025
Study completion
2025
First posted
Jan 14, 2025
Registry last updated
Feb 21, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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