By InvitationPhase 3

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

United States1,400 participantsStarted 2022-11-30

Plain-language summary

The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)

Timeframe: Baseline up to 36 months

Change From Baseline in Platelet Count

Timeframe: Baseline up to 36 months

Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)

Timeframe: Baseline up to 36 months

Change From Baseline in Urine Protein Creatinine Ratio (UPCR)

Timeframe: Baseline up to 36 months

NCT IDNCT05667493

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-08

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

What they're measuring

Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)

Timeframe: Baseline up to 36 months

Change From Baseline in Platelet Count

Timeframe: Baseline up to 36 months

Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)

Timeframe: Baseline up to 36 months

Change From Baseline in Urine Protein Creatinine Ratio (UPCR)

Timeframe: Baseline up to 36 months