TRANSCEND
Research type
Research Study
Full title
TRansthyretin Amyloidosis: Neuropathy, Senility, Cardiomyopathy, Evaluation, Natural history and Diagnosis
IRAS ID
109481
Contact name
Julian Gillmore
Contact email
Sponsor organisation
R&D Department, Royal Free Hospital
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Transthyretin (ATTR) amyloidosis is a life-threatening disorder in which abnormal proteins are deposited in tissues as insoluble amyloid deposits. ATTR amyloid deposits may cause hereditary or acquired (wild-type) ATTR amyloidosis; the inherited condition typically causes disease in the heart and nerves, and the acquired condition causes a predominant heart failure. The natural history of ATTR amyloidosis is poorly characterized and few data are available regarding its response to novel ‘disease modifying’ therapies. \n\nThe objectives of this observational study are to determine the disease characteristics, natural history, and outcome of ATTR amyloidosis by means of systematic collection of the following data:\n• Organ involvement by amyloid and organ dysfunction due to amyloid in the different types of ATTR amyloidosis\n• Rate and nature of change in organ function over time\n• Side effects and toxicities of therapy\n• Hospitalisations\n• Quality of Life (QOL) throughout the disease course (with and without disease modifying therapy)\n• Survival.\n\nParticipants will be assessed according to standard NHS clinical care at the UK National Amyloidosis Centre (NAC) at 0, 12, 24, 36, 48 and 60 months. A subset of patients with hereditary ATTR amyloidosis affecting the nerves will also undergo specialised neuropathy assessments according to standard NHS clinical care. QoL questionnaires will be completed at baseline and then annually to 60 months according to standard of care (SoC). Details of hospitalisations, alterations in medications, and outcomes will be obtained between NAC attendances by telephone or via the Medopad digital application (app). NAC staff will continue to liaise with the patient’s GP and other local healthcare professionals (HCPs) involved in the patient’s care to promote a ’shared care’ model; this is also SoC.\n
REC name
North of Scotland Research Ethics Committee 1
REC reference
19/NS/0136
Date of REC Opinion
24 Jul 2019
REC opinion
Favourable Opinion