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

NCT06772870 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: DTX-216P2-011
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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.

Study Arms (2)

DT-216P2

EXPERIMENTAL

Drug: DT-216P2

Placebo

PLACEBO COMPARATOR

Drug: Saline

Interventions

DT-216P2 DRUG

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

DT-216P2

Saline DRUG

Normal saline solution will be used as placebo control.

Placebo

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Design Therapeutics, Inc.: lead

Study Sites (1)

Nucleus Network, Level 5, 89 Commercial Road

Melbourne, Victoria, 3004, Australia

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Ofer Gronen, MD

  Nucleus Network

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Uyen Nguyen, BS

CONTACT

+1 858-293-4902; uyen@designtx.com

Matthias Kurth, MD, PhD

CONTACT

+1 619-987-7861; mkurth@designtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2025
• First Posted: January 14, 2025
• Study Start: February 1, 2025
• Primary Completion: September 1, 2025
• Study Completion: December 1, 2025
• Last Updated: February 21, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)