A Phase 1/2 interventional study of DT-216P2 in Friedreich Ataxia, sponsored by Design Therapeutics, Inc.. Recruiting at 3 sites in Australia. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2025-05-18.
Sponsored by Design Therapeutics, Inc. · Phase 1/2, Interventional, and Treatment
Among 13 studies in Friedreich Ataxia with a site in Australia — 3 open to participants now, 7 under way, 2 of them phase 3. Friedreich Ataxia · Australia.
Among the 13 studies in Friedreich Ataxia with a site in Australia, this one is one of 4 that started in 2025, is one of 3 in phase 1/2, and was the 12th registered.
110 studies on the registry are indexed under Friedreich Ataxia; 27 are open to participants now.
Browse Friedreich Ataxia studies →3 of the 13 studies in Friedreich Ataxia with a site in Australia are open now. Recruiting first, the newest first:
Friedreich's Ataxia Research Alliance · Sites in Australia, United States, Austria and 14 more countries
Design Therapeutics, Inc. · Sites in Australia
A study can be open while its sites in Australia have not opened yet or have stopped. Only those recruiting at a site in Australia →
Of the 18 Friedreich Ataxia studies recruiting now, 9 (50%) set no upper age limit and 9 (50%) stop at 75 or younger; 15 take children under 18; 3 take healthy volunteers.
This study: 18 Years to 65 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
13 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 4 · Phase 2 6 · Phase 3 3
First in 2001; this study’s year is marked. 4 started in 2025.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 20 is below the median of 30 across 75 interventional studies indexed under Friedreich Ataxia.
Median enrollment 80 across the 9 interventional studies, 1,150 across the 4 observational. Of the 5 that completed or were terminated, 2 (40%) have results posted.
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7 of 13 have an industry lead sponsor. The 7 most frequent:
Design Therapeutics, Inc. is the lead sponsor of 5 studies on the registry; 3 are open to participants now.
Design Therapeutics, Inc. has 4 studies filed under Friedreich Ataxia — fewer than ten, so there is no comparison to draw.
2 of the studies in Friedreich Ataxia with a site in Australia that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06953583Phase 3BiogenA Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia | 2027-11-16 |
| NCT06874010Design Therapeutics, Inc.A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia | 2026-12 |
Among the 13 studies in Friedreich Ataxia with a site in Australia, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: DT-216P2
Active
Frequency of treatment-emergent adverse events (TEAE)
Time frame: Up to Week 12
Area under the curve (AUClast, AUCinf)
Time frame: Up to Week 12
Maximum concentration (Cmax)
Time frame: Up to Week 12
Time to Cmax (Tmax)
Time frame: Up to Week 12
Half-life (t1/2)
Time frame: Up to Week 12
Frataxin expression at baseline and after treatment will be measured
Time frame: Up to Week 12
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Jun 2025; primary completion for Dec 2026, 18 months later.
How these figures are worked out, and which studies are left out of them.
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Design Therapeutics, Inc.