30 closest matches · ranked by relevance
Familial Amyloid Polyneuropathy
Primary: Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 — 45.3; 29.5 percentage of participants
Familial Amyloid Polyneuropathy · Familial Amyloidosis
Primary: Neurologic Impairment Score + 7 (NIS+7) — 8.2; 26.3; 6.2; 12.5 units on a scale — p=<0.001
Cardiac Amyloidosis
Primary: Mean Left Ventricular Myocardial F-18 Florbetapir SUVmean — 3.2; 1.7; 5.0 ratio
Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) · Transthyretin Cardiomyopathy (TTR-CM) · Familial Amyloid Cardiomyopathy
Primary: Demographical Characteristics of Participants — 20; 11; 39; 19 participants
Amyloidosis
Primary: Amyloid Cardiomyopathy: BNP — 883 pg/mL — p=0.035
Amyloidosis; Heart (Manifestation) · Senile Cardiac Amyloidosis
Primary: Changes in Strain Echocardiography — 9.1 percent LV shortening
Cardiac Amyloidosis
Primary: Rate of Cardiac Amyloidosis Diagnosis — 24 Participants
AL Amyloidosis
Primary: Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation — 4; 3; 6; 13…
Amyloid Cardiomyopathy, Transthyretin-Related
Primary: Prevalence of Transthyretin Cardiac Amyloidosis (ATTR-CA) in Cohort of Caribbean Hispanics and Blacks With Heart Failure (HF) — 7.8; 0 percentage of participants
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Primary: Percentage of Participants With Cardiac Fixation at the Radionuclide Bone Scintigraphy and/or Single Photon Emission Computed Tomography (SPECT): FAS1 — 32.0 Percentage…
ATTR-CM · TTR-CM
Primary: Percentage of Participants With Categories of Patient Global Assessment (PGA) for Follow-up Visit: Month 12 — 0; 16.1; 9.7; 51.6 percentage of participants
Primary Systemic Amyloidosis · Light Chain Deposition Disease
Primary: Complete Hematologic Response — 16 participants
Multiple Myeloma
Primary: Number of Patients Removed From Study Treatment Due to Toxicities — 31 participants
Multiple Myeloma
Primary: Number of Participants With Hematologic Response — 7 participants
Amyloidosis
Primary: Maximum Tolerated Dose — 2 participants with DLT
AL Amyloidosis
Primary: Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 2 Milligram Dose — 0 Participants
FAP · Familial Amyloid Polyneuropathy · TTR
Primary: Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66 — 4.16; 23.89 Scores on a Scale — p=0.00000004
Light-Chain Amyloidosis
Primary: Number of Participants With at Least One Treatment Emergent Adverse Event (TEAE) and Serious Adverse Event (SAE) — 6; 5; 5; 10 Participants
Multiple Myeloma · Plasma Cell Neoplasm
Primary: Tolerance for Human Immune Globulin Intravenous (IGIV), as Reflected by the Number and Severity of Toxicity Incidents Occurring in Ten Patients Receiving at Least One…
Familial Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 participants
Amorphous, Eosinophilic, and Acellular Deposit · Constipation · Diarrhea
Primary: Assess Efficacy by Confirmed Overall Hematologic Response — 77.1 percentage of paticipants
Multiple Myeloma
Primary: Number of Participants With Hematologic Response — 6 participants
Multiple Myeloma and Plasma Cell Neoplasm
Primary: Hematologic and Organ Response — 11; 7; 18 participants
Amyloidosis
Primary: Number of Participants With Disease Response — 20 Participants
AL Amyloidosis
Primary: Number of Participants With Cardiac Response and Non-Response — 26; 30; 40; 33 Participants — p=0.3190
Multiple Myeloma and Plasma Cell Neoplasm
Primary: Hematologic Response Rate — 55.9; 69.1 percentage of participants
Multiple Myeloma and Plasma Cell Neoplasm
Primary: Number of Participants Who Achieved a Confirmed Response Defined as a Complete Response (CR), Very Good Partial Response (VGPR) or Partial Response (PR) — 21 participants
AL Amyloidosis
Primary: Assess the Number of Patients Who Respond to Treatment — 5; 13; 2; 1 Participants
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) · Amyloidosis, Hereditary · Amyloid Neuropathies, Familial
Primary: 6 Minute Walk Distance (6-MWD)
Amyloidosis · Amyloid Cardiomyopathy · Transthyretin Amyloidosis
Primary: A Hierarchical Combination of All-Cause Mortality, Cumulative Frequency of CV-related Hospitalization, Change From Baseline in NT-proBNP and Change From Baseline in 6MWT…