Clinical trials: Hereditary Amyloidosis
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
- Trial name
- NCT06128629
- Testing
- NTLA-2001
- Where
- 132 sites in United States, Argentina and 24 more
- Volunteers
- 1,200 people
- Sponsor
- Intellia Therapeutics
Cardiac Amyloidosis
Individualized Exercise Training in Cardiac Amyloidosis
Numerous clinical trials have demonstrated that transthyretin (TTR)-directed medical therapies can reduce the rate of functional decline in patients with transthyretin cardiac amyloidosis (ATTR-CM).
- Trial name
- GET-FIT ATTR (NCT07835867)
- Testing
- Exercise Intervention, Physical Activity Handout
- Volunteers
- 60 people
- Sponsor
- Oregon Health and Science University
Amyloidosis, Amyloid Cardiomyopathy
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause…
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- NCT06563895
- Testing
- Acoramidis
- Where
- 104 sites in United States, Argentina and 22 more
- Volunteers
- 587 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors
This pilot trial studies different high-dose chemotherapy regimens with or without total-body irradiation (TBI) to compare how well they work when given before autologous stem cell transplant (ASCT) in treating patients with…
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT00536601
- Testing
- etoposide, cyclophosphamide, carmustine
- Where
- United States
- Volunteers
- 174 people
- Sponsor
- Roswell Park Cancer Institute
Amyloid Plaque, Beta-Amyloid
A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)
The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.
- Conditions
- Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease
- Trial name
- NCT05462106
- Testing
- ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a
- Where
- 26 sites in United States, Spain and 1 more
- Volunteers
- 304 people
- Sponsor
- AC Immune SA
AL Amyloidosis (AL), Cardiac Amyloidosis
Multimodal Radiomics Model (18F-FAPI PET/CT + CMR) for AL Cardiac Amyloidosis Prognosis
1. Goal of the Study: The goal of this prospective observational study is to develop and validate a novel, non-invasive method for predicting the prognosis of patients with light-chain cardiac amyloidosis (AL-CA).
- Trial name
- AL-CA (NCT07103863)
- Where
- China
- Volunteers
- 49 people
- Sponsor
- Beijing Anzhen Hospital
Heart Failure With Reduced Ejection Fraction, Heart Failure With Preserved Ejection Fraction
Administration of the SGLT-2 Inhibitor Dapagliflozin in the Patients With Amyloid Cardiomyopathy
Efficacy and safety of early administration of the SGLT-2 inhibitor dapagliflozin will be evaluated in patients with HF, regardless of LVEF, due to amyloid cardiomyopathy.
- Conditions
- Heart Failure With Reduced Ejection Fraction, Heart Failure With Preserved Ejection Fraction, Heart Failure With Mid Range Ejection Fraction, Amyloid Cardiomyopathy
- Trial name
- NCT05795400
- Testing
- Dapagliflozin 10mg Tab
- Where
- Russia
- Volunteers
- 50 people
- Sponsor
- National Medical Research Center for Cardiology, Ministry of Health of Russian Federation
HCM - Hypertrophic Cardiomyopathy, Anderson Fabry Disease
Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes
This observational study aims to evaluate myocardial perfusion abnormalities using quantitative and qualitative cardiac magnetic resonance (CMR) perfusion imaging in patients with hypertrophic cardiomyopathy (HCM) phenotypes…
- Conditions
- HCM - Hypertrophic Cardiomyopathy, Anderson Fabry Disease, Cardiac Magnetic Resonance Imaging, Amyloid Cardiomyopathy
- Trial name
- MyoPerf-HCM (NCT07382128)
- Where
- Italy
- Volunteers
- 250 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Amyloid Cardiomyopathy
Enavogliflozin for the Management of Patients With Amyloid CardiomyopaThy
This study aims to evaluate the safety and effectiveness of Enavogliflozin 0.3 mg, an SGLT2 inhibitor, in patients with amyloid cardiomyopathy.
- Trial name
- EMPACT (NCT07240844)
- Testing
- Enavogliflozin 0.3mg
- Where
- 2 sites in South Korea
- Volunteers
- 68 people
- Sponsor
- Seoul St. Mary's Hospital
Heart Failure
Heart Failure: Don't Forget the Role of Amyloidosis
Heart failure is defined as the inability of the heart to provide sufficient output to meet the needs of the body. It can occur in the course of a myocardial infarction, angina pectoris, hypertension, etc.
- Trial name
- TEAM-HF (NCT05501847)
- Testing
- Patient with no ventricular hypertrophy
- Where
- 2 sites in Guadeloupe, Martinique
- Volunteers
- 446 people
- Sponsor
- University Hospital Center of Martinique
Amyloidosis Cardiac, Virtual Reality
Virtual Reality Assisted Patient Empowerment: Diagnose ATTR-Amyloidosis And Start Treatment
ATTR amyloidosis is a serious condition with significant morbidity and mortality. In Germany, there are numerous unreported cases of untreated patients, and diagnosing and initiating treatment often requires multiple specialized…
- Trial name
- VRAP-DAAST (NCT05938218)
- Testing
- Intervention
- Where
- Germany
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Heinrich-Heine University, Duesseldorf
Observational
Progression of Renal Amyloidosis of FMF and Relation to Serum SAA Level
Purpose of this study is to determine whether keeping SAA on normal or near normal level will delay progression of renal failure in patients with amyloidosis secondary to FMF.
- Trial name
- NCT01168570
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- Sheba Medical Center
Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy
Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations
The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of…
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
- Trial name
- sTTRing (NCT07654855)
- Testing
- None ( observational study )
- Where
- 6 sites in United States, Canada and 4 more
- Volunteers
- 320 people
- Sponsor
- AstraZeneca