Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04988386|
Recruitment Status : Enrolling by invitation
First Posted : August 3, 2021
Last Update Posted : April 1, 2022
|Condition or disease||Intervention/treatment||Phase|
|Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy||Drug: Acoramidis (AG10)||Phase 3|
The primary objective of this prospective multi-center, open-label study is to evaluate the long-term safety and tolerability of acoramidis in the context of background heart failure therapy. Selected secondary efficacy endpoints and exploratory pharmacokinetic, pharmacodynamic/biomarker endpoints may also be assessed. All participants who complete 30 months of blinded study treatment and the Month 30 assessments of the double-blind treatment period of the Phase 3 ATTRibute-CM trial (AG10-301) may be eligible to participate in this Open Label Extension (OLE) study of acoramidis. The Day 1 visit in Study AG10-304 may be the same day as the Month 30 visit in Study AG10-301. Under these circumstances, the last dose of Investigational Medicinal Product (IMP) in Study AG10-301 will be the night before the day of the Month 30 visit and the first dose of acoramidis in Study AG10-304 will be during the Study AG10-304 Day 1 visit after baseline assessments have been completed.
Currently, tafamidis is approved for the treatment of ATTR-CM in some regions. Participants are not allowed to be treated with tafamidis or any other ATTR-CM-specific approved or investigational treatments, or therapies used off-label or as non-prescription supplements for ATTR-CM at any time during the study. If participants choose treatment with a prohibited medication, they may be asked to discontinue acoramidis and they may be asked to discontinue/withdraw from the study. Under these circumstances, participants will be asked to complete an early termination visit, and a follow-up visit prior to discontinuation/withdrawal.
Participants are not permitted to participate in another interventional clinical trial (with the exception of Study AG10-301) within 30 days prior to dosing and throughout Study AG10-304. Participants who choose to participate in another interventional clinical trial may be asked to withdraw from acoramidis and/or the study. In such cases, participants will be asked to complete an early termination visit and a follow-up visit. Participation in observational and/or registry studies should be discussed with the Medical Monitor.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||545 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open-label study extension study from the AG10-301 double-blind study.|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)|
|Actual Study Start Date :||October 27, 2021|
|Estimated Primary Completion Date :||April 15, 2028|
|Estimated Study Completion Date :||May 15, 2028|
Open-label study all participants will receive AG10 during this study.
Drug: Acoramidis (AG10)
Acoramidis (AG10) twice daily
- Incidence of treatment-emergent Adverse Events [Safety and Tolerability] [ Time Frame: 60 months ]Incidence of each treatment-emergent adverse events measured over 60 Months or study completion
- Evaluate all-cause mortality and cardiovascular mortality [ Time Frame: 60 months ]Assessment of All-cause mortality and CV mortality during study period
- Evaluate the effect of acoramidis on the 6-minute walk test (6MWT) [ Time Frame: 60 months ]Change from Baseline in distance walked during the 6MWT during study period
- Evaluate the effect of acoramidis on health-related quality of life Kansas City Cardiomyopathy Questionnaire [ Time Frame: 60 months ]Change from Baseline in Kansas City Cardiomyopathy Questionnaire Overall Score during study period [scale is scored from 0-100, where 0=Poor, 100=Excellent]
- Evaluate the effect of acoramidis on the frequency of CV-related hospitalization [ Time Frame: 60 months ]assess CV-related hospitalization during the study period
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04988386