Friedreich AtaxiaPhase 1NCT06772870
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
A Phase 1 study testing DT-216P2 and Saline in people with Friedreich Ataxia, run by Design Therapeutics, Inc. at 1 site in Australia.
Trial summary
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants. Approximately 36 participants will be enrolled into this study. Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- DT-216P2 and Saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 1 siteAustralia
- Study dates
- Feb 2025to Dec 2025
- Phase
- Phase 1
- 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 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Participants must be 18-45 years of age inclusive, at the time of signing the informed consent.
- Participants who are overtly healthy as determined by medical evaluation including medical history, physical examination, laboratory tests, and ECG.
- Agree to abstain from strenuous physical activity outside of daily norm, until end of study.
- Body mass index between 16 and 32 kg/m2 (inclusive) 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.
- Is not willing to comply with the contraceptive requirements during the study period, as per protocol.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Australia1 site
Nucleus Network, Level 5, 89 Commercial Road
Melbourne, Victoria, 3004
Ofer Gronen, MD, +61 1300 715 787, o.gonen@nucleusnetwork.com.au
Ofer Gronen, MD
About this study
Official title: A Phase 1, Double-Masked, Randomized, Placebo-Controlled, Single Ascending Dose Crossover Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Normal Healthy Participants
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants. Approximately 36 participants will be enrolled into this study.
- Conditions
- Friedreich Ataxia
- Being tested
- DT-216P2, Saline
- 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
DT-216P2 will be administered as subcutaneous injection, subcutaneous infusion and intravenous infusion.
SalineDrug
Normal saline solution will be used as placebo control.
| Study group | Role | What they receive |
|---|---|---|
| DT-216P2 | Experimental | Not described Receives: Drug: DT-216P2 |
| Placebo | Placebo comparator | Not described Receives: Drug: Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of treatment emergent adverse events (TEAEs)
To evaluate the safety and tolerability of single ascending doses of DT-216P2 in normal healthy participants by frequency of treatment-emergent adverse events (TEAEs).
Measured: From first dose to end of study, Day 30 post first dose administration.
Secondary outcomes (3)
Maximum Plasma Concentration (Cmax) of DT-216P2
Measured: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
Time to Maximum Plasma Concentration (Tmax) of DT-216P2
Measured: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
Area Under the Concentration-time Curve (AUC) of DT-216P2
Measured: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2025
- Study startFeb 2025 (estimated)
- Primary completionSep 2025 (estimated)
- Study completionDec 2025 (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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Design Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ofer Gronen, MD, Principal investigator, Nucleus Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06772870
- Sponsor's protocol ID
- DTX-216P2-011
- Status last verified
- Feb 2025
- First submitted
- 6 Jan 2025
- Last updated
- 21 Feb 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 21 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Double-Masked, Randomized, Placebo-Controlled, Single Ascending Dose Crossover Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Normal Healthy Participants
- ClinicalTrials.gov ID
- NCT06772870
- Acronym / sponsor ID
- DTX-216P2-011
- Conditions
- Friedreich Ataxia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Design Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2025 (estimated)
- Primary completion
- Sep 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 14 Jan 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.
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants. Approximately 36 participants will be enrolled into this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| DT-216P2 | Experimental | Drug: DT-216P2 |
| Placebo | Placebo comparator | Drug: Saline |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Participants must be 18-45 years of age inclusive, at the time of signing the informed consent.
- Participants who are overtly healthy as determined by medical evaluation including medical history, physical examination, laboratory tests, and ECG.
- Agree to abstain from strenuous physical activity outside of daily norm, until end of study.
- Body mass index between 16 and 32 kg/m2 (inclusive) at screening; weight should be <= 100 kg at screening.
- Male and/or female using protocol defined and regulatory approved contraception.
Key exclusion criteria (3 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.
- 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-216P2DT-216P2 will be administered as subcutaneous injection, subcutaneous infusion and intravenous infusion. | Drug | DT-216P2 |
| SalineNormal saline solution will be used as placebo control. | Drug | Placebo |
Primary outcome
Frequency of treatment emergent adverse events (TEAEs)
Time frame: From first dose to end of study, Day 30 post first dose administration.
To evaluate the safety and tolerability of single ascending doses of DT-216P2 in normal healthy participants by frequency of treatment-emergent adverse events (TEAEs).
3 secondary outcomes
Maximum Plasma Concentration (Cmax) of DT-216P2
Time frame: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
Time to Maximum Plasma Concentration (Tmax) of DT-216P2
Time frame: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
Area Under the Concentration-time Curve (AUC) of DT-216P2
Time frame: Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
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 21 Feb 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.
Ofer Gronen, MD principal investigator
Nucleus Network
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing DT-216P2 and Saline in people with Friedreich Ataxia, run by Design Therapeutics, Inc. at 1 site 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 3 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: opening soon, as of the sponsor's last update on 21 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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 Uyen Nguyen, BS (+1 858-293-4902, uyen@designtx.com), 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 (TEAEs) (time frame: From first dose to end of study, Day 30 post first dose administration.). 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 21 Feb 2025.