To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)

ConditionsAmyloidosis in Transthyretin (TTR)Amyloidosis, Familial Amyloid Cardiomyopathy Amyloid Cardiomyopathy, Transthyretin-Related Amyloidogenic Transthyretin (ATTR) Amyloidosis Heart Disease Cardiomyopathies Amyloidosis, Hereditary, Transthyretin-Related Polyneuropathies Amyloidosis

Trial statusNot yet recruiting

Trial phasePhase 3

Trial typeInterventional

Biological sexAll

Age18+

SponsorEidos Therapeutics, a BridgeBio company

About this trial

The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

Participants who completed the AG10-501 study with a diagnosis of ATTR-PN only or who permanently discontinued study drug prior to diagnosis of ATTR-CM in AG10-501.

History of light-chain amyloidosis (AL) or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).

History of a monoclonal paraprotein or abnormal light chains in serum or urine (ie, monoclonal gammopathy of undetermined significance, MGUS) in which AL has not been ruled out.

Stage IV or V chronic kidney disease.

Trial design

Design model

Single group

Treatments tested in this trial

Acoramidis (AG10)
Drug
TTR stabilizer administered orally twice daily (BID)

Treatment groups

250 Participants

are divided into 1 treatment group

Group A: AcoramidisExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

To evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM

Proportion of participants with: treatment-emergent AEs and SAEs, AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal ECG parameters of clinical relevance, changes in clinical safety laboratory parameters of potential concern

Time frame

The duration of study participation for the participants will be up to 5 years.

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Eidos Therapeutics, a BridgeBio company

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: