Topics

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin‑Mediated Amyloid Cardiomyopathy (ATTR CM)

2019-10-28 13:57:10 | BioPortfolio

Summary

To evaluate the efficacy of AKCEA-TTR-LRx compared to placebo for 120 weeks in patients with ATTR-CM receiving available standard of care (SoC).

Description

This is a multicenter, double-blind study in up to 750 participants, who will be randomized to receive subcutaneous (SC) injections of either AKCEA-TTR-LRx or placebo once every 4 weeks. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A.

Study Design

Conditions

Transthyretin‑Mediated Amyloid Cardiomyopathy (ATTR CM)

Intervention

AKCEA-TTR-LRx, Placebo

Status

Not yet recruiting

Source

Ionis Pharmaceuticals, Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2019-10-28T13:57:10-0400

Clinical Trials [669 Associated Clinical Trials listed on BioPortfolio]

NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

To evaluate the efficacy and safety of AKCEA-TTR-LRx after administration for 65 weeks to patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), as compared to ...

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

Phase 3 efficacy and safety study to evaluate AG10 800 mg compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

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

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with c...

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

Phase 3 extension study to evaluate the safety of oral daily dosing of 20 mg or 80 mg tafamidis meglumine in subjects with either transthyretin genetic variants or wild-type transthyretin ...

PubMed Articles [6660 Associated PubMed Articles listed on BioPortfolio]

Estimating the Prevalence of Transthyretin Amyloid Cardiomyopathy in a Large In-Hospital Database in Japan.

Transthyretin amyloid cardiomyopathy (ATTR-CM)-a debilitating, fatal disease resulting from the deposition of transthyretin (TTR) amyloid fibrils-can be hereditary due to mutations in the TTR gene (AT...

Amyloid cardiomyopathy in a large integrated health care system.

Light Chain (AL) and transthyretin (ATTR) amyloidosis are the most common forms of amyloid cardiomyopathy. Population based studies describing the epidemiology and clinical features of amyloid cardiom...

Transthyretin cardiac amyloidosis: an update on diagnosis and treatment.

Transthyretin cardiac amyloidosis (ATTR-CA) demonstrates progressive, potentially fatal, and infiltrative cardiomyopathy caused by extracellular deposition of transthyretin-derived insoluble amyloid f...

Transthyretin Amyloidosis: Putting Myopathy On The Map.

While peripheral neuropathy and cardiomyopathy are well recognized manifestations of transthyretin (ATTR) amyloidosis, myopathy has been rarely reported.

Wild-type ATTR amyloidosis may be associated with unexpected death among the elderly.

Wild-type ATTR amyloidosis (ATTR-wt) is characterized by the accumulation of amyloid in the heart, leading to fatal heart failure and arrhythmia. In this study, we investigated the amyloid deposits in...

Medical and Biotech [MESH] Definitions

Inherited disorders of the peripheral nervous system associated with the deposition of AMYLOID in nerve tissue. The different clinical types based on symptoms correspond to the presence of a variety of mutations in several different proteins including transthyretin (PREALBUMIN); APOLIPOPROTEIN A-I; and GELSOLIN.

A nonproliferative disorder of the PLASMA CELL characterized by excessive production and misfolding of IMMUNOGLOBULIN LIGHT CHAINS that form insoluble amyloid fibrils (see AMYLOID DEPOSITS) in various tissues. Clinical features include LIVER FAILURE; MULTIPLE MYELOMA; NEPHROTIC SYNDROME; RESTRICTIVE CARDIOMYOPATHY, and neuropathies.

A group of diseases in which the dominant feature is the involvement of the CARDIAC MUSCLE itself. Cardiomyopathies are classified according to their predominant pathophysiological features (DILATED CARDIOMYOPATHY; HYPERTROPHIC CARDIOMYOPATHY; RESTRICTIVE CARDIOMYOPATHY) or their etiological/pathological factors (CARDIOMYOPATHY, ALCOHOLIC; ENDOCARDIAL FIBROELASTOSIS).

A type of extracellularly deposited substance composed of an amyloid protein and additional components including HEPARAN SULFATE PROTEOGLYCAN; LAMININ; COLLAGEN TYPE IV; SERUM AMYLOID P-COMPONENT; and APOLIPOPROTEINS E which together form characteristic amyloid fibrils. The core of amyloid fibrils is formed by the stacking of overlapping beta-pleated sheet domains of the amyloid protein. There are many different amyloid proteins that have been found forming the core of the fibrils in vivo. However, amyloid can be formed from any protein that exposes beta-pleated strand conformations during unfolding or refolding. A common characteristic of amyloid is the ability to bind such dyes as CONGO RED and thioflavine.

Proteins that form the core of amyloid fibrils. For example, the core of amyloid A is formed from amyloid A protein, also known as serum amyloid A protein or SAA protein.

More From BioPortfolio on "CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin‑Mediated Amyloid Cardiomyopathy (ATTR CM)"

Quick Search

Relevant Topic

Nutrition
Within medicine, nutrition (the study of food and the effect of its components on the body) has many different roles. Appropriate nutrition can help prevent certain diseases, or treat others. In critically ill patients, artificial feeding by tubes need t...


Searches Linking to this Trial