An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
- Conditions
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
- Interventions
- Registration Number
- NCT05667493
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
- Detailed Description
This is a multicenter, open-label extension, Phase 3 study in up to approximately 1400 participants from the 682884-CS2 study. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or until after eplontersen is approved and available in the site's country, whichever occurs first. Participants will also receive daily supplemental doses of the recommended daily allowance (RDA) of vitamin A.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 1400
- 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.
- Investigator is willing to treat the participant with open-label eplontersen.
- Willingness to adhere to vitamin A supplementation per protocol.
- Permanently discontinued study drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study).
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Eplontersen Eplontersen Eplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first.
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) Baseline up to 36 months Change From Baseline in Platelet Count Baseline up to 36 months Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) Baseline up to 36 months Change From Baseline in Urine Protein Creatinine Ratio (UPCR) Baseline up to 36 months
- Secondary Outcome Measures
Name Time Method Change From Baseline in 6-minute Walk Test (6MWT) Baseline up to 36 months Change From Baseline in Transthyretin (TTR) Serum Levels Baseline up to 36 months Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Baseline up to 36 months
Trial Locations
- Locations (96)
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Cedars-Sinai Medical Center
🇺🇸Beverly Hills, California, United States
Altman Clinical and Translational Research Institute - Center for Clinical Research
🇺🇸La Jolla, California, United States
Stanford University (Leland Stanford Junior University)
🇺🇸Stanford, California, United States
University of Colorado Hospital - Anschutz Medical Campus
🇺🇸Aurora, Colorado, United States
MedStar Washington Hospital Center
🇺🇸Washington, District of Columbia, United States
The George Washington Medical Faculty Associates - Foggy Bottom North Pavilion
🇺🇸Washington, District of Columbia, United States
Cleveland Clinic Florida
🇺🇸Weston, Florida, United States
Piedmont Heart of Fayetteville
🇺🇸Atlanta, Georgia, United States
Emory Heart and Vascular Center - Emory Clifton Campus
🇺🇸Atlanta, Georgia, United States
Scroll for more (86 remaining)Mayo Clinic🇺🇸Rochester, Minnesota, United States