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

NCT ID: NCT06772870

Last Updated: 2025-02-21

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-02-28
• Study Completion Date: 2025-12-31

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.

Conditions

Friedreich Ataxia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

DT-216P2

Group Type: EXPERIMENTAL

DT-216P2

Intervention Type: DRUG

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

Placebo

Group Type: PLACEBO_COMPARATOR

Saline

Intervention Type: DRUG

Normal saline solution will be used as placebo control.

Interventions

DT-216P2

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

Intervention Type: DRUG

Saline

Normal saline solution will be used as placebo control.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Design Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ofer Gronen, MD

Role: PRINCIPAL_INVESTIGATOR

Nucleus Network

Locations

Nucleus Network, Level 5, 89 Commercial Road

Melbourne, Victoria, Australia

Countries

Australia

Central Contacts

Uyen Nguyen, BS

Role: CONTACT

uyen@designtx.com

+1 858-293-4902

Matthias Kurth, MD, PhD

Role: CONTACT

mkurth@designtx.com

+1 619-987-7861

Facility Contacts

Ofer Gronen, MD

Role: primary

o.gonen@nucleusnetwork.com.au

+61 1300 715 787

Ofer Gronen, MD

Role: backup

Other Identifiers

DTX-216P2-011

Identifier Type: -

Identifier Source: org_study_id