Ionis Pharmaceuticals, Inc.: Amyloidosis trials

The registry lists 11 Amyloidosis studies with Ionis Pharmaceuticals, Inc. as lead sponsor. 1 are open to participants now and 5 have results posted.

Phase 1 1Phase 2 2Phase 3 5Phase 4 1No phase 4(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)Enrolling by invitationNCT05667493Phase 3
• A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PNTerminatedNCT04306510Phase 4Results posted
• 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 PolyneuropathyCompletedNCT04136184Phase 3Results posted
• Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).CompletedNCT03728634Phase 1/2Results posted
• Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)CompletedNCT02175004Phase 3Results posted
• Efficacy and Safety of Inotersen in Familial Amyloid PolyneuropathyCompletedNCT01737398Phase 2/3Results posted
• The CARDIO-TTRansform Scintigraphy Sub-studyCompletedNCT06073587
• CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyCompletedNCT06073574
• A Non-interventional Cohort Safety Study of Patients With hATTR-PNActive, not recruitingNCT04850105
• CARDIO-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 Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)CompletedNCT04136171Phase 3
• ATTR Expanded Access Program (EAP) by IonisApproved for marketingNCT03400098