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 is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.

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 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 vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran or the reference comparator patisiran during the Treatment Period followed by a Treatment Extension Period during which all participants in the patisiran group will switch to vutrisiran. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the co-primary and most other efficacy endpoints.

The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated 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 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 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 three-stage stage study is to identify the recommended phase 2 dose (RP2D) of STI-6129 by assessing the safety, preliminary effectiveness and pharmacokinetics of this anti-CD38-Duostatin 5.2 antibody-drug conjugate (ADC) for the treatment of relapsed or refractory systemic AL amyloidosis. The patients that will be treated with STI-6129 in this trial are relapsed or refractory systemic AL amyloidosis patients who have received prior lines of treatment.

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.

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 primary aim of this study will be to examine the diagnostic utility of 18-F Florbetapir PET/MR (Positron Emission Tomography/Magnetic Resonance) in imaging patients with pathologically-confirmed systemic amyloidosis involving the peripheral nerves and compare these results to non-amyloid diseased controls.

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 is to provide additional treatment with NEOD001 and evaluate long term safety and tolerability for patients wih light chain amyloidosis who completed the previous study NEOD001-001.

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

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.

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.  

A Phase 1, open-label, 3+3 dose escalation component to determine the safety, tolerability, PK, PD, and MTD of IV PRX004 when given as a single agent to up to 36 subjects with hATTR amyloidosis An expansion component in up to 2 anticipated PRX004 RP2D cohorts selected from dose escalation (up to an additional 3 subjects in each expanded cohort for up to 6 evaluable subjects total in each expanded cohort; an evaluable subject is defined as a subject who has completed the first 28 days following the first administration of PRX004 or who discontinued within 28 days due to study ...

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 evaluate vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy.

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

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 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 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 the effectiveness and safety of patisiran in participants with Transthyretin Amyloidosis With Cardiomyopathy.

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

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 evaluate the safety and effectiveness of long-term dosing with (ALN-TTR02) patisiran in patients who have familial transthyretin mediated amyloidosis with polyneuropathy, and have completed a prior clinical study on patisiran.

This is a phase 3, randomized, controlled, open-label, multicenter study of the oral formulation of dexamethasone plus IXAZOMIB compared with treatment chosen by the investigator from a prespecified list of regimens available in clinical practice. Treatment options will include: dexamethasone alone, dexamethasone plus an alkylating agent (melphalan or cyclophosphamide), or dexamethasone plus an immunomodulatory drug (IMiD, thalidomide or lenalidomide) in patients with relapsed or refractory AL amyloidosis. Crossover to the investigational treatment arm is not permitted during participation in this study.

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 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.

Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose.

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 use magnetic resonance elastography to assess heart muscle stiffness in patients with cardiac amloidosis compared to normal subjects.

This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached.

The purpose of this study is to

The primary objective of this study is to evaluate the long-term safety and tolerability of AG10 administered to adult patients with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM) in patients who have completed the study AG10-201.

The purpose of this study is to evaluate the efficacy and safety of ISIS-TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy

This study evaluates the safety and tolerability of extended dosing with ISIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).

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.

Evaluating the efficacy of ION-682884 after administration for 65 weeks, as compared to the historical control of the placebo cohort (inotersen) in the NEURO-TTR trial, based on the change from Baseline in serum TTR concentration, mNIS+7 and in the Norfolk Quality of Life.

Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) in patients with hATTR. To evaluate the efficacy of ION-682884, as compared to the placebo cohort in the NEURO-TTR  trial, based on the change from Baseline in the following measures:

• Neuropathy Symptom and Change Score (NSC)
• Physical component summary (PCS) score of 36-Item Short ...

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 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/.

This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

The purpose of this study is to assess the effectiveness and safety of NEOD001 administered intravenously in adults with light chain amyloidosis who have had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.

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 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 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.

The characteristics and role of gut microbiome rare plasma cell disorders- POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, skin changes) and amyloid light-chain (AL) amyloidosis, have not been explored; and their pathophysiology is quite elusive. To help understand rare plasma cell disorders and its association with gut microbiome, we will study the stool samples of newly diagnosed POEMS patients and AL amyloidosis and compare it with patients with newly diagnosed monoclonal gammopathy of undetermined significance and multiple myeloma as well as healthy controls. Moreover, gut microbiome in newly diagnosed POEMS patients will be compared to POEMS patients in remission. It is our overall ...

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.