Alnylam Pharmaceuticals: Amyloidosis trials

The registry lists 27 Amyloidosis studies with Alnylam Pharmaceuticals as lead sponsor. 6 are open to participants now and 10 have results posted.

Phase 1 5Phase 2 5Phase 3 9No phase 8(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.

• DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World SettingRecruitingNCT07358078
• TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With PolyneuropathyRecruitingNCT07223203Phase 3
• TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With CardiomyopathyRecruitingNCT07052903Phase 3
• A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyEnrolling by invitationNCT06679946Phase 3
• Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance ProgramRecruitingNCT05040373
• ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)RecruitingNCT04561518
• HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyActive, not recruitingNCT04153149Phase 3Results posted
• APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)CompletedNCT03997383Phase 3Results posted
• Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver TransplantCompletedNCT03862807Phase 3Results posted
• The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver TransplantCompletedNCT02595983Phase 2Results posted
• The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)CompletedNCT02510261Phase 3Results posted
• ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)CompletedNCT02319005Phase 3Results posted
• A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac AmyloidosisCompletedNCT02292186Phase 2Results posted
• The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)CompletedNCT01961921Phase 2Results posted
• APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated AmyloidosisCompletedNCT01960348Phase 3Results posted
• Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) AmyloidosisCompletedNCT01617967Phase 2Results posted
• Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With CardiomyopathyNo longer availableNCT05505838
• A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A MutationCompletedNCT04201418
• A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) DiseaseTerminatedNCT03774784
• Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)Approved for marketingNCT02939820
• A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy SubjectsCompletedNCT02797847Phase 1
• DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac AmyloidosisCompletedNCT02252653
• A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy VolunteersCompletedNCT02053454Phase 1
• Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac AmyloidosisCompletedNCT01981837Phase 2
• A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy VolunteersCompletedNCT01814839Phase 1
• Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer SubjectsCompletedNCT01559077Phase 1
• Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) AmyloidosisCompletedNCT01148953Phase 1