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An observational, non-interventional study for the follow-up of patients with amyloidosis who received miridesap followed by dezamizumab in a phase 1 study.
Richards, Duncan; Millns, Helen; Cookson, Louise; Lukas, Mary Ann.
Afiliação
  • Richards D; University of Oxford, Oxford, UK.
  • Millns H; GlaxoSmithKline, Stevenage, Hertfordshire, UK.
  • Cookson L; GlaxoSmithKline, Cambridge, UK.
  • Lukas MA; GlaxoSmithKline, Philadelphia, PA, USA. mary.ann.lukas@gsk.com.
Orphanet J Rare Dis ; 17(1): 259, 2022 07 09.
Article em En | MEDLINE | ID: mdl-35810311
BACKGROUND: Miridesap depletes circulating serum amyloid P (SAP) and dezamizumab (anti-SAP monoclonal antibody) targets SAP on amyloid deposits, triggering amyloid removal. In a phase 1, first-in-human study (FIHS), progressive amyloid removal was observed in some patients after ≤ 3 cycles of miridesap/dezamizumab. METHODS: This observational, non-interventional study in patients who received miridesap/dezamizumab during the FIHS (planned follow-up: 5 years) evaluated response to treatment based on routine assessments of disease status and key organ function. In a post hoc analysis, patients responding to treatment in the FIHS during follow-up were identified as responders and further categorized as sustained or declining responders. RESULTS: In the FIHS, 17/23 patients were treatment responders. Of these patients, seven (immunoglobulin light chain [AL], n = 6; serum amyloid A, n = 1) were considered sustained responders and ten (fibrinogen-a alpha chain [AFib], n = 5; AL, n = 4; apolipoprotein A-I, n = 1) were considered declining responders. We primarily present responder patient-level data for functional, cardiac, laboratory and imaging assessments conducted during the follow-up period, with non-responder data presented as supplementary. CONCLUSION: No further development of miridesap/dezamizumab is planned in amyloidosis. However, long-term follow-up of these patients may provide insight into whether active removal of amyloid deposits has an impact on disease progression. TRIAL REGISTRATION: ClinicalTrials.gov, NCT01777243. Registered 28 January 2013, https://clinicaltrials.gov/ct2/show/study/NCT01777243 .
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Placa Amiloide / Amiloidose Tipo de estudo: Observational_studies / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Placa Amiloide / Amiloidose Tipo de estudo: Observational_studies / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article