Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Trial

Objective. This study evaluated subcutaneous injections of tetrodotoxin (TTX) for the treatment of moderate to severe, inadequately controlled cancer-related pain. Methods. Eligible patients were randomized to receive TTX (30 μg) or placebo subcutaneously twice daily for four consecutive days. Effic...

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Main Authors: Neil A. Hagen, Lyne Cantin, John Constant, Tina Haller, Gilbert Blaise, May Ong-Lam, Patrick du Souich, Walter Korz, Bernard Lapointe
Format: Article
Language:English
Published: Wiley 2017-01-01
Series:Pain Research and Management
Online Access:http://dx.doi.org/10.1155/2017/7212713
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author Neil A. Hagen
Lyne Cantin
John Constant
Tina Haller
Gilbert Blaise
May Ong-Lam
Patrick du Souich
Walter Korz
Bernard Lapointe
author_facet Neil A. Hagen
Lyne Cantin
John Constant
Tina Haller
Gilbert Blaise
May Ong-Lam
Patrick du Souich
Walter Korz
Bernard Lapointe
author_sort Neil A. Hagen
collection DOAJ
description Objective. This study evaluated subcutaneous injections of tetrodotoxin (TTX) for the treatment of moderate to severe, inadequately controlled cancer-related pain. Methods. Eligible patients were randomized to receive TTX (30 μg) or placebo subcutaneously twice daily for four consecutive days. Efficacy was assessed using pain and composite endpoints (including pain and quality of life measures), and safety was evaluated using standard measures. Results. 165 patients were enrolled at 19 sites in Canada, Australia, and New Zealand, with 149 patients in the primary analysis “intent-to-treat” population. The primary analysis supports a clinical benefit of TTX over placebo based on the pain endpoint alone with a clinically significant estimated effect size of 16.2% (p=0.0460). The p value was nominally statistically significant after prespecified (Bonferroni Holm) adjustment for the two primary endpoints but not at the prespecified two-sided 5% level. The mean duration of analgesic response was 56.7 days (TTX) and 9.9 days (placebo). Most common adverse events were nausea, dizziness, and oral numbness or tingling and were generally mild to moderate and transient. Conclusions. Although underpowered, this study demonstrates a clinically important analgesic signal. TTX may provide clinically meaningful analgesia for patients who have persistent moderate to severe cancer pain despite best analgesic care. This clinical study is registered with ClinicalTrials.gov (NCT00725114).
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spelling doaj-art-ed60ff06802d4a0a9fa69a9b64ff54e92025-08-20T02:22:10ZengWileyPain Research and Management1203-67651918-15232017-01-01201710.1155/2017/72127137212713Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design TrialNeil A. Hagen0Lyne Cantin1John Constant2Tina Haller3Gilbert Blaise4May Ong-Lam5Patrick du Souich6Walter Korz7Bernard Lapointe8Tom Baker Cancer Centre and Division of Palliative Medicine, University of Calgary, 1331 29 Street NW, Calgary, AB, T2N4N2, CanadaClinForce Services Inc., Vancouver, BC, CanadaPRA Health Sciences, Victoria, 655 Tyee Rd., Victoria, BC, V9A 6X5, CanadaStatcon Consulting Services, 6 Matson Drive, Caledon, ON, L7E 0A3, CanadaDépartement d’Anesthésiologie, CHUM, 1560, rue Sherbrooke Est, Pavillon Deschamps, Local FS-1136, Montréal, QC, H2L 4M1, CanadaSt. Paul’s Hospital, 1081 Burrard Street, Vancouver, BC, V6Z 1Y6, CanadaDépartement de Pharmacologie et Physiologie, Faculté de Médecine, Université de Montréal, CP 6128 Centre-Ville, Montréal, QC, H3C 3J7, CanadaWEX Pharmaceuticals Inc., 420-1090 West Pender Street, Vancouver, BC, V6E 2N7, CanadaClinical Research Unit, Jewish General Hospital, Room E-872 3755, Chemin de la Côte Sainte-Catherine, Montréal, QC, H3T 1E2, CanadaObjective. This study evaluated subcutaneous injections of tetrodotoxin (TTX) for the treatment of moderate to severe, inadequately controlled cancer-related pain. Methods. Eligible patients were randomized to receive TTX (30 μg) or placebo subcutaneously twice daily for four consecutive days. Efficacy was assessed using pain and composite endpoints (including pain and quality of life measures), and safety was evaluated using standard measures. Results. 165 patients were enrolled at 19 sites in Canada, Australia, and New Zealand, with 149 patients in the primary analysis “intent-to-treat” population. The primary analysis supports a clinical benefit of TTX over placebo based on the pain endpoint alone with a clinically significant estimated effect size of 16.2% (p=0.0460). The p value was nominally statistically significant after prespecified (Bonferroni Holm) adjustment for the two primary endpoints but not at the prespecified two-sided 5% level. The mean duration of analgesic response was 56.7 days (TTX) and 9.9 days (placebo). Most common adverse events were nausea, dizziness, and oral numbness or tingling and were generally mild to moderate and transient. Conclusions. Although underpowered, this study demonstrates a clinically important analgesic signal. TTX may provide clinically meaningful analgesia for patients who have persistent moderate to severe cancer pain despite best analgesic care. This clinical study is registered with ClinicalTrials.gov (NCT00725114).http://dx.doi.org/10.1155/2017/7212713
spellingShingle Neil A. Hagen
Lyne Cantin
John Constant
Tina Haller
Gilbert Blaise
May Ong-Lam
Patrick du Souich
Walter Korz
Bernard Lapointe
Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Trial
Pain Research and Management
title Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Trial
title_full Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Trial
title_fullStr Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Trial
title_full_unstemmed Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Trial
title_short Tetrodotoxin for Moderate to Severe Cancer-Related Pain: A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Trial
title_sort tetrodotoxin for moderate to severe cancer related pain a multicentre randomized double blind placebo controlled parallel design trial
url http://dx.doi.org/10.1155/2017/7212713
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