Friedreich AtaxiaPhase 1NCT05573698
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
A Phase 1 study testing DT-216 in people with Friedreich Ataxia, run by Design Therapeutics, Inc. at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- DT-216
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Sep 2022to 25 Aug 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions.
- Able and willing to sign informed consent form prior to study enrollment.
- Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening.
Who cannot join
- Has any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study.
- Has clinically significant abnormal laboratory results.
- Has significant cardiac disease.
- Received an investigational drug within 3 months of screening.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
- Conditions
- Friedreich Ataxia
- Being tested
- DT-216, Placebo
- 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-216Drug
DT-216 will be administered by intravenous (IV) injection
PlaceboDrug
Matching Placebo will be administered by intravenous (IV) injection
| Study group | Role | What they receive |
|---|---|---|
| Multiple Dose: DT-216 | Experimental | Participants will be administered multiple doses of DT-216 Receives: Drug: DT-216 |
| Multiple Dose: DT-216 matching placebo | Placebo comparator | Participants will be administered multiple doses of placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Measure
Frequency of treatment emergent adverse events (TEAEs)
Measured: Up to approximately 60 days
Secondary outcomes (3)
Pharmacokinetic parameters
Maximum Plasma Concentration (Cmax) of DT-216
Measured: Up to approximately 60 days
Pharmacokinetic parameters
Time to Maximum Plasma Concentration (Tmax) of DT-216
Measured: Up to approximately 60 days
Pharmacokinetic parameters
Area Under the Concentration-time Curve (AUC) of DT-216
Measured: Up to approximately 60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2022
- Study start27 Sep 2022
- Primary completion25 Aug 2023
- Study completion25 Aug 2023
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
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Design Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05573698
- Sponsor's protocol ID
- DTX-216-102
- Status last verified
- Feb 2024
- First submitted
- 6 Oct 2022
- Last updated
- 29 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
- ClinicalTrials.gov ID
- NCT05573698
- Acronym / sponsor ID
- DTX-216-102
- Conditions
- Friedreich Ataxia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Design Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 27 Sep 2022 (actual)
- Primary completion
- 25 Aug 2023 (actual)
- Study completion
- 25 Aug 2023 (actual)
- First posted
- 10 Oct 2022 (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, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Multiple Dose: DT-216 | Experimental | Drug: DT-216Participants will be administered multiple doses of DT-216 |
| Multiple Dose: DT-216 matching placebo | Placebo comparator | Drug: PlaceboParticipants will be administered multiple doses of placebo |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions.
- Able and willing to sign informed consent form prior to study enrollment.
- Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening.
Key exclusion criteria (4 listed)
- Has any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study.
- Has clinically significant abnormal laboratory results.
- Has significant cardiac disease.
- Received an investigational drug within 3 months of screening.
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-216DT-216 will be administered by intravenous (IV) injection | Drug | Multiple Dose: DT-216 |
| PlaceboMatching Placebo will be administered by intravenous (IV) injection | Drug | Multiple Dose: DT-216 matching placebo |
Primary outcome
Safety Measure
Time frame: Up to approximately 60 days
Frequency of treatment emergent adverse events (TEAEs)
3 secondary outcomes
Pharmacokinetic parameters
Time frame: Up to approximately 60 days
Pharmacokinetic parameters
Time frame: Up to approximately 60 days
Pharmacokinetic parameters
Time frame: Up to approximately 60 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Feb 2024.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
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-216 in people with Friedreich Ataxia, run by Design Therapeutics, Inc. at 1 site in United States.
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 3 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: finished, as of the sponsor's last update on 29 Feb 2024. 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 United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety Measure (time frame: Up to approximately 60 days). 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 29 Feb 2024.