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Active and Completed Clinical Trials at CING

 

 

Study ID

Status at site

Indication

Study Drug
(Brand Name)

Description

References
(ClinicalTrials.gov/Pubmed)

ALN-TTRSC04-004 
(TRITON-PN)

 Initiated hATTR-PN Nucresiran A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN).

https://clinicaltrials.gov/study/NCT07223203

ARGX-113-2308 
(ADAPT SERON)

 Active, not recruiting AChR seronegative
gMG
 		 Efgartigimod A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis.

https://clinicaltrials.gov/study/NCT06298552

INC-6601

Startup

 CMT N/A Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others (INC-6601).

https://clinicaltrials.gov/study/NCT01193075

https://inc.rarediseasesnetwork.org/clinical-sites-0
 

ARGX-113-2007 
(ALKIVIA)

 Completed Myositis or IIM (IMNM, DM, PM) Efgartigimod

 		 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.

https://clinicaltrials.gov/study/NCT05523167

ARGX-113-2011 
(ALKIVIA+)

Completed

 Myositis or IIM (IMNM, DM, PM) Efgartigimod

 		 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.

https://clinicaltrials.gov/study/NCT05979441

ARGX-113-2315 
(ADAPT OCULUS)

Completed

 Ocular Myasthenia Gravis Efgartigimod A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia Gravis

https://clinicaltrials.gov/study/NCT06558279

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).

https://clinicaltrials.gov/study/NCT04850105

TEG4002

 Active, not recruiting hATTR-PN Inotersen
(Tegsedi)
 		 A Retrospective, Non-interventional, Multi-centre Study of TEGSEDI-treated Patients to Evaluate Real-world Adherence to, and Effectiveness of the Recommendations for Platelet Monitoring, Dose Adjustment, and Steroid Initiation to Manage Risk of Thrombocytopenia

https://catalogues.ema.europa.eu/node/2403/administrative-details

ALN-TTRSC02-002 
(HELIOS-A)

 Completed
 		 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

https://pubmed.ncbi.nlm.nih.gov/35875890/

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.

https://clinicaltrials.gov/study/NCT05071300

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.

https://pubmed.ncbi.nlm.nih.gov/33638113/

https://clinicaltrials.gov/study/NCT04136184

 

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

https://pubmed.ncbi.nlm.nih.gov/33212063/

ALN-TTR02-004 
(APOLLO)

 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

https://pubmed.ncbi.nlm.nih.gov/29972753/

 

AChR: Acetylcholine Receptor Binding Antibody, CMT: Charcot Marie Tooth Disease, DM: Dermatomyositis, gMG: Generalised Myasthenia Gravis, hATTR: Hereditary Transthyretin Amyloidosis, IIM: Idiopathic Inflammatory Myopathy, IMNM: Immune-Mediated Necrotizing Myopathy, PM: Polymyositis, PN: Polyneuropathy, N/A: Not Applicable. 

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