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|
Study ID |
Status |
Indication |
Study Drug |
Description |
References |
|---|---|---|---|---|---|
|
ARGX-113-2007 |
Active, recruiting | Myositis or IIM (IMNM, DM, PM) | Efgartigimod (Vyvgart) |
A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy. | |
|
ARGX-113-2011 |
Active, recruiting | Myositis or IIM (IMNM, DM, PM) | Efgartigimod (Vyvgart) |
A Phase 3, Single-Arm, Multicenter, Open-label Extension of Study ARGX-113-2007 to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy. | |
|
TEG4001 |
Active, recruiting | hATTR-PN | Inotersen (Tegsedi) |
A Prospective, Non-interventional, Long-term, Multinational Cohort Safety Study of Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN). | |
|
ARGX-117-2301 |
Not active yet | DM | Empasiprubart | A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of Multiple Dose Regimens of Empasiprubart in Adults With Dermatomyositis. | N/A |
|
ALN-TTRSC02-002 |
Active, not recruiting |
hATTR | Vutrisiran (Amvuttra) |
A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis). |
https://clinicaltrials.gov/study/NCT03759379 |
|
ION-682884-CS13 |
Active, not recruiting |
hATTR | Eplontersen (Wainua) |
An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. | |
|
ION-682884-CS3 |
Completed |
hATTR | Eplontersen (Wainua) |
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. | |
|
ALN-TTR02-006 |
Completed | hATTR | Patisiran (Onpattro) |
A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Clinical Study with Patisiran. |
https://clinicaltrials.gov/study/NCT02510261 |
|
ALN-TTR02-004 |
Completed | hATTR | Patisiran (Onpattro) |
A Phase 3 Multicenter, Multinational, Randomized, Doubleblind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP). |
https://clinicaltrials.gov/study/NCT01960348 |
IIM: Idiopathic Inflammatory Myopathy, IMNM: Immune-Mediated Necrotizing Myopathy, DM: Dermatomyositis, PM: Polymyositis, hATTR: hereditary transthyretin amyloidosis, PN: Polyneuropathy, N/A: not available.