Clinical Trials

The purpose of this long-term survey is to better understand and characterize the natural history of  transthyretin-associated amyloidoses (including ATTR-polyneuropathy, ATTR-cardiomyopathy, and wild-type ATTR-CM) by studying a large and diverse patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.

This phase II trial studies how well isatuximab works in treating patients with primary amyloidosis that has come back or does not respond to treatment. Monoclonal antibodies, such as isatuximab, may interfere with the ability of cancer cells to grow and spread.

The purpose of this study is to evaluate the efficacy and safety of daratumumab plus cyclophosphamide, bortezomib and dexamethasone (CyBorD) compared with CyBorD alone in treatment of newly diagnosed amyloid light chain (AL) amyloidosis participants.

The purpose of this study is to evaluate the effectiveness and safety of birtamimab plus standard of care compared to placebo plus standard of care in patients with AL amyloidosis in Mayo Stage IV.

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

For more information, please visit https://www.cardio-ttransform.com/.

The purpose of this study is to evaluate the efficacy and safety of Coagadex in treating active bleeds and to manage peri-operative bleeding in participants with acquired factor X deficiency associated with AL amyloidosis.

The purpose of this study is to evaluate the usefulness of collecting health related quality of life data on newly diagnosed AL amyloidosis patients.

The purpose of this study is to evaluate the effectiveness and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis).

The objectives of this study are to describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients, to characterize the safety and effectiveness of patisiran as part of routine clinical practice in the real-world clinical setting, and to describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) mutation.

The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

The purpose of this study is to assess safety and tolerability of Acoramidis in participants with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM).

The purpose of this study compares the effect of adding a stem cell transplant with melphalan after completing chemotherapy with daratumumab, cyclophosphamide, bortezomib and dexamethasone versus chemotherapy with Dara-VCD alone for treating patients with newly diagnosed amyloid light chain amyloidosis. 

The aims of this prospective observational study will be to include all patients with systemic AL amyloidosis regardless of age or disease severity, in order to convey a ‘real-world’ picture of the disease, its response to myeloma-type chemotherapy regimens, associated toxicity and outcomes in terms of amyloidotic organ function, quality of life (QoL) and survival.

To evaluate the effectiveness of Patisiran on ambulatory status in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation.

The purpose of this study is to characterize the natural history of leukocyte chemotactic factor 2 amyloidotic (ALECT2) disease. In this observational study, participants with ALECT2 disease will be enrolled. Participants who have already reached end-stage renal disease (ESRD) will provide retrospective chart review data and biological specimens at baseline only. Other participants, in addition to retrospective chart review, will be followed prospectively.

The purpose of this study is to perform multi-organ magnetic resonance elastography (MRE) in subjects with AL amyloidosis to determine feasibility of multi-organ MRE technique, and to perform multi-organ MRE in subjects with AL amyloidosis pre- and post-stem cell transplantation to obtain pilot data in patients who undergo stem cell transplantation for detection of changes post transplant.  

The primary objective of this study is to define the intracardiac flow imaging biomarkers in cardiac amyloidosis.  

The purpose of this study is to evaluate the effectiveness and safety of patisiran in participants with Transthyretin Amyloidosis With Cardiomyopathy.

This prospective, randomized, multicenter, double-blind, parallel group, placebo-controlled, dose-ranging study will evaluate the safety, tolerability, PK and PD of AG10 compared to placebo administered on a background of stable heart failure therapy. Screening and randomization will be followed by a 28-day blinded, placebo-controlled treatment period.

The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.

The purpose of this study is to evaluate vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy.

The objective of our study is to develop [11C]PIB PET as a new imaging biomarker for quantitative assessment of AL cardiac amyloidosis. The hypothesis is the degree of amyloid deposition in the myocardium and other organs in whole body could be detected and quantified by [11C]PIB PET imaging, which will diagnose cardiac amyloidosis early, differentiate AL cardiac amyloidosis from others, and monitor therapy response. 

The purpose of this study is to create a data collection and bioregistry of blood samples from Mayo Clinic patients with amyloidosis, suspected amyloidosis, and family members of patients with amyloidosis. This information will be available for future research about this spectrum of diseases.

The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.

The primary purpose of this study is to assess arrhythmic events using the implantable cardiac monitor, Biomonitor 3, in subjects presenting to the clinic with TTR-wt cardiac amyloidosis.

The purpose of this study is to evaluate different heart imaging techniques to detect amyloidosis involving the heart. We are doing this research study to characterize the differences between types of amyloid and other diseases that mimic the appearance of amyloid involving the heart by using several imaging studies.

The purpose of this study with the Health Authorities from the United States (US), Canada and Europe, is to collect long-term safety information (including any potential side effects) for the drug TEGSEDI in patients with hATTR-PN under real-world conditions.

The purpose of this study is to evaluate the efficacy of I-124 evuzamitide for diagnosing cardiac amyloidosis.

The purpose of this Phase 3, open label, single dose imaging study is to evaluate the efficacy and safety of I-124 evuzamitide (radioactive dye) for diagnosing Cardiac Amyloidosis in participants with suspected Cardiac Amyloidosis. The imaging test that will be used in this study is a Positron Emission Tomography Computed Tomography (PET/CT) scan.

The purpose of this study is to evaluate the safety and tolerability of extended dosing with Eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).

The primary objective of this study is to determine the prevalence of transthyretin cardiac amyloidosis (TTR-CA) in a community-based cohort of consecutive heart failure with preserved ejection fraction (HFpEF) patients with increased LV wall thickness using 99mTc-PYP single-photon positive emission computed tomography with computed tomography (SPECT/CT). As a secondary (exploratory) objective, we seek to establish and validate novel biomarker assays to screen for TTR-CA using blood and urine samples collected from study participants.

The purpose of this study is to use magnetic resonance elastography to assess heart muscle stiffness in patients with cardiac amloidosis compared to normal subjects.

The purpose of this study is to determine if CAEL-101 and treatment for plasma cell dyscrasia improves overall survival in Mayo stage IIIb AL amyloidosis patients who are treatment naïve compared to treatment for plasma cell dyscrasia alone, and to evaluate the safety and tolerability of CAEL-101 in combination with treatment for plasma cell dyscrasia.

The purpose of this study is to determine if CAEL-101 improves the overall survival in patients with cardiac AL Amyloidosis.

This research is being done to help us better understand the clinical characteristics and to identify gene(s) and/or protein(s) causing these diseases. The study might contribute to the understanding of these disorders.

The incidence of monoclonal gammopathies and progression to multiple myeloma increases with aging. In order to separate the characteristics of pre-malignant/malignant plasma cells and their microenvironment from normal aging, patient samples and functional assessments need to be evaluated in comparison to healthy, aged participants. With this study, we will recruit healthy, aged partipcants to create a normal hematologic aging resource.

The purpose of this study is to evaluate the effectiveness and safety of AKCEA-TTR-LRx after administration for 65 weeks to patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), as compared to the NEURO-TTR trial (NCT01737398).

For more information, please visit http://www.neuro-ttransform.com/.

The purpose of this study is test the effectiveness and safety of NNC6019-0001 to determine if it can reduce the symptoms of a heart disease due to TTR (Transthyretin ) amyloidosis, such as heart failure.  

The purpose of this study is to evaluate the safety and tolerability, identify dose-limiting toxicities (DLT) and the maximum tolerated dose (MTD and recommended phase II dose (RP2D of APG2575 in combination with Pomalidomide/dexamethasone (Pd) in patients with relapsed/refractory (R/R) multiple myeloma (MM), or immunoglobulin light chain (AL) amyloidosis, and to evaluate the safety and tolerability, identify dose-limiting toxicities (DLT) and the maximum tolerated dose (MTD and recommended phase II dose (RP2D of APG2575 in combination with Daratumumab/Lenalidomide/dexamethasone (DRd) in patients with relapsed/refractory (R/R) multiple myeloma (MM).

The purpose of this study is to improve heart transplantation and clinical outcomes for transplant patients. The study will enroll individuals who are scheduled for heart surgery where samples from their heart will be collected as part of the normal surgical procedure.

The purpose of this study is to pilot the patient appointment companion (PAC) tool in use with patients with amyloidosis and multiple myeloma (MM). Patients with hematologic malignancies have unique information and communication needs compared to those with solid tumors. The internet has shifted the dynamics of the patient–doctor relationship, toward one of more mutual participation whereby power and responsibility are shared.  The PAC was designed as a patient-centered, user-friendly platform that aids patients’ preparation for their visits with clinicians. PAC provides an online tool that empowers patients to direct their treatment preferences and care alongside their physician teams per the provisions ...

The purpose of this study is to determine the prevalence of TTR-CA in a community-based cohort of moderate and severe aortic stenosis patients using 99mTc-PYP single-photon positive emission computed tomography with computed tomography (SPECT/CT).

The purpose of this study is to evaluate the magnitude of financial toxicity in newly diagnosed and relapsed multiple myeloma (MM) and amyloidosis (AL) patients.

This clinical trial is studying long-term follow-up in patients who are or have participated in Children's Oncology Group studies. Developing a way to track patients enrolled in Children's Oncology Group studies will help doctors gather long-term follow-up information and may help the study of cancer in the future.

The purpose of this study is to better understand the role of survey assessment in symptom evaluation of patient with chronic hematologic malignancies.

The purpose of this study is to provide a large database and platform for prospective sub-studies and eventually develop additional collaborations with a platform for clinical studies and trials following the initial pilot phase.