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Results for “Hereditary Transthyretin-Mediated Amyloid Polyneuropathy”

20 trials

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Showing 20 of 20 results

Not applicableLooking for participantsNCT04561518
What this trial is testing

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

Who this might be right for
Transthyretin-Mediated AmyloidosisATTR Amyloidosis
Alnylam Pharmaceuticals 1,500
Large-scale testing (Phase 3)Active Not RecruitingNCT05071300
What this trial is testing

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

Who this might be right for
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Ionis Pharmaceuticals, Inc. 151
Not applicableLooking for participantsNCT05040373
What this trial is testing

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Who this might be right for
Hereditary Transthyretin-mediated (hATTR) AmyloidosisPolyneuropathy
Alnylam Pharmaceuticals 10
Not applicableNot Yet RecruitingNCT04270058
What this trial is testing

TEGSEDI Pregnancy Surveillance Program

Who this might be right for
Hereditary Transthyretin-mediated Amyloidosis With PolyneropathyPregnancy
Akcea Therapeutics 20
Not applicableApproved For MarketingNCT02939820
What this trial is testing

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Who this might be right for
TTR-mediated AmyloidosisAmyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more
Alnylam Pharmaceuticals
Large-scale testing (Phase 3)Active Not RecruitingNCT06672237
What this trial is testing

NTLA-2001 in ATTRv-PN

Who this might be right for
Neuromuscular DiseaseNeuromuscular Diseases (NMD)Neurodegenerative Disease+17 more
Intellia Therapeutics 50
Not applicableLooking for participantsNCT06360289
What this trial is testing

Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy

Who this might be right for
Hereditary Amyloidosis, Transthyretin-RelatedAsymptomatic Carrier State
Alnylam Pharmaceuticals 500
Testing effectiveness (Phase 2)Study completedNCT02595983
What this trial is testing

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 Transplant

Who this might be right for
Transthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)ATTR Amyloidosis+1 more
Alnylam Pharmaceuticals 12
Large-scale testing (Phase 3)Study completedNCT02510261
What this trial is testing

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)

Who this might be right for
Amyloidosis
Alnylam Pharmaceuticals 211
Large-scale testing (Phase 3)Study completedNCT04136184
What this trial is testing

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 Polyneuropathy

Who this might be right for
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Ionis Pharmaceuticals, Inc. 168
Early research (Phase 1)Study completedNCT02797847
What this trial is testing

A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

Who this might be right for
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
Alnylam Pharmaceuticals 80
Post-approval studies (Phase 4)Ended earlyNCT04306510
What this trial is testing

Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN

Who this might be right for
Hereditary Transthyretin Amyloidosis With Polyneuropthy
Akcea Therapeutics 8
Large-scale testing (Phase 3)WithdrawnNCT04882735
What this trial is testing

Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Who this might be right for
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eidos Therapeutics, a BridgeBio company
Large-scale testing (Phase 3)Study completedNCT01960348
What this trial is testing

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Who this might be right for
TTR-mediated AmyloidosisAmyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more
Alnylam Pharmaceuticals 225
Not applicableActive Not RecruitingNCT05873868
What this trial is testing

Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran

Who this might be right for
Transthyretin AmyloidosisAmyloidosis, Hereditary
Rennes University Hospital 20
Large-scale testing (Phase 3)Study completedNCT02319005
What this trial is testing

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Who this might be right for
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)Amyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more
Alnylam Pharmaceuticals 206
Not applicableStudy completedNCT04201418
What this trial is testing

Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Who this might be right for
Hereditary Transthyretin-mediated (ATTRv) AmyloidosisPolyneuropathy
Alnylam Pharmaceuticals 67
Large-scale testing (Phase 3)Looking for participantsNCT07223203
What this trial is testing

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Who this might be right for
Hereditary Transthyretin-Mediated Amyloidosis With PolyneuropathyhATTR-PN
Alnylam Pharmaceuticals 125
Large-scale testing (Phase 3)WithdrawnNCT04418024
What this trial is testing

Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy

Who this might be right for
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eidos Therapeutics, a BridgeBio company
Not applicableLooking for participantsNCT06465810
What this trial is testing

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

Who this might be right for
Transthyretin AmyloidosisATTR-CMATTRv-PN+3 more
AstraZeneca 1,850