NCT03458130 is a Phase 2 clinical trial of AG10 in Familial ATTR-CM (ATTRm-CM, or FAC), sponsored by Eidos Therapeutics, a BridgeBio company.

Who is sponsoring NCT03458130?

NCT03458130 is sponsored by Eidos Therapeutics, a BridgeBio company.

What drugs are being tested in NCT03458130?

Interventions in NCT03458130: AG10, Placebo Oral Tablet.

What condition does NCT03458130 study?

NCT03458130 studies Familial ATTR-CM (ATTRm-CM, or FAC), Wild-type ATTR-CM (ATTRwt-CM).

Is NCT03458130 still recruiting?

NCT03458130 is currently completed. Last updated 16 November 2022.

Are results published for NCT03458130?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2022-11-16 · How we verify

NCT03458130

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Study of AG10 in Amyloid Cardiomyopathy

Completed Phase 2 Results posted Last updated 16 November 2022

What this trial tests

Phase 2 trial testing AG10 in Familial ATTR-CM (ATTRm-CM, or FAC) in 49 participants. Completed in 5 October 2018.

Timeline

27 April 2018

Primary endpoint
5 October 2018

5 October 2018

Quick facts

Lead sponsor	Eidos Therapeutics, a BridgeBio company
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	49
Start date	27 April 2018
Primary completion	5 October 2018
Estimated completion	5 October 2018
Sites	13 locations across United States

Drugs / interventions tested

AG10
Placebo Oral Tablet — full drug profile →

Conditions studied

Familial ATTR-CM (ATTRm-CM, or FAC) — all drugs for Familial ATTR-CM (ATTRm-CM, or FAC) →
Wild-type ATTR-CM (ATTRwt-CM) — all drugs for Wild-type ATTR-CM (ATTRwt-CM) →

Sponsor

Eidos Therapeutics, a BridgeBio company — full company profile →

Who can join

Adults 18 to 90, any sex, with Familial ATTR-CM (ATTRm-CM, or FAC) or Wild-type ATTR-CM (ATTRwt-CM). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change in Diastolic Blood Pressure Primary · Baseline to Day 28

Change in Diastolic Blood Pressure from Baseline to Day 28 (Postdose)

Change in Diastolic Blood Pressure from Baseline to Day 28 (Predose)

Group	Value	95% CI
AG10 400mg	-3.1	± 10.06
AG10 800mg	-3.3	± 11.19
Placebo	-4.3	± 7.78

Change in Diastolic Blood Pressure from Baseline to Day 28 (Postdose)

Group	Value	95% CI
AG10 400mg	-2.6	± 9.66
AG10 800mg	0.3	± 8.54
Placebo	-4.9	± 9.17

Change in Heart Rate Primary · Baseline to Day 28

Change in Heart Rate from Baseline to Day 28 (Postdose)

Change in heart rate from Baseline to Day 28 (Predose)

Group	Value	95% CI
AG10 400mg	-2.7	± 6.88
AG10 800mg	-3.8	± 6.55
Placebo	-0.4	± 6.34

Change in heart rate from Baseline to Day 28 (Postdose)

Group	Value	95% CI
AG10 400mg	-7.3	± 9.84
AG10 800mg	-4.9	± 5.84
Placebo	-1.1	± 8.89

Change in Respiratory Rate Primary · Baseline to Day 28

Change in Respiratory Rate from Baseline to Day 28 (Postdose)

Change in Respiratory Rate from Baseline to Day 28 (Predose)

Group	Value	95% CI
AG10 400mg	-0.1	± 2.23
AG10 800mg	-0.8	± 3.12
Placebo	-0.4	± 2.35

Change in Respiratory Rate from Baseline to Day 28 (Postdose)

Group	Value	95% CI
AG10 400mg	-0.7	± 1.68
AG10 800mg	-1.1	± 3.11
Placebo	-1.1	± 2.93

Change in Temperature Primary · Baseline to Day 28

Change in Temperature from Baseline to Day 28

Change in Temperature from Baseline to Day 28 (Predose)

Group	Value	95% CI
AG10 400mg	0.17	± 0.793
AG10 800mg	0.06	± 0.316
Placebo	-0.08	± 0.506

Change in Temperature from Baseline to Day 28 (Postdose)

Group	Value	95% CI
AG10 400mg	0.22	± 0.781
AG10 800mg	-0.02	± 0.357
Placebo	0.02	± 0.194

Change in Systolic Blood Pressure Primary · Baseline to Day 28

Change in Systolic Blood Pressure from Baseline to Day 28

Change in Systolic Blood Pressure from Baseline to Day 28 (Predose)

Group	Value	95% CI
AG10 400mg	-2.7	± 12.05
AG10 800mg	-8.1	± 12.36
Placebo	-5.3	± 13.50

Change in Systolic Blood Pressure from Baseline to Day 28 (Postdose)

Group	Value	95% CI
AG10 400mg	-2.8	± 18.41
AG10 800mg	-4.1	± 8.88
Placebo	-3.4	± 17.51

Number of Participants With Threshold Levels of Overall % Stabilization >= 95% and >= 99% by Fluorescent Probe Exclusion (FPE) Secondary · Day 1 to Day 28

In specialized lab tests on patient samples that measure the stability of the healthy form of TTR, both doses of AG10 were able to reach near complete stabilization.

% Stabilization > = 95 %

Group	Value	95% CI
AG10 400mg	14
AG10 800mg	15
Placebo	0

% Stabilization > = 99 %

Group	Value	95% CI
AG10 400mg	11
AG10 800mg	13
Placebo	0

Pharmacokinetic (PK): Steady State Trough Concentration of AG10 Secondary · Day 14 and Day 28

Non-fluctuating minimal amount of AG10 in blood at Day 14 and Day 28

AG10: PK Plasma Concentration at Day 14 (Pre-Dose)

Group	Value	95% CI
AG10 400mg	1924.4	± 837.3
AG10 800mg	2257.3	± 982.81

AG10: PK Plasma Concentration at Day 28 (Pre-Dose)

Group	Value	95% CI
AG10 400mg	1841.3	± 483.88
AG10 800mg	2439.4	± 900.08

Adverse events — posted to ClinicalTrials.gov

Time frame: Onset date on or after first dose of study drug and not more than 30 days after last dose of study drug, up to day 58.. Reporting threshold: 4%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

AG10 400mg

Serious: 1/16 (6%)

Deaths: 0/16

AG10 800mg

Serious: 0/16 (0%)

Deaths: 0/16

Placebo

Serious: 2/17 (12%)

Deaths: 0/17

Serious adverse events (4 terms)

Reaction	System	AG10 400mg	AG10 800mg	Placebo
Atrial Fibrillation	Cardiac disorders	—	—	—
Cardiac Failure Congestive	Cardiac disorders	—	—	—
Cellulitis	Infections and infestations	—	—	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—	—

Other adverse events (21 terms — click to expand)

Reaction	System	AG10 400mg	AG10 800mg	Placebo
Constipation	Gastrointestinal disorders	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—
Palpitations	Cardiac disorders	—	—	—
Muscle Spasms	Musculoskeletal and connective tissue disorders	—	—	—
Atrial Fibrillation	Cardiac disorders	—	—	—
Headache	Nervous system disorders	—	—	—
Fatigue	General disorders	—	—	—
Fluid Retention	Metabolism and nutrition disorders	—	—	—
Pain in Extremity	Musculoskeletal and connective tissue disorders	—	—	—
Contusion	Injury, poisoning and procedural complications	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—
Dizziness	Nervous system disorders	—	—	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—
Oedema Peripheral	General disorders	—	—	—
Pollakiuria	Renal and urinary disorders	—	—	—
Venous Pressure Jugular Increased	Investigations	—	—	—
Fluid Overload	Metabolism and nutrition disorders	—	—	—
Abdominal Pain	Gastrointestinal disorders	—	—	—
Conjunctivitis	Infections and infestations	—	—	—
Groin Pain	Musculoskeletal and connective tissue disorders	—	—	—

Most-reported serious reactions: Atrial Fibrillation, Cardiac Failure Congestive, Cellulitis, Dyspnoea.

Data from ClinicalTrials.gov NCT03458130 adverse events section.

Sponsor's own description

This prospective, randomized, multicenter, double-blind, parallel group, placebo-controlled, dose-ranging study will evaluate the safety, tolerability, PK (Pharmacokinetic) and PD (Pharmacodynamic) of AG10 compared to placebo administered on a background of stable heart failure therapy. Screening and randomization will be followed by a 28-day blinded, placebo-controlled treatment period.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy.
Judge DP, Heitner SB, Falk RH, Maurer MS, et al · · 2019 · cited 182× · PMID 30885685 · DOI 10.1016/j.jacc.2019.03.012
Transthyretin cardiac amyloidosis: an update on diagnosis and treatment.
Yamamoto H, Yokochi T. · · 2019 · cited 131× · PMID 31553132 · DOI 10.1002/ehf2.12518
Inflammatory and Molecular Pathways in Heart Failure-Ischemia, HFpEF and Transthyretin Cardiac Amyloidosis.
Michels da Silva D, Langer H, Graf T. · · 2019 · cited 56× · PMID 31083399 · DOI 10.3390/ijms20092322
Amyloidosis-the Diagnosis and Treatment of an Underdiagnosed Disease.
Ihne S, Morbach C, Sommer C, Geier A, et al · · 2020 · cited 50× · PMID 32295695 · DOI 10.3238/arztebl.2020.0159
Molecular Mechanisms of Cardiac Amyloidosis.
Saito Y, Nakamura K, Ito H. · · 2021 · cited 36× · PMID 35008444 · DOI 10.3390/ijms23010025
Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis.
Zhang KW, Stockerl-Goldstein KE, Lenihan DJ. · · 2019 · cited 35× · PMID 31312767 · DOI 10.1016/j.jacbts.2019.02.002
Sex differences in transthyretin cardiac amyloidosis.
Aimo A, Panichella G, Garofalo M, Gasparini S, et al · · 2024 · cited 33× · PMID 37566193 · DOI 10.1007/s10741-023-10339-w
Specific Therapy for Transthyretin Cardiac Amyloidosis: A Systematic Literature Review and Evidence-Based Recommendations.
Marques N, Azevedo O, Almeida AR, Bento D, et al · · 2020 · cited 17× · PMID 32969287 · DOI 10.1161/jaha.120.016614

Verify or expand the search:

Other trials of AG10

Trials testing the same drug.

NCT04418024 — Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy · Phase 3 · withdrawn

Other Eidos Therapeutics, a BridgeBio company trials

Trials by the same sponsor.

NCT04988386 — Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants · Phase 3 · active not recruiting
NCT04769479 — A Single Dose Study to Evaluate the Pharmacokinetics of Acoramidis Modified Release Formulations in Healthy Subjects · Phase 1 · completed
NCT04418024 — Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy · Phase 3 · withdrawn
NCT03860935 — Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy · Phase 3 · completed
NCT03294707 — Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10 · Phase 1 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03458130 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Eidos Therapeutics, a BridgeBio company
Last refreshed: 16 November 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03458130.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing