Friedreich AtaxiaPhase 1NCT05285540
Study to Evaluate 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 intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled. 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
- 39 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Mar 2022to 19 Dec 2022
- 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
- Body mass index (BMI) between 17 and 32 kg/m2
- Stage 5.5 or less on the Functional Staging for Ataxia (FSA)
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
- Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending 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 intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.
- Conditions
- Friedreich Ataxia
- Being tested
- DT-216, DT-216 matching 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
DT-216 matching PlaceboDrug
Placebo will be administered by intravenous (IV) injection
| Study group | Role | What they receive |
|---|---|---|
| Single Dose: DT-216 | Experimental | DT-216 will be administered once Receives: Drug: DT-216 |
| Single Dose: DT-216 matching placebo | Experimental | Placebo will be administered once Receives: Drug: DT-216 matching Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of treatment adverse events (TEAEs)
Frequency of treatment emergent adverse events (TEAEs)
Measured: Up to approximately 30 days
Secondary outcomes (3)
Maximum Plasma Concentration (Cmax) of DT-216
Maximum Plasma Concentration (Cmax) of DT-216
Measured: Up to approximately 30 days
Time to Maximum Plasma Concentration (Tmax) of DT-216
Time to Maximum Plasma Concentration (Tmax) of DT-216
Measured: Up to approximately 30 days
Area Under the Concentration-time Curve (AUC) of DT-216
Area Under the Concentration-time Curve (AUC) of DT-216
Measured: Up to approximately 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2022
- Study start11 Mar 2022
- Primary completion19 Dec 2022
- Study completion19 Dec 2022
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
- 39 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
- NCT05285540
- Sponsor's protocol ID
- DTX-216-101
- Status last verified
- Mar 2023
- First submitted
- 9 Mar 2022
- Last updated
- 28 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
- ClinicalTrials.gov ID
- NCT05285540
- Acronym / sponsor ID
- DTX-216-101
- Conditions
- Friedreich Ataxia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Design Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Mar 2022 (actual)
- Primary completion
- 19 Dec 2022 (actual)
- Study completion
- 19 Dec 2022 (actual)
- First posted
- 17 Mar 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 intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.
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 |
|---|---|---|
| Single Dose: DT-216 | Experimental | Drug: DT-216DT-216 will be administered once |
| Single Dose: DT-216 matching placebo | Experimental | Drug: DT-216 matching PlaceboPlacebo will be administered once |
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
- Body mass index (BMI) between 17 and 32 kg/m2
- Stage 5.5 or less on the Functional Staging for Ataxia (FSA)
Key exclusion criteria (5 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
- Note: Other protocol defined Inclusion/Exclusion criteria may apply.
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 | Single Dose: DT-216 |
| DT-216 matching PlaceboPlacebo will be administered by intravenous (IV) injection | Drug | Single Dose: DT-216 matching placebo |
Primary outcome
Frequency of treatment adverse events (TEAEs)
Time frame: Up to approximately 30 days
Frequency of treatment emergent adverse events (TEAEs)
3 secondary outcomes
Maximum Plasma Concentration (Cmax) of DT-216
Time frame: Up to approximately 30 days
Time to Maximum Plasma Concentration (Tmax) of DT-216
Time frame: Up to approximately 30 days
Area Under the Concentration-time Curve (AUC) of DT-216
Time frame: Up to approximately 30 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 28 Mar 2023.
- 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. 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 5 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 28 Mar 2023. 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. 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?
Frequency of treatment adverse events (TEAEs) (time frame: Up to approximately 30 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 28 Mar 2023.