ClinicalTrials.gov record NCT06874010

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Patients With Friedreich's Ataxia

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).
Age18 years old to 65 years old
SexAll
LocationAustralia
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 1/2 open-label study gives increasing doses of DT-216P2 to people with Friedreich Ataxia to evaluate safety, tolerability, and how the drug is handled in the body. Doses are given by subcutaneous and intravenous routes in sequential cohorts. Participants are monitored for adverse events and blood drug levels, with key measurements collected up to 12 weeks.

What it is studying. The study is testing the safety, tolerability, and pharmacokinetics of multiple ascending subcutaneous and intravenous doses of DT-216P2 in people with Friedreich Ataxia, and will measure frataxin expression before and after treatment.

What the researchers hope to learn. To assess safety and tolerability of DT-216P2, define pharmacokinetic parameters (AUC, Cmax, Tmax, half-life), and measure frataxin expression before and after dosing.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • DT-216P2

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

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.
  • 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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive multiple ascending doses of DT-216P2 administered subcutaneously and/or intravenously. They will attend clinic visits for safety monitoring and pharmacokinetic testing, including blood samples and measurements of frataxin expression, with assessments up to Week 12. Study staff will track and report any treatment-emergent adverse events. Participants must follow the study's contraception requirements during the study period.

How the study is structured. An open-label, interventional Phase 1/2 dose-escalation study. Participants receive DT-216P2 (no placebo); dose levels increase sequentially across cohorts. The study collects safety data and blood samples for drug levels and frataxin measurements, with assessments reported up to Week 12.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in Australia. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
June 2025 (estimated)
Primary completion
December 2026 (estimated)
Study completion
March 2027 (estimated)
Target enrollment
20 participants (estimated)
Last updated on registry
May 18, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Design Therapeutics, Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (3)

Terms used on this page · explanations by MedicalUpdates.ai

pharmacokinetics
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
investigational agent
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
5 half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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