Trials / Completed
CompletedNCT04136184
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 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 168 (actual)
- Sponsor
- Ionis Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 18 Years – 82 Years
- Healthy volunteers
- Not accepted
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/.
Detailed description
This study was a multicenter, open-label study in up to 168 participants, who were randomized to receive subcutaneous (SC) injections of either eplontersen once every 4 weeks or inotersen once a week. Participants also received daily supplemental doses of the recommended daily allowance of vitamin A. Participants included in the inotersen reference arm crossed over to eplontersen at Week 37 after completing the Week 35 assessments.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Inotersen | Inotersen by subcutaneous injection |
| DRUG | Eplontersen | Eplontersen by subcutaneous injection |
Timeline
- Start date
- 2019-12-11
- Primary completion
- 2023-04-11
- Completion
- 2023-07-12
- First posted
- 2019-10-23
- Last updated
- 2024-12-13
- Results posted
- 2024-12-10
Locations
45 sites across 16 countries: United States, Argentina, Australia, Brazil, Canada, Cyprus, France, Germany, Greece, Italy, New Zealand, Portugal, Spain, Sweden, Taiwan, Turkey (Türkiye)
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04136184. Inclusion in this directory is not an endorsement.