An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Overview
- Phase
- Phase 3
- Intervention
- Eplontersen
- Conditions
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Enrollment
- 1400
- Locations
- 245
- Primary Endpoint
- Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
- Status
- Enrolling By Invitation
- Last Updated
- 15 days ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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.
Exclusion Criteria
- •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.
Arms & Interventions
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.
Intervention: Eplontersen
Outcomes
Primary Outcomes
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Time Frame: Baseline up to 36 months
Change From Baseline in Platelet Count
Time Frame: Baseline up to 36 months
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Time Frame: Baseline up to 36 months
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
Time Frame: Baseline up to 36 months
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Time Frame: Baseline up to 36 months
Change From Baseline in Platelet Count
Time Frame: Baseline up to 36 months
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Time Frame: Baseline up to 36 months
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
Time Frame: Baseline up to 36 months
Secondary Outcomes
- 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)
- 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)