Collection of Samples and Clinical Data From Patients With Amyloid Diseases

Официальное название

Data, Tissue, Blood, and Urine Repository for Amyloid Diseases

Обзор

RATIONALE: Collecting and storing samples of blood, urine, tissue and data from patients with amyloid diseases to test in the laboratory may help the study of this disease in the future. PURPOSE: This research study is collecting samples from patients with amyloid diseases

Подробное описание

OBJECTIVES:

* To establish and maintain a database of clinical material (i.e., blood, urine, and tissue) and information on patients with amyloid diseases.

OUTLINE: Blood, urine, tissue, and bone marrow samples are collected during standard laboratory evaluations to maintain a repository of biospecimens in the Gerry Amyloid Research Laboratory, to permit the correlation of clinical results with measured biological events, and for future research studies. Bone marrow RNA samples are examined for immunoglobulin light-chain gene sequences and amino acids by polymerase chain reaction and positional cloning. Blood serum and urine samples are evaluated for amyloid protein stability by high-resolution calorimetry, isothermal-titration calorimetry, and far- and near-UV circular dichroism and fluorescence spectroscopy. Urine samples are also examined for post-translational modifications (e.g., glycosylation, sulfation, and cross-linking) to identify common features unique to amyloid proteins. Tissue samples are analyzed for biochemical and biophysical properties and for post-translational modifications in light chains.

Quality of life is assessed by the 36-Item Short Form Survey (SF-36).

Вмешательства

• Генная терапия clonality analysis
  analyze clonality
• Генная терапия polymerase chain reaction
  analyze polymerase chain reaction
• Генная терапия protein analysis
  analyze protein
• Другое biologic sample preservation procedure
  preserve biologic samples
• Другое laboratory biomarker analysis
  analyze laboratory biomarkers
• Процедура fluorescence spectroscopy
  perform fluorescence spectroscopy on specimens
• Процедура quality-of-life assessment
  assess quality of life at each follow-up for life

Первичные конечные точки

• Establish a repository to promote biochemical research [Срок оценки: life of study]

Вторичные конечные точки (1)

• Collection of clinical material and information [Срок оценки: life of study]

Критерии участия

Критерии включения

DISEASE CHARACTERISTICS:

• Diagnosis or suspicion of amyloidosis

PATIENT CHARACTERISTICS:

• Referred to the Amyloid Treatment and Research Center at Boston University Medical Center

PRIOR CONCURRENT THERAPY:

• Not specified

Критерии исключения

None

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Другое

Центры проведения

США · 1 центр

• Boston University Cancer Research Center — Boston

Публикации

• Ramireddy S, Prokaeva T, Chen H, Spencer B, Wong S, Dasari S, Dao L, McPhail ED, Sanchorawala V, Mendelson L. The Amyloidosis Intersection: Dual Amyloid Types in a Single Host. Eur J Haematol. 2025 Sep;115(3):287-298. doi: 10.1111/ejh.14429. Epub 2025 Jun 17. PMID 40526008
• Staron A, Burks EJ, Lee JC, Sarosiek S, Sloan JM, Sanchorawala V. Assessment of minimal residual disease using multiparametric flow cytometry in patients with AL amyloidosis. Blood Adv. 2020 Mar 10;4(5):880-884. doi: 10.1182/bloodadvances.2019001331. PMID 32130406
• Staron A, Connors LH, Ruberg FL, Mendelson LM, Sanchorawala V. A new era of amyloidosis: the trends at a major US referral centre. Amyloid. 2019 Dec;26(4):192-196. doi: 10.1080/13506129.2019.1640672. Epub 2019 Jul 15. PMID 31306033
• Lilleness B, Ruberg FL, Mussinelli R, Doros G, Sanchorawala V. Development and validation of a survival staging system incorporating BNP in patients with light chain amyloidosis. Blood. 2019 Jan 17;133(3):215-223. doi: 10.1182/blood-2018-06-858951. Epub 2018 Oct 17. PMID 30333122
• Shu J, Lo S, Phillips M, Sun F, Seldin DC, Berenbaum I, Berk JL, Sanchorawala V. Depression and anxiety in patients with AL amyloidosis as assessed by the SF-36 questionnaire: experience in 1226 patients. Amyloid. 2016 Sep;23(3):188-193. doi: 10.1080/13506129.2016.1208081. Epub 2016 Jul 27. PMID 27460276
• Cowan AJ, Skinner M, Seldin DC, Berk JL, Lichtenstein DR, O'Hara CJ, Doros G, Sanchorawala V. Amyloidosis of the gastrointestinal tract: a 13-year, single-center, referral experience. Haematologica. 2013 Jan;98(1):141-6. doi: 10.3324/haematol.2012.068155. Epub 2012 Jun 24. PMID 22733017

Идентификаторы

NCT: NCT00898235 · H-22838 · CDR0000632869

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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