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Amyloid Neuropathies clinical trials

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NCT ID: NCT06414746 Recruiting - Clinical trials for Carpal Tunnel Syndrome

Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia

LOCUS
Start date: December 29, 2023
Phase:
Study type: Observational

This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent surgery for CTS to assess their probability of having ATTR PN.

NCT ID: NCT05697861 Recruiting - Clinical trials for Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Start date: July 10, 2023
Phase:
Study type: Observational

This is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.

NCT ID: NCT05087953 Not yet recruiting - Clinical trials for Autonomic Nervous System Disease

Autonomic Evaluation of Patients With Hereditary Amyloidotic Cardiomyopathy: Hereditary Amyloidotic Heart Disease

Start date: January 2022
Phase:
Study type: Observational

Transthyretin amyloidosis exhibits a variety of possible phenotypes, the hereditary neurological form being the most commonly found and studied (familial amyloidotic polyneuropathy or FAP), which can present from oligosymptomatic patients to patients with peripheral sensorimotor polyneuropathy of varying degrees and dysautonomia. Although a specific mutation usually causes a specific phenotype, that is, with a predominantly cardiac or preferential neurological profile, with the increase in the number of diagnosed cases, an overlapping of clinical presentations has been observed. The assessment of the autonomic profile in individuals with familial amyloidotic cardiomyopathy (FAC) has not been well studied, and it is not known whether patients with an exclusively cardiac profile of the disease may present dysautonomia or whether even mutation carriers without cardiac involvement may exhibit it. In this study, the autonomic profiles of patients with familial amyloidotic heart disease will be compared with the profiles of patients who have mutations but without established heart disease and healthy individuals (control group).

NCT ID: NCT05071300 Recruiting - Clinical trials for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

A Study to 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

Start date: January 4, 2022
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the safety and tolerability of extended dosing with Eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).

NCT ID: NCT04985734 Not yet recruiting - Heart Failure Clinical Trials

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) in Patients With Idiopathic Peripheral Neuropathy

Start date: October 1, 2021
Phase: N/A
Study type: Interventional

The goal of the present study is to determine the occurrence of wild-type and hereditary transthyretin amyloidosis cardiomyopathy among patients with the diagnosis of idiopathic peripheral neuropathy in the setting of a state-of-the-art diagnostic work-up; the investigators believe that the identification of patients with ATTR-CM in this setting can contribute to the early diagnosis of a largely underrecognized condition and, therefore, offer conditions to timely initiation of appropriate therapy with impact on prognosis of patients.

NCT ID: NCT04963985 Recruiting - Clinical trials for Transthyretin Amyloidosis

The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients

Start date: June 1, 2021
Phase: Phase 4
Study type: Interventional

Transthyretin amyloid polyneuropathy (ATTR-PN) is a fatal illness resulting from autosomal dominantly inherited single-point mutations on the transthyretin gene. Tafamidis is a specific stabilizer of both variant and wild-type TTR. Tafamidis binds to TTR at the thyroxine binding sites and inhibits TTR tetramer dissociation, the rate limiting step in the amyloidogenic process. The result disrupts the amyloid cascade and fibril formation and interrupts disease progression. This study provides the basis for the study of the effect of tafamidis on the stability of transthyretin and its safety, tolerance and efficacy in patients with transthyretin amyloid polyneuropathy.

NCT ID: NCT04882735 Withdrawn - Clinical trials for Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

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

Start date: September 2021
Phase: Phase 3
Study type: Interventional

Phase 3 efficacy and safety of acoramidis in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)

NCT ID: NCT04828993 Completed - Clinical trials for Transthyretin Amyloid Polyneuropathy (ATTR-PN)

The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

Start date: April 28, 2021
Phase: Phase 4
Study type: Interventional

This is a single-arm, open-label, multicenter study designed to evaluate the efficacy, safety, tolerability as well as pharmacodynamics of tafamidis meglumine in ATTR-PN participants in China. Approximately 10-15 participants are planned to be enrolled. All enrolled participants will receive oral tafamidis meglumine 20 mg soft capsules once daily for 72 weeks (18 months).

NCT ID: NCT04601051 Active, not recruiting - Clinical trials for Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

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)

Start date: November 5, 2020
Phase: Phase 1
Study type: Interventional

This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM) or wild type cardiomyopathy (ATTRwt-CM)

NCT ID: NCT04418024 Withdrawn - Clinical trials for Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Polyneurophathy

ATTRibute-PN
Start date: October 21, 2020
Phase: Phase 3
Study type: Interventional

See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)