ATTR Amyloidosis With Cardiomyopathy: Expanded Access Protocol to Provide Patisiran

What is the Purpose of this Study?

This study is an Expanded Access Program that focuses on individuals who have transthyretin amyloidosis with cardiomyopathy (ATTR amyloidosis with cardiomyopathy). Expanded Access Programs are a means by which investigational treatments are made available to treat patients with serious diseases who are unable to participate in an ongoing clinical trial or whose treatment options are otherwise limited. In this study, an experimental drug called patisiran is being offered for the treatment of ATTR amyloidosis with cardiomyopathy. (Patisiran is an approved therapy in the U.S. for the treatment of hereditary ATTR amyloidosis with polyneuropathy.) ATTR amyloidosis is a rare and serious disease that affects multiple systems of the body, including the heart. It is caused by certain differences in a protein called transthyretin (TTR). Patisiran blocks the ability of ribonucleic acid (RNA) to produce normal and abnormal forms of the TTR protein. This decrease in production is expected to lower TTR levels in the blood and reduce the progression of amyloid build up in organs of patients with ATTR amyloidosis. Amyloid accumulation can often compromise the function of important organs.


Eligibility

  • Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hATTR amyloidosis with cardiomyopathy or wtATTR amyloidosis with cardiomyopathy; AND
  • Had an inadequate response to or could not tolerate standard of care, in the opinion of the investigator.
  • Is not eligible for on-label use of commercial patisiran in the opinion of the investigator.
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Where can I participate?

Beverly

More about this Clinical Trial

What is the full name of this clinical trial?

Expanded Access Protocol to Provide Patisiran to Patients with Transthyretin-mediated Amyloidosis with Cardiomyopathy

Study Details
Disease Type/Condition

ATTR-CM, Cardiac Amyloidosis

Principal Investigator

Nikolova, Andriana

Age Group

Adult

Phase

N/A

IRB Number

STUDY00002065

ClinicalTrials.gov ID

NCT05505838

Key Eligibility
ClinicalTrials.gov

How do I learn more about this study?
Email
clinicaltrials@cshs.org
Study Detail
Disease Type/Condition

ATTR-CM, Cardiac Amyloidosis

Principal Investigator

Nikolova, Andriana

Age Group

Adult

Phase

N/A

IRB Number

ALN-TTR02-014

ClinicalTrials.gov ID

NCT05505838

Key Eligibility
ClinicalTrials.gov

Contact
Email
clinicaltrials@cshs.org