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

doi:10.3109/13506129.2012.678509

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

Neurology

Research output: Contribution to journal › Article › peer-review

36 Scopus citations

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 language	English (US)
Pages (from-to)	37-38
Number of pages	2
Journal	Amyloid
Volume	19
Issue number	SUPPL. 1
DOIs	https://doi.org/10.3109/13506129.2012.678509
State	Published - Jun 2012

Keywords

Clinical trials
Diflunisal
FAP
Neuropathy
transthyretin

ASJC Scopus subject areas

Internal Medicine

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title = "The Diflunisal Trial: Study accrual and drug tolerance",

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.",

keywords = "Clinical trials, Diflunisal, FAP, Neuropathy, transthyretin",

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

note = "Funding Information: Declaration of Interest: Multiple authors contributing to this work have a consulting relationship with Pfizer Pharmaceuticals. This work is supported by NIH R01 NS051306, NIH UL1 RR025771, Orphan Products Development Grant FD-R002532 and the Young Family Research Fund.",

year = "2012",

month = jun,

doi = "10.3109/13506129.2012.678509",

language = "English (US)",

volume = "19",

pages = "37--38",

journal = "Amyloid",

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AU - Berk, John L.

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AU - Sekijima, Yoshiki

AU - Yamashita, Taro

AU - Heneghan, Michael

AU - Zeldenrust, Steven R.

AU - Ando, Yukio

AU - Ikeda, Shu Ichi

AU - Gorevic, Peter

AU - Merlini, Giampaolo

AU - Kelly, Jeffrey W.

AU - Skinner, Martha

AU - Bisbee, Alice B.

AU - Dyck, Peter J.

AU - Obici, Laura

N1 - Funding Information: Declaration of Interest: Multiple authors contributing to this work have a consulting relationship with Pfizer Pharmaceuticals. This work is supported by NIH R01 NS051306, NIH UL1 RR025771, Orphan Products Development Grant FD-R002532 and the Young Family Research Fund.

PY - 2012/6

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AB - 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.

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KW - Neuropathy

KW - transthyretin

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The Diflunisal Trial: Study accrual and drug tolerance

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Keywords

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