An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

NCT05667493
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Phase
Phase 3
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Recruitment status
Enrolling by invitation
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Participants
1,400
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Lead sponsor
Ionis Pharmaceuticals, Inc.
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Start date
2022-11-30
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Number of sites
134
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Summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
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Short title
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
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Official title
An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
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Control arm
Open label
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Registry reports results
No
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Listing at source
Listed
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Recruitment status
Enrolling by invitation
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Phase
Phase 3
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Study type
Interventional
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Completion date
2029-08-01
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First posted
2022-12-28
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Registry last updated
2026-04-17
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Primary completion date
2029-08-01
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Start date
2022-11-30
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Participants
1,400
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Participant count basis
Estimated
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Planned participants
1,400
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Protocol amendments
1
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Number of sites
134
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Data monitoring committee
No
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Registry sponsor category
Industry
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Lead sponsor
Ionis Pharmaceuticals, Inc.
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Source registry
ClinicalTrials.gov
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Registry ID
NCT05667493
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ClinicalTrials.gov ID
NCT05667493
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Registry record
Open registry record
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Allocation
Not applicable
Model
Single group
Purpose
Treatment
Masking
No masking
Sources for study design
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  1. Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)

    Timeframe: Baseline up to 36 months

  2. Change From Baseline in Platelet Count

    Timeframe: Baseline up to 36 months

  3. Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)

    Timeframe: Baseline up to 36 months

  4. Change From Baseline in Urine Protein Creatinine Ratio (UPCR)

    Timeframe: Baseline up to 36 months

  1. Change From Baseline in Transthyretin (TTR) Serum Levels

    Timeframe: Baseline up to 36 months

  2. Change From Baseline in 6-minute Walk Test (6MWT)

    Timeframe: Baseline up to 36 months

  3. Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)

    Timeframe: Baseline up to 36 months

Sources for outcomes
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Sex
All
Age groups
Adults, Older adults
Minimum age
18 Years
Healthy volunteers
No

  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.

  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.
Sources for eligibility
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  • Mayo ClinicScottsdale, United States
  • Cedars-Sinai Medical CenterBeverly Hills, United States
  • Altman Clinical and Translational Research Institute - Center for Clinical ResearchLa Jolla, United States
  • University Of California San Francisco Urology PracticeSan Francisco, United States
  • Stanford University (Leland Stanford Junior University)Stanford, United States
  • University of Colorado Hospital - Anschutz Medical CampusAurora, United States
  • MedStar Washington Hospital CenterWashington D.C., United States
  • The George Washington Medical Faculty Associates - Foggy Bottom North PavilionWashington D.C., United States
  • Cleveland Clinic FloridaWeston, United States
  • Piedmont Heart of FayettevilleAtlanta, United States
  • Emory Heart and Vascular Center - Emory Clifton CampusAtlanta, United States
  • Piedmont Atlanta HospitalFayetteville, United States
  • Northwestern UniversityChicago, United States
  • Loyola University Medical CenterMaywood, United States
  • Indiana University Health University HospitalIndianapolis, United States
  • University of Kansas Medical Center (KUMC)Kansas City, United States
  • University of Kansas Medical CenterKansas City, United States
  • University of Maryland Medical CenterBaltimore, United States
  • Massachusetts General HospitalBoston, United States
  • Brigham and Women's HospitalBoston, United States
  • Boston University School of MedicineBoston, United States
  • Henry Ford Health SystemDetroit, United States
  • Allina Health Minneapolis Heart Institute - Abbott Northwestern's Heart HospitalMinneapolis, United States
  • Mayo ClinicRochester, United States
  • Saint Luke's Hospital of Kansas CityKansas City, United States

And 109 more, listed on the interactive page.

Sources for sites
clinicaltrials.gov

Other registry identifiers

  • 2022-000826-74
Sources for references
clinicaltrials.gov

Related records

Drugs (1)

Collaborators (1)

Lead sponsors (1)

Conditions (1)

Targets (1)