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 — Transthyretin-mediated Amyloidosis With Cardiomyopathy
Status: No longer available
http://inclinicaltrials.com/transthyretin-mediated-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/
An Open-label Single Center, Single Patient Study of an Experimental Antisense Oligonucleotide (ASO) Treatment in AA Amyloidosis
The purpose of this study is to assess the safety and efficacy of a subcutaneous injection of nL-SAA1-01in a patient with AA Amyloidosis.
NCT06397001 — AA Amyloidosis
Status: Active, not recruiting
http://inclinicaltrials.com/aa-amyloidosis/NCT06397001/
Daratumumab and Dexamethasone Combined With Pomalidomide (DPD) or ASCT in the Treatment of Newly Diagnosed Systemic Light Chain Amyloidosis (AL Amyloidosis): a Prospective, Single Center Clinical Trial
This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen, and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen, and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment. The purpose of this study is to observe the efficacy and safety of Daratumumab, in the treatment of newly diagnosed systemic AL amyloidosis.
NCT06376214 — Light Chain (AL) Amyloidosis
Status: Recruiting
http://inclinicaltrials.com/light-chain-al-amyloidosis/NCT06376214/
Screening for AL Amyloidosis in Smoldering Multiple Myeloma
In this multicenter study, we will recruit 400 patients 40 years of age or older at 15 centers with a diagnosis of smoldering multiple myeloma (SMM), a group of patients for whom standard of care is observation not treatment. The main goal of this study is to screen for the diagnosis of light-chain amyloidosis (AL) before the onset of symptomatic disease and to develop a training set for a likelihood algorithm.
NCT06365060 — Smoldering Multiple Myeloma
Status: Not yet recruiting
http://inclinicaltrials.com/smoldering-multiple-myeloma/NCT06365060/