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64 results for “light chain amyloidosis”
Neurofilament light chain (NfL) as a biomarker of hereditary transthyretin-mediated amyloidosis
<p>To identify changes in the proteome associated with onset and progression of ATTRv amyloidosis, we performed an observational, case-controlled study which compared proteomes of patients with ATTRv amyloidosis and healthy controls.</p> <p>Plasma levels of >1,000 proteins were measured in patients with ATTRv amyloidosis with polyneuropathy who received either placebo or patisiran in the APOLLO study and in healthy controls. The impact of patisiran on the time profile of each protein was determined by linear mixed model at 0, 9, and 18 months. Neurofilament light chain (NfL) was further assessed using an orthogonal quantitative approach.</p> <p>Levels of 66 proteins were significantly changed with patisiran vs placebo, with NfL change most significant (p < 10<sup>−20</sup>). Analysis of changes in protein levels demonstrated that the proteome of patisiran-treated patients trended toward healthy controls at 18 months. Healthy controls' NfL levels were 4-fold lower than in patients with ATTRv amyloidosis with polyneuropathy (16.3 vs 69.4 pg/mL, effect: −53.1 pg/mL, 95% CI [–60.5 to −45.9]). NfL levels at 18 months increased with placebo (99.5 vs 63.2 pg/mL, 36.3 pg/mL, [16.5–56.1]) and decreased with patisiran treatment (48.8 vs 72.1 pg/mL, −23.3 pg/mL, [–33.4 to −13.1]) from baseline. At 18 months, improvement in modified Neuropathy Impairment Score +7 following patisiran significantly correlated with reduced NfL (R = 0.43, [0.29–0.55]).</p> <p>Findings suggest NfL may serve as a biomarker of nerve damage and polyneuropathy in ATTRv amyloidosis, may enable earlier diagnosis of patients with ATTRv amyloidosis, and facilitate monitoring of disease progression.</p> <p>This study provides Class III evidence that NfL levels may enable earlier diagnosis of polyneuropathy in patients with ATTRv amyloidosis and facilitate monitoring of disease progression.</p>
The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis
ClinicalTrials.gov study NCT02632786. IPD Sharing: Not stated. Countries: 10. Publications: 0.
Bortezomib, Melphalan, and Dexamethasone in Treating Patients With Primary Amyloidosis or Light Chain Deposition Disease
ClinicalTrials.gov study NCT00520767. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Melphalan and Dexamethasone With or Without Bortezomib in Treating Patients With Previously Untreated Systemic Light-Chain Amyloidosis
ClinicalTrials.gov study NCT01078454. IPD Sharing: Not stated. Countries: 1. Publications: 0.
A Study to Investigate Safety and Efficacy With SAR445514 in Participants With Relapsed/Refractory Multiple Myeloma (RRMM) and Relapsed/Refractory Light-chain Amyloidosis (RRLCA)
ClinicalTrials.gov study NCT05839626. IPD Sharing: YES. Countries: 7. Publications: 0.
Bortezomib, Cyclophosphamide, and Dexamethasone in Treating Patients With Primary Systemic Light Chain Amyloidosis
ClinicalTrials.gov study NCT01072773. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Study in Subjects With Light Chain (AL) Amyloidosis
ClinicalTrials.gov study NCT03154047. IPD Sharing: UNDECIDED. Countries: 10. Publications: 0.
A Trial to Learn if Linvoseltamab is Safe and Works in Adults With Relapsed or Refractory Systemic Light Chain Amyloidosis (AL Amyloidosis)
ClinicalTrials.gov study NCT06292780. IPD Sharing: YES. Countries: 4. Publications: 0.
Isatuximab and Bendamustine in Systemic Light Chain Amyloidosis
ClinicalTrials.gov study NCT04943302. IPD Sharing: NO. Countries: 0. Publications: 33.
Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
ClinicalTrials.gov study NCT02613182. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Neurofilament light chain (NfL) as a biomarker of hereditary transthyretin-mediated amyloidosis
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Tumor cells in light-chain amyloidosis and multiple myeloma show different transcriptional rewiring of the normal plasma cell development [bulk RNA-seq]
GEO Series GSE175384. Homo sapiens. 111 samples. Type: Expression profiling by high throughput sequencing.
Phenotypic, transcriptomic and genomic characterization of clonal plasma cells in light chain amyloidosis [Copy number analysis]
GEO Series GSE73041. Homo sapiens. 18 samples. Type: Genome variation profiling by SNP array.
Daratumumab Maintenance Therapy for Improving Survival in Patients With Light Chain Amyloidosis, EMILIA Trial
ClinicalTrials.gov study NCT05898646. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Study Evaluating the Efficacy and Safety of CM336 Injection in the Treatment of Light-Chain Amyloidosis
ClinicalTrials.gov study NCT07039578. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Molecular Imaging of the Underlying Mechanism of Cardiotoxicity in Patients With Light Chain Amyloidosis Using PET/CT
ClinicalTrials.gov study NCT04105634. IPD Sharing: Not stated. Countries: 1. Publications: 0.
A EUropean REgistry and Sample Sharing networK to Promote the Diagnosis and Management of Light Chain Amyloidosis (EUREKA)
ClinicalTrials.gov study NCT06205953. IPD Sharing: NO. Countries: 6. Publications: 0.
Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis
ClinicalTrials.gov study NCT00017680. IPD Sharing: Not stated. Countries: 1. Publications: 0.
A Safety Study of Carfilzomib in Patients With Previously-Treated Systemic Light Chain Amyloidosis
ClinicalTrials.gov study NCT01789242. IPD Sharing: Not stated. Countries: 1. Publications: 0.
A Clinical Study on Minimal Residual Disease in Patients With Systemic Light Chain Amyloidosis
ClinicalTrials.gov study NCT07215494. IPD Sharing: UNDECIDED. Countries: 1. Publications: 0.
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