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

PHASE3RECRUITING

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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Study details:

This is a multicenter, open-label, Phase 3 study in up to approximately 1400 participants. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or 6 months 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.

This study will consist of the following periods: less than or equal to (≤) 10-week screening assessment period, up to 36-month treatment period, and up to 6-month post-treatment evaluation period.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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.

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2022-11-30

Primary completion: 2029-04-01

Study completion finish: 2029-08-01

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT05667493

Intervention or treatment

DRUG: Eplontersen

Conditions

• Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Image related to Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Condition: Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
DRUG: Eplontersen
Liverpool, Not Specified, Australia and more
Sponsor: Ionis Pharmaceuticals, Inc.

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

Liverpool Hospital

Research sites nearby

Select from list below to view details:

Liverpool Hospital
Liverpool, Not Specified, Australia
Advara HeartCare Murdoch
Murdoch, West Australia, Australia
Advara HeartCare
Joondalup, Western Australia, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: 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.	DRUG: Eplontersen Eplontersen will be administered by SC injection.

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)	Not Specified	Baseline up to 36 months
Change From Baseline in Platelet Count	Not Specified	Baseline up to 36 months
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)	Not Specified	Baseline up to 36 months
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)	Not Specified	Baseline up to 36 months
Change From Baseline in AST	Not Specified	Baseline up to 36 months
Change From Baseline in ALT	Not Specified	Baseline up to 36 months
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters	Not Specified	Baseline up to 36 months
Number of Participants With Clinically Significant Changes From Baseline in Thyroid-Stimulating Hormone (TSH)	Not Specified	Baseline up to 36 months
Percentage of Participants With Anti-Drug Antibodies (ADA)	Not Specified	Baseline up to 36 months

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Change From Baseline in Transthyretin (TTR) Serum Levels	Not Specified	Baseline up to 36 months
Change From Baseline in 6-minute Walk Test (6MWT)	Not Specified	Baseline up to 36 months
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)	Not Specified	Baseline up to 36 months
Change From Baseline in Short Form Health Survey Questionnaire (SF-36)	Not Specified	Baseline up to 36 months
Change From Baseline in 5 Level EuroQol - 5 Dimension (EQ-5D-5L)	Not Specified	Baseline up to 36 months
Change From Baseline in Biomarker: N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)	Not Specified	Baseline up to 36 months
Change From Baseline in Biomarker: High Sensitivity Cardiac Troponin T (hs-cTnT)	Not Specified	Baseline up to 36 months

Frequently Asked Questions

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You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

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