The Diflunisal Trial: Study accrual and drug tolerance

John L. Berk, Ole B. Suhr, Yoshiki Sekijima, Taro Yamashita, Michael Heneghan, Steven R. Zeldenrust, Yukio Ando, Shu Ichi Ikeda, Peter Gorevic, Giampaolo Merlini, Jeffrey W. Kelly, Martha Skinner, Alice B. Bisbee, Peter J Dyck, Laura Obici

Research output: Contribution to journalArticle

33 Citations (Scopus)

Abstract

Familial amyloidotic polyneuropathy (FAP) is a protein folding disorder that induces neuropathy and cardiomyopathy, leading to death within 715 years after onset of clinical disease. In vitro, small ligands binding the thyroid hormone docking site stabilize tetrameric transthyretin, inhibiting amyloid fibril formation. We undertook a randomized, placebo-controlled clinical trial to determine whether diflunisal, a well-known non-steroidal anti-inflammatory drug (NSAID) alters neurologic disease progression in FAP. We enrolled 130 subjects with wide age and FAP mutation representation. To date, few recognized complications of NSAIDs have occurred in the study cohort. Data collection will be completed by November 2012.

Original languageEnglish (US)
Pages (from-to)37-38
Number of pages2
JournalAmyloid
Volume19
Issue numberSUPPL. 1
DOIs
StatePublished - Jun 2012

Fingerprint

Diflunisal
Drug Tolerance
Polyneuropathies
Proteostasis Deficiencies
Prealbumin
Non-Steroidal Anti-Inflammatory Agents
Nervous System Diseases
Cardiomyopathies
Thyroid Hormones
Amyloid
Disease Progression
Cohort Studies
Anti-Inflammatory Agents
Randomized Controlled Trials
Placebos
Ligands
Mutation
Pharmaceutical Preparations

Keywords

  • Clinical trials
  • Diflunisal
  • FAP
  • Neuropathy
  • transthyretin

ASJC Scopus subject areas

  • Internal Medicine

Cite this

Berk, J. L., Suhr, O. B., Sekijima, Y., Yamashita, T., Heneghan, M., Zeldenrust, S. R., ... Obici, L. (2012). The Diflunisal Trial: Study accrual and drug tolerance. Amyloid, 19(SUPPL. 1), 37-38. https://doi.org/10.3109/13506129.2012.678509

The Diflunisal Trial : Study accrual and drug tolerance. / Berk, John L.; Suhr, Ole B.; Sekijima, Yoshiki; Yamashita, Taro; Heneghan, Michael; Zeldenrust, Steven R.; Ando, Yukio; Ikeda, Shu Ichi; Gorevic, Peter; Merlini, Giampaolo; Kelly, Jeffrey W.; Skinner, Martha; Bisbee, Alice B.; Dyck, Peter J; Obici, Laura.

In: Amyloid, Vol. 19, No. SUPPL. 1, 06.2012, p. 37-38.

Research output: Contribution to journalArticle

Berk, JL, Suhr, OB, Sekijima, Y, Yamashita, T, Heneghan, M, Zeldenrust, SR, Ando, Y, Ikeda, SI, Gorevic, P, Merlini, G, Kelly, JW, Skinner, M, Bisbee, AB, Dyck, PJ & Obici, L 2012, 'The Diflunisal Trial: Study accrual and drug tolerance', Amyloid, vol. 19, no. SUPPL. 1, pp. 37-38. https://doi.org/10.3109/13506129.2012.678509
Berk JL, Suhr OB, Sekijima Y, Yamashita T, Heneghan M, Zeldenrust SR et al. The Diflunisal Trial: Study accrual and drug tolerance. Amyloid. 2012 Jun;19(SUPPL. 1):37-38. https://doi.org/10.3109/13506129.2012.678509
Berk, John L. ; Suhr, Ole B. ; Sekijima, Yoshiki ; Yamashita, Taro ; Heneghan, Michael ; Zeldenrust, Steven R. ; Ando, Yukio ; Ikeda, Shu Ichi ; Gorevic, Peter ; Merlini, Giampaolo ; Kelly, Jeffrey W. ; Skinner, Martha ; Bisbee, Alice B. ; Dyck, Peter J ; Obici, Laura. / The Diflunisal Trial : Study accrual and drug tolerance. In: Amyloid. 2012 ; Vol. 19, No. SUPPL. 1. pp. 37-38.
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