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Antisense Oligonucleotide

ION-682884 for Transthyretin Amyloid Cardiomyopathy

Phase 2

Waitlist Available

Research Sponsored by Brigham and Women's Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 65-85 years

Only patient who have completed 24 months of therapy in the open label clinical trial of inotersen, "A 24-month open-label study of the tolerability and efficacy of an antisense oligonucleotide (inotersen) in patients with transthyretin (TTR) amyloid cardiomyopathy" (Protocol #:2018-P001436) will be enrolled. All patients in that study had either wild-type transthyretin amyloidosis (ATTRwt) or mutant transthyretin amyloidosis (ATTRm) cardiac amyloidosis, defined by standard criteria.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 48 months

Awards & highlights

Study Summary

This trial is testing a new drug to see if it can slow or stop the progression of a disease called TTR amyloid cardiomyopathy. The disease is caused by a protein called transthyretin, which is produced in the liver. The new drug, ION-682884, is designed to reduce production of the protein by the liver. The trial will last for at least 36 months, and the patients will be monitored for safety and tolerability.

Who is the study for?

This trial is for patients aged 65-85 with TTR amyloid cardiomyopathy who have completed a previous 24-month inotersen study. They must be stable (NYHA class I-III), able to self-administer injections, and willing to take Vitamin A supplements. Excludes those with severe heart issues, uncontrolled infections or arrhythmias, certain kidney conditions, blood disorders, high bilirubin levels, recent major cardiovascular events or surgeries.Check my eligibility

What is being tested?

The trial tests the drug ION-682884's ability to slow down or stop heart damage caused by TTR amyloid deposits. It involves subcutaneous injections every four weeks and compares results against historical data using advanced cardiac imaging techniques over an open-ended period until FDA approval or discontinuation.See study design

What are the potential side effects?

While specific side effects of ION-682884 are not listed here, similar drugs can cause injection site reactions, liver problems (like raised enzyme levels), potential kidney issues reflected in urine protein levels, and may require Vitamin A supplementation due to its reduction.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 65 and 85 years old.

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I completed 24 months in the inotersen study for TTR amyloid cardiomyopathy.

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I am willing to take Vitamin A supplements during and 3 months after the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 48 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~48 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in cardiac MRI

Echocardiographic change

change in 6 minute walk

+3 more

Secondary outcome measures

Response of transthyretin levels to therapy

Side effect profile: platelets

Side-effect profile: renal function

+1 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Drug subcutaneous injectionExperimental Treatment1 Intervention

Monthly injection of ION 682884, administered subcutaneously at a dose of 45 mg.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Brigham and Women's HospitalLead Sponsor

1,609 Previous Clinical Trials

11,470,310 Total Patients Enrolled

Rodney H FalkStudy DirectorBrigham and Women's Hospital

Media Library

ION-682884 (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04843020 — Phase 2

Transthyretin Amyloid Cardiomyopathy Research Study Groups: Drug subcutaneous injection

Transthyretin Amyloid Cardiomyopathy Clinical Trial 2023: ION-682884 Highlights & Side Effects. Trial Name: NCT04843020 — Phase 2

ION-682884 (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04843020 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can adults participate in this experiment?

"This particular clinical trial is only seeking patients that are aged 65 to 85. In contrast, there are 16 other studies currently underway for treatments targeting those under 18 years old and 245 trials for interventions focused on elderly care."

Answered by AI

How does ION 682884's safety profile compare to other treatments?

"ION 682884 falls into Phase 2 of clinical trials, meaning that while there is some evidence supporting its safety, there is no data yet to suggest whether or not the medication is effective."

Answered by AI

Who would be a good candidate for this particular clinical trial?

"This study is enrolling 17 participants who are currently 65-85 years old and have amyloidosis. The following additional criteria must be met: Patients should, in the opinion of the Investigator, be in a stable state in terms of New york Heart Association (NYHA) class. Class I-III patients will be recruited., Male, or non-pregnant, non-lactating females. If a male partners with a premenopausal woman, he must be willing to use the following methods of contraception: condoms, oral/hormonal contraception, Intrauterine Device, diaphragm, or"

Answered by AI

Are we still looking for more individuals to enroll in this research?

"Unfortunately, the most recent update on clinicaltrials.gov shows that this particular study is not presently looking for patients. The trial was initially posted June 1st, 2021 but has not been updated since April 12th of the same year. That said, there are 253 other trials which are recruiting patients as we speak."

Answered by AI

What are the goals of this research?

"The primary objective of this long-term study is to observe changes in cardiopulmonary testing over 48 months. Additionally, the trial will track secondary objectives such as the effect of therapy on renal function and platelet count, as well as response of transthyretin levels to weekly inotersen injections."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

ION-682884 in Patients With TTR Amyloid Cardiomyopathy

Rodney H. Falk, MD 2021. "ION-682884 in Patients With TTR Amyloid Cardiomyopathy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04843020.

All > Cardiomyopathy > Amyloidosis