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NCT04136184 · ClinicalTrials.gov registry record · Phase 3
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
A Phase 3 study, sponsored by Ionis Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 168
- Enrollment target
NCT04136184: Completed Phase 3 study, sponsored by Ionis Pharmaceuticals.
NCT04136184 is a Phase 3 study that has completed, run by Ionis Pharmaceuticals. The registered enrollment target is 168 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (78% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04136184, a Phase 3 study, has completed, sponsored by Ionis Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 168 participants
- Enrollment target
Study Summary
The main objective of this study was to evaluate the efficacy of eplontersen as compared with the historical control of the placebo cohort in the NEURO-TTR trial (NCT01737398/2012-001831-30), in subjects with hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN). For more information, please visit http://www.neuro-ttransform.com/.
Primary Outcome
mNIS+7 composite score is a measure of neurologic impairment evaluating muscle weakness, sensation, reflexes, nerve conduction, and autonomic function. mNIS+7 consists of 2 composite scores: the NIS composite score (maximum of 244 points) \& the modified +7 composite score (maximum of 102.32 points). mNIS+7 composite total score range= -22.32 to 346.32. Higher score indicated a lower function. Least square (LS) mean \& standard error (SE) were analyzed using Mixed Effects Model with Repeated Mea
Interventions
- DRUG Eplontersen
- DRUG Inotersen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 168 participants |
| Start Date | 2019-12-11 |
| Est. Completion | 2023-07-12 |
| Phase | Phase 3 |
What the finished NCT04136184 record still lists
NCT04136184 is an interventional study that assigns participants to a tested intervention. The registered 168 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (78% lower).
The record links to 0 conditions, and to 2 interventions - of which Eplontersen is the first listed.
NCT04136184 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04136184 about?
NCT04136184 is a clinical study titled "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". The main objective of this study was to evaluate the efficacy of eplontersen as compared with the historical control of the placebo cohort in the NEURO-TTR trial (NCT01737398/2012-001831-30), in subjects with hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN). For more informati...
What is the current status of trial NCT04136184?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 168 participants. The study started on 2019-12-11. Estimated completion is 2023-07-12.
What interventions are being tested in trial NCT04136184?
The interventions under investigation include: Eplontersen (DRUG), Inotersen (DRUG).
Who is sponsoring clinical trial NCT04136184?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04136184's enrollment target sits among peer trials
168 1459th of 2000 higher than 538 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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