Friedreich AtaxiaPhase 1/2NCT06874010
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
A Phase 1/2 study testing DT-216P2 in people with Friedreich Ataxia, run by Design Therapeutics, Inc. at 3 sites in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- DT-216P2
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesAustralia
- Study dates
- Jun 2025to Mar 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Australia3 sites, 3 recruiting
- Recruiting
Doherty Clinical Trials
East Melbourne, Victoria, 3002
Varlli Scott, +613 8341 6228, Varlli.scott@mcri.edu.au
- Recruiting
Nucleus Network
Melbourne, Victoria, 3004
Varlli Scott, +613 8341 6228, Varlli.scott@mcri.edu.au
- Recruiting
Scientia Clinical Research Ltd
Sydney, New South Wales, 2031
Varlli Scott, +613 8341 6228, Varlli.scott@mcri.edu.au
About this study
Official title: 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
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
- Being tested
- DT-216P2
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DT-216P2Drug
Active
| Study group | Role | What they receive |
|---|---|---|
| DT-216P2 | Experimental | Not described Receives: Drug: DT-216P2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of treatment-emergent adverse events (TEAE)
Measured: Up to Week 12
Secondary outcomes (5)
Area under the curve (AUClast, AUCinf)
Measured: Up to Week 12
Maximum concentration (Cmax)
Measured: Up to Week 12
Time to Cmax (Tmax)
Measured: Up to Week 12
Half-life (t1/2)
Measured: Up to Week 12
Frataxin expression at baseline and after treatment will be measured
Measured: Up to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2025
- Study startJun 2025 (estimated)
- Primary completionDec 2026 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Design Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06874010
- Sponsor's protocol ID
- DTX-216P2-101
- Status last verified
- May 2025
- First submitted
- 25 Feb 2025
- Last updated
- 18 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06874010
- Acronym / sponsor ID
- DTX-216P2-101
- Conditions
- Friedreich Ataxia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Design Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2025 (estimated)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 13 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| DT-216P2 | Experimental | Drug: DT-216P2 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DT-216P2Active | Drug | DT-216P2 |
Primary outcome
Frequency of treatment-emergent adverse events (TEAE)
Time frame: Up to Week 12
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing DT-216P2 in people with Friedreich Ataxia, run by Design Therapeutics, Inc. at 3 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Varlli Scott (+613 8341 6228, varlli.scott@mcri.edu.au), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of treatment-emergent adverse events (TEAE) (time frame: Up to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2025.