Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers

Study Identifier:
D9400L00001
ClinicalTrials.gov Identifier:
NCT07306949
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
1-855-752-2356 or [email protected]
Recruiting

Study Details

Medical Condition
  • Cardiomyopathy, Dilated
Study Drug
  • Drug: Acoramidis
Date
Oct 2025 - Mar 2028
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 100 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Treatment history of ATTR-CM is one of the following:
  • Naive participants: newly diagnosed with ATTR-CM and no prior treatment with drugs for ATTR-CM
  • Switch participants: Participants who are using tafamidis, a TTR stabilizer, as treatment for ATTR-CM and who, in the judgment of the post-marketing clinical trial investigator (co-principal investigator), can be expected to benefit from switching to acoramidis.
  • Naive participants must meet the following requirements:
  • History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics
  • Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm)
  • Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods
  • Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry.
  • Bone scintigraphy showing strong accumulation \*\* (Perugini score ≥ 2) consistent with myocardium and no M protein, negating the possibility of AL amyloidosis
Exclusion Criteria
  • Have confirmed diagnosis of AL amyloidosis
  • Switch participants: prior treatment with gene silencing agents (pachysilane sodium, butrisilane sodium) as treatment for ATTR-CM (including when specifically scheduled to start treatment with a gene silencing agent)
  • Likelihood of receiving a heart transplant within 1 year from the time screening begins
  • Hypersensitivity to acoramidis, its metabolites, or additives in the formulation has been confirmed.
  • Pregnant or lactating women
  • Has a clinically significant medical condition, an abnormal laboratory test result, or a condition that may jeopardize the safety of the study participant, increase the risk of participation in the post-marketing clinical trial, or affect the study
  • Participating in an interventional study other than this study, including a clinical trial
  • In the opinion of the responsible (sub)physician for the post-marketing clinical trial, has a history of drug abuse, alcoholism, or psychiatric disorder that would preclude compliance with this Post-Marketing Clinical Study Protocol
Sex
Female & Male
Age
18 - 100 Years

Study Details

Medical Condition
  • Cardiomyopathy, Dilated
Study Drug
  • Drug: Acoramidis
Date
Oct 2025 - Mar 2028
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 100 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Treatment history of ATTR-CM is one of the following:
  • Naive participants: newly diagnosed with ATTR-CM and no prior treatment with drugs for ATTR-CM
  • Switch participants: Participants who are using tafamidis, a TTR stabilizer, as treatment for ATTR-CM and who, in the judgment of the post-marketing clinical trial investigator (co-principal investigator), can be expected to benefit from switching to acoramidis.
  • Naive participants must meet the following requirements:
  • History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics
  • Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm)
  • Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods
  • Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry.
  • Bone scintigraphy showing strong accumulation \*\* (Perugini score ≥ 2) consistent with myocardium and no M protein, negating the possibility of AL amyloidosis
Exclusion Criteria
  • Have confirmed diagnosis of AL amyloidosis
  • Switch participants: prior treatment with gene silencing agents (pachysilane sodium, butrisilane sodium) as treatment for ATTR-CM (including when specifically scheduled to start treatment with a gene silencing agent)
  • Likelihood of receiving a heart transplant within 1 year from the time screening begins
  • Hypersensitivity to acoramidis, its metabolites, or additives in the formulation has been confirmed.
  • Pregnant or lactating women
  • Has a clinically significant medical condition, an abnormal laboratory test result, or a condition that may jeopardize the safety of the study participant, increase the risk of participation in the post-marketing clinical trial, or affect the study
  • Participating in an interventional study other than this study, including a clinical trial
  • In the opinion of the responsible (sub)physician for the post-marketing clinical trial, has a history of drug abuse, alcoholism, or psychiatric disorder that would preclude compliance with this Post-Marketing Clinical Study Protocol

Protocol Summary

The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.

Trial Locations

Location
Status
Location
Research Site
Bunkyō City, Japan, 113-8431
Status
Not yet recruiting
Location
Research Site
Bunkyō City, Japan, 113-8603
Status
Not yet recruiting
Location
Research Site
Kumamoto, Japan, 860-8556
Status
Not yet recruiting
Location
Research Site
Kurume-shi, Japan, 830-0011
Status
Recruiting
Location
Research Site
Kyoto, Japan, 606-8507
Status
Not yet recruiting
Location
Research Site
Mitaka-shi, Japan, 181-8611
Status
Not yet recruiting
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