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Results for “Familial Amyloid Polyneuropathy (FAP)”

115 trials

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Showing 20 of 115 results

Early research (Phase 1)Study completedNCT04601051
What this trial is testing

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Who this might be right for
Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyTransthyretin-Related (ATTR) Familial Amyloid CardiomyopathyWild-Type Transthyretin Cardiac Amyloidosis
Intellia Therapeutics 72
Not applicableStudy completedNCT01432587
What this trial is testing

The Effect of Diflunisal on Familial Transthyretin Amyloidosis

Who this might be right for
Amyloidosis
Umeå University 55
Testing effectiveness (Phase 2)Active Not RecruitingNCT06260709
What this trial is testing

A Research Study to Look at Long-term Treatment With a Medicine Called NNC6019-0001 for People Who Have Heart Failure Due to Transthyretin Amyloidosis

Who this might be right for
Transthyretin Amyloid Cardiomyopathy (ATTR CM)
Novo Nordisk A/S 80
Not applicableActive Not RecruitingNCT06393465
What this trial is testing

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy

Who this might be right for
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Pfizer 50
Not applicableLooking for participantsNCT07205666
What this trial is testing

The Eplontersen Pregnancy and Lactation Outcomes Study

Who this might be right for
Transthyretin Amyloidosis
AstraZeneca 10
Large-scale testing (Phase 3)Study completedNCT03759379
What this trial is testing

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Who this might be right for
Amyloidosis, HereditaryTransthyretin Amyloidosis
Alnylam Pharmaceuticals 164
Not applicableLooking for participantsNCT06907186
What this trial is testing

An Interventional Pilot Study to Investigate the Feasibility and Acceptance of a Structured Psychological Support Program for Patients, Caregivers, and Presymptomatic Carriers in Hereditary Transthyretin Amyloidosis With Cardiomyopathy

Who this might be right for
Hereditary Transthyretin Amyloidosis
Fondazione IRCCS Policlinico San Matteo di Pavia 150
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)Active Not RecruitingNCT06128629
What this trial is testing

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Who this might be right for
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Intellia Therapeutics 1,200
Large-scale testing (Phase 3)Enrolling By InvitationNCT06679946
What this trial is testing

Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Who this might be right for
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Alnylam Pharmaceuticals 700
Not applicableLooking for participantsNCT04849754
What this trial is testing

Analysis of the Evolution of SUVmax by Quantitative Analysis Method of Bisphosphonate Scintigraphy

Who this might be right for
Cardiac Amyloidosis
GCS Ramsay Santé pour l'Enseignement et la Recherche 30
Not applicableLooking for participantsNCT07235462
What this trial is testing

Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting

Who this might be right for
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Bayer 2,000
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
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)Active Not RecruitingNCT04153149
What this trial is testing

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Who this might be right for
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Alnylam Pharmaceuticals 655
Not applicableStudy completedNCT03352089
What this trial is testing

Positron Emission Tomography / Magnetic Resonance Imaging in Aortic Stenosis

Who this might be right for
Aortic StenosisTransthyretin Amyloidosis
University of Edinburgh 22
Not applicableLooking for participantsNCT05444920
What this trial is testing

The Italian Transthyretin Amyloidosis Web-Network

Who this might be right for
ATTR Amyloidosis
Fondazione IRCCS Policlinico San Matteo di Pavia 1,000
Testing effectiveness (Phase 2)Study completedNCT00630864
What this trial is testing

The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis

Who this might be right for
Transthyretin-associated Amyloidosis With Polyneuropathy
Pfizer 21
Testing effectiveness (Phase 2)Looking for participantsNCT06539208
What this trial is testing

A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Who this might be right for
Transthyretin Amyloidosis PolyneuropathyTransthyrexin Amyloidosis Cardiomyopathy
YolTech Therapeutics Co., Ltd 31
Not applicableLooking for participantsNCT06101108
What this trial is testing

Early Diagnosis of Age-Linked CArdiac TransThyRetin Amyloidosis by Selective Screening in Spinal Stenosis Surgery

Who this might be right for
Transthyretin Amyloid Cardiomyopathy
Philippe Debonnaire 400
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