Friedreich AtaxiaPhase 1NCT07869654
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
A Phase 1 study testing ATI-105 in people with Friedreich Ataxia and Cardiomyopathies, run by Adjuvia Therapeutics, Inc. at 1 site in United States.
Trial summary
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food. The outcomes of this clinical trial will help to determine if ATI-105 may be able to treat patients with Friedreich's ataxia, a rare and life-threatening… Read more about this study
- Who is it for?
- Ages 18 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases
- What is being tested?
- ATI-105
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Oct 2026to 27 Jun 2027
- 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 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Willing and able to provide written informed consent and comply with scheduled visits, laboratory tests, and other study procedures.
- Males and females 18-60 years of age, inclusive.
- Medically healthy by history, physical examination, vital signs, 12-lead electrocardiogram (ECG), and clinical laboratory evaluations (hematology, serum chemistry, coagulation, urinalysis).
- Body mass index (BMI) of 18.0-35.0 kg/m2, inclusive.
- Able to swallow capsules.
- Males and females of childbearing potential must be willing to use an effective method of contraception (hormonal or double barrier method of birth control; abstinence) from the time consent is signed until 37 days after treatment discontinuation (i.e., 30 days + 5 half-lives of ATI-105).
Who cannot join
- 1. Any clinically significant disease or condition which, in the opinion of the Investigator, could affect participant safety or confound study results.
- 2. Pregnant or lactating subjects. 3. Use of prescription/over-the-counter (OTC) medications, herbal products, or supplements within 7 days or 5 half-lives (whichever is longer) prior to dosing, or current use of any investigational medication. Thyroid medications with a stable dose for 1 year are acceptable.
- 4. Use of glucagon-like peptide-1 (GLP-1) agonists. 5. Positive test for drugs of abuse or alcohol. 6. Positive test for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV), per institutional laboratory test.
- 7. Positive test for influenza or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), positive test for SARS-CoV-2 within 30 days prior to screening, or current long COVID.
- 8. Laboratory abnormalities including but not limited to:
- Hemoglobin <10 g/dL
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- White blood cells <3.0 × 103/μL
- Neutrophils ≤1500/μL
- Platelet count <150 × 109/L
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) above the upper limit of normal (ULN)
- Total bilirubin >1.2 mg/dL
- Estimated glomerular filtration rate (eGFR) <90 mL/min/1.73 m2
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase 1 Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food. The outcomes of this clinical trial will help to determine if ATI-105 may be able to treat patients with Friedreich's ataxia, a rare and life-threatening nerve-muscle disease that worsens over time. The main questions this study is designed to answer include the following:
- How do single doses or doses for 14 days work in the body?
- Are these doses safe and tolerated?
- Should ATI-105 be taken with or without food?
Read more
Some participants will receive a placebo, which is an inactive substance, so the drug can be compared against it. This will help researchers determine if the drug is tolerated by participants, if there are adverse effects, and how the drug acts in the body and if there are any changes.
The study will include some participants who will be assigned by chance to receive an inactive substance (placebo) that appears like the active drug product. Qualifying participants who enroll into the first of two study portions will randomly be assigned to one of three dose groups to receive a single dose of ATI-105 or a placebo. Some of the participants will receive a second dose to inform whether ATI-105 should be taken with or without food. Qualifying participants who enroll into the second study portion will receive ATI-105 or placebo for 14 days and will be randomly assigned to one of three dosing groups.
For both portions, a safety committee will review the safety and tolerability data for each dosing group to determine if the dose can be increased for the next dosing group.
Participants will be required to stay over night in the clinic for a few days and come back for visits after for procedures that include a check of their vitals and blood draws.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases, Mitochondrial Diseases, Mitochondrial Dysfunction
- Being tested
- ATI-105, Placebo
- Keywords
- FRDA, FA, Friedreich's, neuromuscular, mitochondria, oxidative stress, astaxanthin, ATI-105, nanoemulsion, Haematococcus pluvialis algae, HP algae, antioxidant, frataxin
- Medical subject headings
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases, Mitochondrial Diseases, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATI-105Drug
ATI-105 is manufactured as an oral capsule containing astaxanthin, botanical drug product
PlaceboDrug
ATI-105 matching placebo capsule
| Study group | Role | What they receive |
|---|---|---|
| ATI-105 | Experimental | ATI-105 oral single or multiple ascending doses Receives: Drug: ATI-105 |
| Placebo control | Placebo comparator | Matching placebo control capsule Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reporting of treatment-emergent adverse events and serious adverse events
incidence and severity of adverse events
Measured: 8-21 days
Secondary outcomes (8)
Change from baseline to end of study of pharmacokinetic parameter Maximum observed plasma drug Concentration (Cmax)
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Time to maximum observed plasma drug concentration (Tmax)
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Area under the plasma-drug concentration-time curve (AUC) from time 0 to the last quantifiable concentration (AUC0-t)
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter AUC extrapolated to infinity
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter AUC over the dosing interval (tau)
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Apparent terminal phase elimination rate constant
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Apparent plasma clearance
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Apparent volume of distribution at the terminal phase, if appropriate
Measured: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2026
- Study start26 Oct 2026 (estimated)
- Primary completion27 May 2027 (estimated)
- Study completion27 Jun 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Placebo-controlled, dose-escalation, fed/fasted
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Adjuvia Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Quotient Sciences
- Responsible party
- Sponsor
- Investigators
- Nester Quezada, MD, Principal investigator, Quotient Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07869654
- Sponsor's protocol ID
- ATI-105-101
- Status last verified
- Oct 2026
- First submitted
- 6 Oct 2026
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Principal Investigator
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
- ClinicalTrials.gov ID
- NCT07869654
- Acronym / sponsor ID
- ATI-105-101
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases, Mitochondrial Diseases, Mitochondrial Dysfunction
- Keywords
- FRDA, FA, Friedreich's, neuromuscular, mitochondria, oxidative stress, astaxanthin, ATI-105
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Adjuvia Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Quotient Sciences
- Study start
- 26 Oct 2026 (estimated)
- Primary completion
- 27 May 2027 (estimated)
- Study completion
- 27 Jun 2027 (estimated)
- First posted
- 9 Oct 2026 (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 goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food. The outcomes of this clinical trial will help to determine if ATI-105 may be able to treat patients with Friedreich's ataxia, a rare and life-threatening nerve-muscle disease that worsens over time. The main questions this study is designed to answer include the following:
- How do single doses or doses for 14 days work in the body?
- Are these doses safe and tolerated?
- Should ATI-105 be taken with or without food?
Some participants will receive a placebo, which is an inactive substance, so the drug can be compared against it. This will help researchers determine if the drug is tolerated by participants, if there are adverse effects, and how the drug acts in the body and if there are any changes.
The study will include some participants who will be assigned by chance to receive an inactive substance (placebo) that appears like the active drug product. Qualifying participants who enroll into the first of two study portions will randomly be assigned to one of three dose groups to receive a single dose of ATI-105 or a placebo. Some of the participants will receive a second dose to inform whether ATI-105 should be taken with or without food. Qualifying participants who enroll into the second study portion will receive ATI-105 or placebo for 14 days and will be randomly assigned to one of three dosing groups.
For both portions, a safety committee will review the safety and tolerability data for each dosing group to determine if the dose can be increased for the next dosing group.
Participants will be required to stay over night in the clinic for a few days and come back for visits after for procedures that include a check of their vitals and blood draws.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Placebo-controlled, dose-escalation, fed/fasted
| Arm | Type | Receives |
|---|---|---|
| ATI-105 | Experimental | Drug: ATI-105ATI-105 oral single or multiple ascending doses |
| Placebo control | Placebo comparator | Drug: PlaceboMatching placebo control capsule |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Willing and able to provide written informed consent and comply with scheduled visits, laboratory tests, and other study procedures.
- Males and females 18-60 years of age, inclusive.
- Medically healthy by history, physical examination, vital signs, 12-lead electrocardiogram (ECG), and clinical laboratory evaluations (hematology, serum chemistry, coagulation, urinalysis).
- Body mass index (BMI) of 18.0-35.0 kg/m2, inclusive.
- Able to swallow capsules.
Key exclusion criteria (12 listed)
- 1. Any clinically significant disease or condition which, in the opinion of the Investigator, could affect participant safety or confound study results.
- 2. Pregnant or lactating subjects. 3. Use of prescription/over-the-counter (OTC) medications, herbal products, or supplements within 7 days or 5 half-lives (whichever is longer) prior to dosing, or current use of any investigational medication. Thyroid medications with a stable dose for 1 year are acceptable.
- 4. Use of glucagon-like peptide-1 (GLP-1) agonists. 5. Positive test for drugs of abuse or alcohol. 6. Positive test for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV), per institutional laboratory test.
- 7. Positive test for influenza or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), positive test for SARS-CoV-2 within 30 days prior to screening, or current long COVID.
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 |
|---|---|---|
| ATI-105ATI-105 is manufactured as an oral capsule containing astaxanthin, botanical drug product | Drug | ATI-105 |
| PlaceboATI-105 matching placebo capsule | Drug | Placebo control |
Primary outcome
Reporting of treatment-emergent adverse events and serious adverse events
Time frame: 8-21 days
incidence and severity of adverse events
8 secondary outcomes
Change from baseline to end of study of pharmacokinetic parameter Maximum observed plasma drug Concentration (Cmax)
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Time to maximum observed plasma drug concentration (Tmax)
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Area under the plasma-drug concentration-time curve (AUC) from time 0 to the last quantifiable concentration (AUC0-t)
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter AUC extrapolated to infinity
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter AUC over the dosing interval (tau)
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Apparent terminal phase elimination rate constant
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Apparent plasma clearance
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
Change from baseline to end of study of pharmacokinetic parameter Apparent volume of distribution at the terminal phase, if appropriate
Time frame: Multiple on Days 1-7 or Days 1-2 and Days 14-20
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 9 Oct 2026.
- Individual participant dataSharing planned: Yes. Principal Investigator
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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.
Nester Quezada, MD principal investigator
Quotient Sciences
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 ATI-105 in people with Friedreich Ataxia and Cardiomyopathies, run by Adjuvia 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 6 inclusion and 12 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 9 Oct 2026. 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?
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 Laura Gonzalez (305-644-9903, laura.gonzalez@quotientsciences.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reporting of treatment-emergent adverse events and serious adverse events (time frame: 8-21 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 9 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.