Eplontersen Long-Term Safety for Transthyretin-Mediated Amyloid Cardiomyopathy
Eplontersen
Amyloidosis+1
+ Metabolic Diseases
+ Nutritional and Metabolic Diseases
Treatment Study
Summary
Study start date: November 30, 2022
Actual date on which the first participant was enrolled.This study aims to evaluate the long-term safety of a medication called Eplontersen (ION-682884) in treating patients with a condition known as Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM). This condition affects the heart and is caused by the accumulation of abnormal proteins. ATTR-CM can lead to heart failure and other severe health issues, so finding a safe and effective long-term treatment is crucial for improving patients' quality of life. The study includes up to 1400 participants who were previously part of another study involving Eplontersen. Participants in this study will receive Eplontersen treatment every four weeks for up to 36 months, or until the medication becomes available in their country. Additionally, they will take daily doses of vitamin A as recommended. The study will monitor several health indicators to determine the safety of the treatment, including kidney function, platelet count, and protein levels in urine. Researchers will also keep track of any adverse events or serious side effects that participants experience to ensure the medication is safe for long-term use.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.1400 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 18 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Inclusion Criteria: 1. Satisfactory completion of Treatment Period and the End of Treatment Visit of the Index Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participation on ISIS 420915- CS101 study as judged by the Investigator and Sponsor. 2. Investigator is willing to treat the participant with open-label eplontersen. 3. Willingness to adhere to vitamin A supplementation per protocol. Exclusion Criteria: 1. Permanently discontinued study drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study). 2. Have any new condition or worsening of an existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrolment, or which could interfere with the participant participating in or completing the study, including the need for treatment with medications disallowed in the Index Study.
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 122 locations
Cedars-Sinai Medical Center
Beverly Hills, United StatesAltman Clinical and Translational Research Institute - Center for Clinical Research
La Jolla, United StatesUniversity Of California San Francisco Urology Practice
San Francisco, United States