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A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIb AL Amyloidosis
Jacksonville, Fla.,
Rochester, Minn.,
Scottsdale/Phoenix, Ariz.
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.
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A Pilot to Assess the Feasibility of Collecting Longitudinal Health-Related Quality of Life in Light Chain Amyloidosis
Rochester, Minn.
The purpose of this study is to evaluate the usefulness of collecting health related quality of life data on newly diagnosed AL amyloidosis patients.
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A Prospective ‘Real World’ Study of Chemotherapy in Patients with Immunoglobulin Light (or Heavy) Chain Amyloidosis
Rochester, Minn.
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.
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ALN-TTR02-006: A Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who have Completed a Prior Patisiran Clinical Study
Rochester, Minn.
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.
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APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)
Rochester, Minn.
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis
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Artificial Intelligence Enhanced ECG to detect Cardiac Amyloidosis:
Protocol for a Pragmatic Cluster Randomized Clinical Trial (PREDICT-AMY) (PREDICT-AMY)
Rochester, Minn.,
Jacksonville, Fla.,
Eau Claire, Wis.,
Scottsdale/Phoenix, Ariz.
This pragmatic trial will be the first study to prospectively evaluate the use of the AI ECG dashboard along with an augmented report in everyday practice. The findings will also guide future implementation strategies and inform the translation of many of the current and future AI algorithms into the clinical setting. The participants are the providers/clinicians. Basic demographic information about the providers will be collected as well as their reactions to the trial based educational guidance supplied to them. Patient health information will be collected on their patients using a HIPPA waiver. There will be no patient contact. We will reveiew 29,000 ekgs.
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C16011 - A Phase 3, Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of Dexamethasone Plus MLN9708 or Physician's Choice of Treatment Administered to Patients With Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis
Rochester, Minn.
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.
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CAEL101-302 A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment Naïve Patients With Mayo Stage IIIa AL Amyloidosis (CAEL101-302)
Rochester, Minn.,
Jacksonville, Fla.,
Scottsdale/Phoenix, Ariz.
The purpose of this study is to determine if CAEL-101 improves the overall survival in patients with cardiac AL Amyloidosis.
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Gut Microbiome Studies in Patients with POEMS Syndrome and Other Plasma Cell Disorders (GMB POEMS)
Rochester, Minn.
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 hypothesis that in POEMS patients the gut microbiome signature will differ between active disease (at diagnosis) and inactive disease (in remission), and the gut microbiome in POEMS patients will be different from patients with other plasma cell disorders and healthy controls
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MC1682, Ixazomib, Lenalidomide, and Dexamethasone for Patients With POEMS Syndrome
Rochester, Minn.
This pilot clinical trial studies how well ixazomib citrate, lenalidomide, and dexamethasone work in treating patients with polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome. Ixazomib citrate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as lenalidomide and dexamethasone, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ixazomib citrate, lenalidomide, and dexamethasone may work better in treating patients with POEMS syndrome.
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THAOS (B3461001): A Global, Multi-Center, Longitudinal, Observational Survey of Patients with Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis (THAOS)
Rochester, Minn.
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.
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