A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

NCT06874010 | Recruiting Phase 1 DMC

• 1 other identifier: DTX-216P2-101
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (1)

• Frequency of treatment-emergent adverse events (TEAE)

  Up to Week 12

Secondary Outcomes (5)

• Area under the curve (AUClast, AUCinf)

  Up to Week 12

• Maximum concentration (Cmax)

  Up to Week 12

• Time to Cmax (Tmax)

  Up to Week 12

• Half-life (t1/2)

  Up to Week 12

• Frataxin expression at baseline and after treatment will be measured

  Up to Week 12

Study Arms (1)

DT-216P2

EXPERIMENTAL

Drug: DT-216P2

Interventions

DT-216P2 DRUG

Active

DT-216P2

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must be 18-65 years of age inclusive, at the time of signing the informed consent.
• Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene.
• Stage 5.5 or less on the FSA at screening.
• BMI between 16 and 32 kg/m2 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.
• Received an investigational agent within the last 30 days or 5 half-lives, whichever longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment. Exception: Potential participants who are currently on Omaveloxolone must be on stable doses for at least 3 months.
• Is not willing to comply with the contraceptive requirements during the study period, as per protocol.

Sponsors & Collaborators

• Design Therapeutics, Inc.: lead

Study Sites (3)

Scientia Clinical Research Ltd

Sydney, New South Wales, 2031, Australia

RECRUITING

Doherty Clinical Trials

East Melbourne, Victoria, 3002, Australia

RECRUITING

Nucleus Network

Melbourne, Victoria, 3004, Australia

RECRUITING

MeSH Terms

Conditions

Friedreich Ataxia

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

Central Study Contacts

Varlli Scott

CONTACT

+613 8341 6228; varlli.scott@mcri.edu.au

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 25, 2025
• First Posted: March 13, 2025
• Study Start: June 1, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): March 1, 2027
• Last Updated: May 18, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3)