Cardiac amyloidosis future or investigational therapies: Difference between revisions

Jump to navigation Jump to search
No edit summary
Line 9: Line 9:
Potential future treatment options targeting cardiac TTR amyloidosis include:
Potential future treatment options targeting cardiac TTR amyloidosis include:


* AG10: AG10 is a selective, oral TTR stabilizer which mimic a protective TTR mutation known as T119M. In a phase 2 clinical trial, AG10 was found to be safe and effective. This trial randomized 49 patients with mutant or wild-type TTR amyloid cardiomyopathy with NYHA class II to III symptoms to AG10 400 mg, 800 mg, or placebo twice daily for 28 days, and found the medication to be well tolerated and achieve near-complete stabilization of TTR.<ref>{{Cite web|url=https://www.sciencedirect.com/science/article/pii/S0735109719339208?via%3Dihub|title=Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy|last=Judge|first=Daniel P.|date=10/29/2019|website=|archive-url=|archive-date=|dead-url=|access-date=}}</ref> A phase 3 trial (ATTRIBUTE-CM) is ongoing (NCT03458130).
* AG10: AG10 is a selective, oral TTR stabilizer which mimics a protective TTR mutation known as T119M. In a phase 2 clinical trial, AG10 was found to be safe and effective. This trial randomized 49 patients with mutant or wild-type TTR amyloid cardiomyopathy with NYHA class II to III symptoms to AG10 400 mg, 800 mg, or placebo twice daily for 28 days, and found the medication to be well tolerated and achieve near-complete stabilization of TTR.<ref>{{Cite web|url=https://www.sciencedirect.com/science/article/pii/S0735109719339208?via%3Dihub|title=Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy|last=Judge|first=Daniel P.|date=10/29/2019|website=|archive-url=|archive-date=|dead-url=|access-date=}}</ref> A phase 3 trial (ATTRIBUTE-CM) is ongoing (NCT03458130).
* Patisiran: this is a siRNA molecule which has shown promise in patients with polyneuropathy from hereditary TTR amyloidosis. A cardiac subgroup study showed favorable biomarker results as well.<ref>{{Cite web|url=https://www.nejm.org/doi/10.1056/NEJMoa1716153?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%3dwww.ncbi.nlm.nih.gov|title=Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis|last=Adams|first=David|date=10/29/19|website=|archive-url=|archive-date=|dead-url=|access-date=}}</ref>
* Patisiran: this is a siRNA molecule which has shown promise in patients with polyneuropathy from hereditary TTR amyloidosis. A cardiac subgroup study showed favorable biomarker results as well.<ref>{{Cite web|url=https://www.nejm.org/doi/10.1056/NEJMoa1716153?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%3dwww.ncbi.nlm.nih.gov|title=Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis|last=Adams|first=David|date=10/29/19|website=|archive-url=|archive-date=|dead-url=|access-date=}}</ref>



Revision as of 18:10, 30 October 2019

Cardiac amyloidosis Microchapters

Home

Patient Information

Overview

Historical Perspective

Classification

Pathophysiology

Causes

Differentiating Cardiac Amyloidosis from other Diseases

Epidemiology and Demographics

Risk Factors

Screening

Natural History, Complications and Prognosis

Diagnosis

Diagnostic Study of Choice

History and Symptoms

Physical Examination

Laboratory Findings

Electrocardiogram

X-ray

Echocardiography and Ultrasound

CT scan

MRI

Other Imaging Findings

Other Diagnostic Studies

Treatment

Medical Therapy

Surgery

Primary Prevention

Secondary Prevention

Cost-Effectiveness of Therapy

Future or Investigational Therapies

Case Studies

Case #1

Cardiac amyloidosis future or investigational therapies On the Web

Most recent articles

Most cited articles

Review articles

CME Programs

Powerpoint slides

Images

American Roentgen Ray Society Images of Cardiac amyloidosis future or investigational therapies

All Images
X-rays
Echo & Ultrasound
CT Images
MRI

Ongoing Trials at Clinical Trials.gov

US National Guidelines Clearinghouse

NICE Guidance

FDA on Cardiac amyloidosis future or investigational therapies

CDC onCardiac amyloidosis future or investigational therapies

Cardiac amyloidosis future or investigational therapies in the news

Blogs on Cardiac amyloidosis future or investigational therapies

Directions to Hospitals Treating Cardiac amyloidosis

Risk calculators and risk factors for Cardiac amyloidosis future or investigational therapies

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Raviteja Guddeti, M.B.B.S. [2]; Aarti Narayan, M.B.B.S [3]

Overview

Several investigational products targeting ATTR amyloid are being studied in clinical trials. These include small interfering RNA (siRNA) molecules which reduce the production of the amyloid precursor misfolded protein and ATTR stabilization molecules.

Future or Investigational Therapies

Potential future treatment options targeting cardiac TTR amyloidosis include:

  • AG10: AG10 is a selective, oral TTR stabilizer which mimics a protective TTR mutation known as T119M. In a phase 2 clinical trial, AG10 was found to be safe and effective. This trial randomized 49 patients with mutant or wild-type TTR amyloid cardiomyopathy with NYHA class II to III symptoms to AG10 400 mg, 800 mg, or placebo twice daily for 28 days, and found the medication to be well tolerated and achieve near-complete stabilization of TTR.[1] A phase 3 trial (ATTRIBUTE-CM) is ongoing (NCT03458130).
  • Patisiran: this is a siRNA molecule which has shown promise in patients with polyneuropathy from hereditary TTR amyloidosis. A cardiac subgroup study showed favorable biomarker results as well.[2]

References

  1. Judge, Daniel P. (10/29/2019). "Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy". Check date values in: |date= (help)
  2. Adams, David (10/29/19). "Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis". Check date values in: |date= (help)


Template:WikiDoc Sources CME Category::Cardiology