A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
NCT05071300 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 151
Last updated 2025-12-04
Summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
Conditions
- Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Interventions
- DRUG
-
Eplontersen
Eplontersen will be administered by SC injection.
Sponsors & Collaborators
-
Ionis Pharmaceuticals, Inc.
lead INDUSTRY
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2022-01-04
- Primary Completion
- 2029-08-31
- Completion
- 2029-08-31
- FDA Drug
- Yes
Countries
- United States
- Argentina
- Australia
- Brazil
- Canada
- Cyprus
- France
- Italy
- Portugal
- Spain
- Sweden
- Taiwan
- Turkey (Türkiye)
Study Locations
More Related Trials
-
The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis
NCT00630864 ·Status: COMPLETED ·Phase: PHASE2
-
A Phase II Multi-centre, Extension Study to Investigate the Long Term Safety of ONO-2506PO in Patients Diagnosed With Amyotrophic Lateral Sclerosis (ALS)
NCT00694941 ·Status: COMPLETED ·Phase: PHASE2
-
Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)
NCT04882735 ·Status: WITHDRAWN ·Phase: PHASE3
-
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
NCT05160558 ·Status: TERMINATED ·Phase: PHASE1
-
A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
NCT02053454 ·Status: COMPLETED ·Phase: PHASE1
-
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
NCT06393712 ·Status: RECRUITING ·Phase: PHASE2
-
Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation
NCT07084311 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
NCT05531890 ·Status: UNKNOWN ·Phase: PHASE1
-
A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT05039099 ·Status: COMPLETED ·Phase: PHASE2
-
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
NCT06672445 ·Status: RECRUITING ·Phase: PHASE1
-
4-Aminopyridine in Episodic Ataxia Type 2
NCT01543750 ·Status: WITHDRAWN ·Phase: PHASE2
-
A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)
NCT06069934 ·Status: NO_LONGER_AVAILABLE
-
CLG561 Proof-of-Concept Study as a Monotherapy and in Combination With LFG316 in Subjects With Geographic Atrophy (GA)
NCT02515942 ·Status: COMPLETED ·Phase: PHASE2
-
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
NCT04849741 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
NCT04201418 ·Status: COMPLETED
-
A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT04944784 ·Status: TERMINATED ·Phase: PHASE3
-
Extension Study Evaluating NTX100 Neuromodulation System for Medication-Refractory Primary RLS
NCT05196828 ·Status: COMPLETED ·Phase: NA
-
A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis
NCT00800501 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
NCT06673056 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
Long-Term Follow-Up Study for Safety, Efficacy and Tolerability of Rotigotine in Adolescents With Restless Legs Syndrome
NCT01498120 ·Status: COMPLETED ·Phase: PHASE2
-
Therapeutic Efficacy of Tiludronic Acid on Inner Ear Involvement in Advanced Otosclerosis
NCT01617057 ·Status: TERMINATED ·Phase: PHASE3
-
Dexpramipexole Renal PK Study
NCT01424176 ·Status: COMPLETED ·Phase: PHASE1
-
A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis
NCT05006352 ·Status: COMPLETED ·Phase: PHASE1
-
Treating Amyotrophic Lateral Sclerosis (ALS) With R(+) Pramipexole Dihydrochloride Monohydrate at 60 mg/Day
NCT00596115 ·Status: TEMPORARILY_NOT_AVAILABLE
-
ALS Phase II Study of NX210c
NCT06365216 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2