Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) With Cardiomyopathy
The objective of the study is to assess the long-term safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy as assessed by a review of adverse events (AEs).
NCT05505838 — ATTR Amyloidosis With Cardiomyopathy
Status: No longer available
http://inclinicaltrials.com/attr-amyloidosis-with-cardiomyopathy/NCT05505838/
Cardiac Amyloidosis Registry Study - A Multi-Center, Longitudinal, Observational Survey of Patients With Cardiac Amyloidosis
This registry is a observational, multi-center study designed to collect data and analyze it retrospectively on patients with cardiac amyloidosis who have been evaluated and treated at major amyloid centers across the US and internationally between 1997 and 2025.
NCT05174338 — Amyloidosis, Immunoglobulin Light-chain
Status: Enrolling by invitation
http://inclinicaltrials.com/amyloidosis-immunoglobulin-light-chain/NCT05174338/
ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: - Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients - Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting - Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant
NCT04561518 — ATTR Amyloidosis
Status: Recruiting
http://inclinicaltrials.com/attr-amyloidosis/NCT04561518/
An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver transplant.
NCT03862807 — Transthyretin Amyloidosis
Status: Completed
http://inclinicaltrials.com/transthyretin-amyloidosis/NCT03862807/
HELIOS-A: 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)
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran subcutaneous (SC) injection once every 3 months (q3M) or the reference comparator patisiran intravenous (IV) injection once every 3 weeks (q3w) during the 18 month Treatment Period. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints during the 18 Month Treatment Period. Following the 18 Month Treatment Period, all participants will be randomized to receive vutrisiran SC injection once every 6 months (q6M) or q3M in the Randomized Treatment Extension (RTE) Period.
NCT03759379 — Transthyretin Amyloidosis
Status: Active, not recruiting
http://inclinicaltrials.com/transthyretin-amyloidosis/NCT03759379/
Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
NCT02939820 — Amyloidosis, Hereditary
Status: No longer available
http://inclinicaltrials.com/amyloidosis-hereditary/NCT02939820/
Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) 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)
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)
NCT04601051 — Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Status: Active, not recruiting
http://inclinicaltrials.com/transthyretin-related-attr-familial-amyloid-polyneuropathy/NCT04601051/
DapagliFLOzin in Renal AL Amyloidosis (FLORAL)
The goal of this clinical trial is to learn if an oral drug called dapagliflozin is safe and can reduce high protein levels in the urine of patients with renal amyloid light-chain (AL) amyloidosis using a decentralized study design. Participants will be: - screened for the trial via an online platform - contacted by study personal to obtain electronic consent - enrolled in the trial if eligible and consented - contacted by study personal for further instructions and directions - sent dapagliflozin oral medication (supplied by the site pharmacy) - followed up regularly with the study team via telemedicine or other online avenues - monitored using lab work, inquiries about side effects and assessment of protocol adherence at 1 month, 3 months and 6 months - continue treatment for 6 months
NCT06420167 — Renal AL Amyloidosis
Status: Not yet recruiting
http://inclinicaltrials.com/renal-al-amyloidosis/NCT06420167/
A Multicenter Observational Retrospective-prospective Study of Prevalence, Clinical Characteristics of Hereditary Transthyretin Amyloidosis Polyneuropathy in Russian Patients Undergoing Surgery for CTS in Real Clinical Practice
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.
NCT06414746 — Carpal Tunnel Syndrome
Status: Recruiting
http://inclinicaltrials.com/carpal-tunnel-syndrome/NCT06414746/
Exercise Training and Rehabilitation In Cardiac Amyloidosis: ERICA-Study
Notwithstanding the dramatic improvement associated with Tafamidis in Heart Failure (HF) due to wild-type transthyretin cardiac amyloidosis (ATTRwt-CA), remarkable morbidity and mortality still burden this disease. Exercise training (ET) is a first-line recommended treatment for unselected HF patients, whose effects on ATTRwt-CA form remain however unexplored. The investigators hereby present rationale and design of the Exercise training and Rehabilitation in Cardiac Amyloidosis (ERICA) study, whose aim is to determine whether a tailored, supervised ET program might improve exercise capacity in HF due to ATTRwt-CA. This interventional, controlled study will randomize ATTRwt-CA patients into a control group (C) and a primary training group (ET-1). After 12 weeks, patients in group C will be offered to undergo the same ET program (ET-2) for further 12 weeks, considering the last observation as baseline. Primary endpoint will be the distance obtained at the 6-minute walk test (6MWD) performed at baseline and after 12-weeks of treatment in pooled ET-1 and ET-2 groups compared to C. Quality of life, peak oxygen consumption, left and right heart architecture and function, natriuretic peptides will be secondary endpoints. This study will be the first testing the effects of ET in patients with ATTRwt-CA.
NCT06412432 — Cardiac Amyloidosis
Status: Enrolling by invitation
http://inclinicaltrials.com/cardiac-amyloidosis/NCT06412432/