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Antisense Oligonucleotide

Eplontersen for Transthyretin-Mediated Amyloid Polyneuropathy

Phase 3

Waitlist Available

Research Sponsored by Ionis Pharmaceuticals, Inc.

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 35, week 66

Awards & highlights

Study Summary

This trial is testing a new drug for a disease that causes nerve damage. The new drug is being compared to another drug that is already being used to treat this disease.

Eligible Conditions

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 35, week 66

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 35, week 66

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 35, week 66 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 from baseline in mNIS+7 at Week 66

Change from baseline in modified neuropathy impairment score plus 7 (mNIS+7) at Week 35

Change from baseline in the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66

+2 more

Secondary outcome measures

Change from baseline in Neuropathy Symptom and Change (NSC) score at Weeks 35 and 66

Change from baseline in Norfolk QOL-DN at Week 35

Change from baseline in Polyneuropathy Disability (PND) score at Week 65

+2 more

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: EplontersenExperimental Treatment1 Intervention

Eplontersen by subcutaneous injection once every 4 weeks.

Group II: InotersenActive Control2 Interventions

Inotersen by subcutaneous injection once weekly through week 34. Participants will then convert to Eplontersen administered subcutaneously once every 4 weeks until the end of study.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Eplontersen

2020

Completed Phase 3

~200

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Ionis Pharmaceuticals, Inc.Lead Sponsor

146 Previous Clinical Trials

15,114 Total Patients Enrolled

Frequently Asked Questions

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

What is the FDA's decision on Eplontersen?

"Eplontersen has received a score of 3 for safety by our team at Power. This is due to it being a Phase 3 trial, which means that there is both efficacy and safety data available."

Answered by AI

Will this trial be open to individuals who have not yet reached their 55th birthday?

"The age range for eligible patients in this trial is 18 to 82 years old."

Answered by AI

Has a study like this one been conducted before?

"There have been a total of 3 clinical trials for Eplontersen. The first trial was conducted in 2020 and 168 people participated. The sponsor for this trial was Ionis Pharmaceuticals, Inc. and it successfully completed Phase 3. There have been no new trials since 2020."

Answered by AI

Who is this study looking for as participants?

"This trial is meant for patients with polyneuropathies who are also between 18 and 82 years old. Up to 168 people can participate."

Answered by AI

What are the precedent studies for Eplontersen?

"Eplontersen is being studied in 3 concurrent Phase 3 clinical trials. These international studies are based in 209 different locations, with the majority of tests being conducted in Messina, Guishan District."

Answered by AI

Are there still open slots in this trial for new participants?

"This clinical trial is not currently recruiting participants anymore. The study was first posted on 1/15/2020 and was most recently edited on 9/29/2022. If you are looking for for other studies, there are presently 123 studies actively admitting patients with polyneuropathies and 3 trials for Eplontersen actively looking for participants."

Answered by AI

Could you explain what the main goals are for this research?

"The purpose of this clinical trial is to evaluate the effect of the medication on Percent change from baseline in serum TTR concentration at Week 66. Additionally, the study will also look at secondary outcomes including Change from baseline in Neuropathy Symptom and Change (NSC) score at Weeks 35 and 66, Change from baseline in the Physical Component Summary (PCS) score of the 36-Item Short Form Survey (SF-36) at Week 65, and Change from baseline in Polyneuropathy Disability (PND) score at Week 65."

Answered by AI

Recent research and studies

Neurology and TherapyJournal

Design and Rationale of the Global Phase 3 Neuro-ttransform Study of Antisense Oligonucleotide Akcea-ttr-lrx (ION-682884-CS3) in Hereditary Transthyretin-mediated Amyloid Polyneuropathy

Coelho, Teresa, Yukio Ando, Merrill D. Benson, John L. Berk, Márcia Waddington-Cruz, Peter J. Dyck, Julian D. Gillmore, et al.. 2021. “Design and Rationale of the Global Phase 3 Neuro-ttransform Study of Antisense Oligonucleotide Akcea-ttr-lrx (ION-682884-CS3) in Hereditary Transthyretin-mediated Amyloid Polyneuropathy”. Neurology and Therapy. Springer Science and Business Media LLC. doi:10.1007/s40120-021-00235-6.

clinicaltrials.govStudy

NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

2020. "NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04136184.