Amyloid Cardiomyopathy, Transthyretin-RelatedPhase 1NCT03294707
Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
A Phase 1 study testing AG10 oral tablet in people with Amyloid Cardiomyopathy, Transthyretin-Related, run by Eidos Therapeutics, a BridgeBio company at 1 site in United States.
Trial summary
This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyloid Cardiomyopathy, Transthyretin-Related
- What is being tested?
- AG10 oral tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Sep 2017to 18 May 2018
- 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
- Healthy volunteers accepted
- Adult
Who may be able to join
- Weight between >50 kg and ≤110 kg;
- BMI of 18 to 32 kg/m2;
- Subjects who are healthy as determined by medical history, physical examination, 12 lead ECG and standard laboratory tests;
- Subjects who are negative for drugs of abuse and alcohol tests;
- Subjects who are non-smokers;
Who cannot join
- Subjects who have used prescription drugs within 4 weeks of first dosing;
- Subjects who have a prior cholecystectomy;
- Subjects who have used any over-the-counter medications within 7 days prior to Day -1;
- Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, hematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, or connective tissue diseases or disorders;
- Subjects who have an abnormal screening ECG;
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Randomized, Placebo-controlled, Single and Multiple Ascending Dose Study of the Tolerability, Pharmacokinetics and Pharmacodynamics of AG10 in Healthy Subjects
This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
Read the detailed description
Up to 48 healthy volunteers will be given a single dose of AG10 or placebo and be monitored for safety and tolerability over a 5-day period. Up to 48 healthy volunteers will be given multiple doses of AG10 or placebo and be monitored for safety and tolerability over a 15-day period.
- Being tested
- AG10 oral tablet, Placebo Oral Tablet
- Medical subject headings
- Amyloid Neuropathies, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AG10 oral tabletDrug
Also called Eidos Therapeutics AG10
Active single ascending dose
Placebo Oral TabletDrug
Also called Matching placebo
Placebo single dose
| Study group | Role | What they receive |
|---|---|---|
| AG10 single oral dose | Experimental | AG10 oral tablet, administered by mouth, once Receives: Drug: AG10 oral tablet |
| Placebo single oral dose | Placebo comparator | Placebo Oral Tablet, administered by mouth, once Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety & tolerability: individual and summary blood pressures, heart rate, ECG and lab data presented in tabular form with descriptive statistics. Adverse events will be tabulated and summarized by Part A (SAD) vs. B (MAD), and treatment.
To evaluate the safety and tolerability of single and multiple doses of AG10 administered to healthy adult subjects
Measured: 30 days
Secondary outcomes (12)
Pharmacokinetic Assessments: T1/2
Plasma half-life (t1/2)
Measured: 30 days
Pharmacokinetic Assessments: Tmax
Time to maximum concentration (Tmax)
Measured: 30 days
Pharmacokinetic Assessments: Cmax
Maximum concentration (Cmax)
Measured: 30 days
Pharmacokinetic Assessments: Cmin
Cmin
Measured: 30 days
Pharmacokinetic Assessments: AUC
Area under the plasma concentration-time curve (AUC)
Measured: 30 days
Pharmacokinetic Assessments: Clearance
Apparent clearance (CL/F)
Measured: 30 days
Pharmacokinetic Assessments: volume of distribution
Apparent volume of distribution (Vss/F)
Measured: 30 days
Pharmacodynamic Assessments: Assessments of TTR stabilization will be listed and summarized by part, treatment, and time point using appropriate descriptive statistics.
AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays, including Fluorescent Polarization Exclusion Assay (FPE) and Immunoblotting (Western Blot) and quantitation of prealbumin (TTR).
Measured: 30 days
Pharmacodynamic Assessments: Western blot
AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: Immunoblotting (Western Blot)
Measured: 30 days
Pharmacodynamic Assessments: prealbumin
AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: quantitation of prealbumin (TTR).
Measured: 30 days
Food effect: AUC
To evaluate the effect of food on the PK of AG10. The log transformed values of total AUC will be analyzed using a linear mixed effect model with formulation, period, sequence, and carryover as fixed effects and subject as a random effect.
Measured: 30 days
Food effect: Cmax
To evaluate the effect of food on the PK of AG10. The log transformed values of Cmax will be analyzed using a linear mixed effect model with formulation, period, sequence, and carryover as fixed effects and subject as a random effect.
Measured: 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2017
- Study start11 Sep 2017
- Primary completion5 Feb 2018
- Study completion18 May 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyloid Cardiomyopathy, Transthyretin-RelatedStudies 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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Celerion
- Responsible party
- Sponsor
- Investigators
- Terry O'Reilly, M.D., Principal investigator, Celerion
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03294707
- Sponsor's protocol ID
- AG10-001
- Status last verified
- May 2018
- First submitted
- 18 Sep 2017
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
- 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 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Randomized, Placebo-controlled, Single and Multiple Ascending Dose Study of the Tolerability, Pharmacokinetics and Pharmacodynamics of AG10 in Healthy Subjects
- ClinicalTrials.gov ID
- NCT03294707
- Acronym / sponsor ID
- AG10-001
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Eidos Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Collaborators
- Celerion
- Study start
- 11 Sep 2017 (actual)
- Primary completion
- 5 Feb 2018 (actual)
- Study completion
- 18 May 2018 (actual)
- First posted
- 27 Sep 2017 (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 is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
Detailed description
Up to 48 healthy volunteers will be given a single dose of AG10 or placebo and be monitored for safety and tolerability over a 5-day period. Up to 48 healthy volunteers will be given multiple doses of AG10 or placebo and be monitored for safety and tolerability over a 15-day period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AG10 single oral dose | Experimental | Drug: AG10 oral tabletAG10 oral tablet, administered by mouth, once |
| Placebo single oral dose | Placebo comparator | Drug: Placebo Oral TabletPlacebo Oral Tablet, administered by mouth, once |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Weight between >50 kg and ≤110 kg;
- BMI of 18 to 32 kg/m2;
- Subjects who are healthy as determined by medical history, physical examination, 12 lead ECG and standard laboratory tests;
- Subjects who are negative for drugs of abuse and alcohol tests;
- Subjects who are non-smokers;
Key exclusion criteria (5 listed)
- Subjects who have used prescription drugs within 4 weeks of first dosing;
- Subjects who have a prior cholecystectomy;
- Subjects who have used any over-the-counter medications within 7 days prior to Day -1;
- Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, hematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, or connective tissue diseases or disorders;
- Subjects who have an abnormal screening ECG;
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 |
|---|---|---|
| AG10 oral tabletAlso: Eidos Therapeutics AG10Active single ascending dose | Drug | AG10 single oral dose |
| Placebo Oral TabletAlso: Matching placeboPlacebo single dose | Drug | Placebo single oral dose |
Primary outcome
Safety & tolerability: individual and summary blood pressures, heart rate, ECG and lab data presented in tabular form with descriptive statistics. Adverse events will be tabulated and summarized by Part A (SAD) vs. B (MAD), and treatment.
Time frame: 30 days
To evaluate the safety and tolerability of single and multiple doses of AG10 administered to healthy adult subjects
12 secondary outcomes
Pharmacokinetic Assessments: T1/2
Time frame: 30 days
Pharmacokinetic Assessments: Tmax
Time frame: 30 days
Pharmacokinetic Assessments: Cmax
Time frame: 30 days
Pharmacokinetic Assessments: Cmin
Time frame: 30 days
Pharmacokinetic Assessments: AUC
Time frame: 30 days
Pharmacokinetic Assessments: Clearance
Time frame: 30 days
Pharmacokinetic Assessments: volume of distribution
Time frame: 30 days
Pharmacodynamic Assessments: Assessments of TTR stabilization will be listed and summarized by part, treatment, and time point using appropriate descriptive statistics.
Time frame: 30 days
Pharmacodynamic Assessments: Western blot
Time frame: 30 days
Pharmacodynamic Assessments: prealbumin
Time frame: 30 days
Food effect: AUC
Time frame: 30 days
Food effect: Cmax
Time frame: 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 19 Sep 2024.
- 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.
Terry O'Reilly, M.D. principal investigator
Celerion
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 AG10 oral tablet in people with Amyloid Cardiomyopathy, Transthyretin-Related, run by Eidos Therapeutics, a BridgeBio company 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 5 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 19 Sep 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 & tolerability: individual and summary blood pressures, heart rate, ECG and lab data presented in tabular form with descriptive statistics. Adverse events will be tabulated and summarized by Part A (SAD) vs. B (MAD), and treatment. (time frame: 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 19 Sep 2024.