A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects With Light Chain (AL) Amyloidosis

A Phase 3 study to evaluate the efficacy and safety of birtamimab plus standard of care compared to placebo plus standard of care in Mayo Stage IV patients with AL amyloidosis.

开始日期2021-08-30

申办/合作机构

Prothena Biosciences Ltd.

EUCTR2021-000037-14-ES / Unknown status临床3期

开始日期2021-07-01

申办/合作机构

Prothena Biosciences Ltd.

EUCTR2016-000489-50-IT / Unknown status临床2期IIT

A Phase II, Single Arm, Open Label, Efficacy and Safety Study of NEOD001 in Subjects with Light Chain (AL) Amyloidosis with Hepatic Involvement - ---

开始日期2017-10-03

申办/合作机构-

100 项与 SAA1 x 淀粉样蛋白轻链相关的临床结果

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100 项与 SAA1 x 淀粉样蛋白轻链相关的转化医学

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0 项与 SAA1 x 淀粉样蛋白轻链相关的专利（医药）

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项与 SAA1 x 淀粉样蛋白轻链相关的文献（医药）

2020-09-01·Protoplasma3区 · 生物学

Renal amyloidosis: an update on diagnosis and pathogenesis

3区 · 生物学

Review

作者: Gupta, Nimisha ; Kaur, Harshdeep ; Wajid, Saima

Amyloidosis is a diverse group of protein conformational disorder which is caused by accumulation and deposition of insoluble protein fibrils in vital tissues or organs, instigating organ dysfunction. Renal amyloidosis is characterized by the acellular Congo red-positive pathologic deposition of amyloid fibrils within glomeruli and/or the interstitium. It is generally composed of serum amyloid A-related protein or an immunoglobulin light chain; other rare forms lysozyme, gelsolin, fibrinogen alpha chain, transthyretin, apolipoproteins AI/AII/AIV/CII/CIII; and the recently identified form ALECT2. This disease typically manifests with heavy proteinuria, nephrotic syndrome, and finally progression to end-stage renal failure. Early diagnosis of renal amyloidosis is arduous as its symptoms appear in later stages with prominent amyloid deposition. The identification of the correct type of amyloidosis is quite troublesome as it can be confused with another related form. Therefore, the exact typing of amyloid is essential for prognosis, treatment, and correct management of renal amyloidosis. The emanation of new techniques of proteomic analysis, for instance, mass spectroscopy/laser microdissection, has provided greater accuracy in amyloid typing. This in-depth review emphasizes on the clinical features, renal pathological findings, and diagnosis of the AL and non-AL forms of renal amyloidosis.

2003-02-27·Transplantation2区 · 医学

Orthotopic liver transplantation for hereditary fibrinogen amyloidosis

2区 · 医学

Article

作者: R. Wiesner ; M. Gertz ; T. Schwab ; M. Benson ; T. Uemichi ; J. Björnsson ; S. Zeldenrust

Systemic amyloidosis results from the deposition of insoluble protein fibrils in various organs and tissues. To date, several different proteins have been associated with amyloid fibril formation, including immunoglobulin light chain, serum amyloid A protein, and transthyretin. Recent reports have shown that variant fibrinogen chains can form amyloid in certain kindreds. Hepatic transplantation has previously been reported in the treatment of hereditary amyloidosis associated with variant transthyretin proteins, which are mainly synthesized in the liver. This article reports the first use and long-term follow-up of combined hepatic and renal transplantation in the successful treatment of two patients with hereditary fibrinogen amyloidosis. Both patients experienced sustained improvement in renal function and nutritional status at 61/2 years and 28 months of follow-up, respectively. Orthotopic liver transplantation is effective and potentially curative treatment of hereditary fibrinogen amyloidosis.