Testing DT-216P2 in patients with Friedreich's Ataxia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the safety, tolerability, and pharmacokinetics of multiple ascending doses of DT-216P2 in patients diagnosed with Friedreich's Ataxia (FA). Participants aged 18-65 with a confirmed genetic diagnosis of FA will receive the treatment to assess its effects. The study aims to gather data on how the drug is processed in the body and its potential therapeutic benefits for this condition.

Why it matters

Who can join: the full rules

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.

Where it's running

• Scientia Clinical Research Ltd
  Sydney, New South Wales, Australia Recruiting
• Doherty Clinical Trials
  East Melbourne, Victoria, Australia Recruiting
• Nucleus Network
  Melbourne, Victoria, Australia Recruiting

Study contacts

• Study coordinator: Varlli Scott
• Email: varlli.scott@mcri.edu.au
• Phone: +613 8341 6228

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Nomlabofusp by injection for Friedreich's Ataxia: efficacy and safety

  This study tests whether subcutaneous nomlabofusp helps adults and children with Friedreich's Ataxia compared with placebo.

  Recruiting at 3 sites Phase 3 Larimar Therapeutics, Inc. · Tampa, Florida and 3 other locations · NCT07778836

• LX2006 Gene Therapy for Friedreich Ataxia Cardiomyopathy (FA-CM)

  A Phase 2 study tests LX2006 gene therapy in people with Friedreich ataxia who also have cardiomyopathy to see if it helps heart function and safety.

  Recruiting Phase 2 Lexeo Therapeutics · Tampa, Florida · NCT07721025

• Nerve ultrasound in people with Friedreich ataxia

  This project uses routine peripheral nerve ultrasound to see if adults with genetically confirmed Friedreich ataxia have enlarged nerves.

  Recruiting Centre Hospitalier Universitaire de Nice · Nice, Alpes Maritimes · NCT07508631

• Omaveloxolone for swallowing problems in French people with Friedreich's ataxia

  This project will test whether omaveloxolone (SKYCLARYS) improves swallowing in French people aged 16 and older with genetically confirmed Friedreich's ataxia who have used the drug for at least six months.

  Recruiting Centre Hospitalier Universitaire de Nice · Nice, Alpes Maritimes · NCT07013292

• Clinical course of cardiomyopathy in people with Friedreich ataxia

  This study follows people with Friedreich ataxia who have cardiomyopathy to see how their heart disease changes over time and to identify who may later be eligible for a gene therapy study.

  Recruiting at 18 sites Lexeo Therapeutics · La Jolla, California and 18 other locations · NCT06865482

• Health effects of omaveloxolone taken during pregnancy or breastfeeding in people with Friedreich's Ataxia

  See if taking BIIB141 (omaveloxolone) during pregnancy or while breastfeeding affects the health of people with Friedreich's Ataxia and their babies.

  Recruiting Biogen · Morrisville, North Carolina · NCT06628687

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.