A multicenter, randomized, double-blind, placebo-controlled trial of long-term ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL)

The study protocol [EudraCT no.: 2006-000032-27]

Davide Pareyson, Angelo Schenone, Gian Maria Fabrizi, Lucio Santoro, Luca Padua, Aldo Quattrone, Giuseppe Vita, Franco Gemignani, Francesco Visioli, Alessandra Solari, E. Salsano, V. Scaioli, C. Ciano, M. Rimoldi, G. Lauria, E. Rizzetto, F. Camozzi, M. Grandis, E. Narciso, L. Nobbio & 33 others L. Benedetti, N. Rizzuto, T. Cavallaro, L. Bertolasi, A. Casano, F. Manganelli, M. Nolano, C. Pazzaglia, P. Valentino, R. Nisticò, D. Pirritano, A. Lucisano, M. Canino, A. Mazzeo, M. Aguennouz, R. Di Leo, P. Girlanda, G. Majorana, A. Ciranni, N. Lanzano, F. Brindani, C. Lettieri, P. Bogani, R. A C Hughes, G. L. Mancardi, G. Cavaletti, S. Galimberti, D. Radice, D. Calabrese, G. Ferrari, M. Rimoldi., V. Scaioli, G. Lauria

Research output: Contribution to journalArticle

40 Citations (Scopus)

Abstract

There is no treatment for Charcot-Marie-Tooth disease 1A (CMT1A), but ascorbic acid (AA) is efficacious in the transgenic mouse model. Thus, a clinical trial of AA in CMT1A is warranted. The CMT-TRIAAL is a phase III randomized, double-blind, placebo-controlled study involving 222 CMT1A adults from eight Italian centers. Eligible for the study are symptomatic adults with genetically confirmed CMT1A. Treatment consists of 2-year oral AA (1500 mg/day) or placebo. The primary trial endpoint is an improvement in CMT Neuropathy Score. Secondary efficacy endpoints are changes in distal arm and leg maximum voluntary isometric contraction; 10 m timed walking; 9-hole-peg test; overall neuropathy limitations scale; pain and fatigue visual analog scales; health-related quality of life (SF-36); and electrophysiology. Clinical-electrophysiological assessments are performed at baseline and every 6 months thereafter. In consenting patients from three centers, skin biopsy is performed to evaluate PMP22 expression. The study will last 34 months, starting from March 2006.

Original languageEnglish
Pages (from-to)436-441
Number of pages6
JournalPharmacological Research
Volume54
Issue number6
DOIs
Publication statusPublished - Dec 1 2006

Fingerprint

Charcot-Marie-Tooth Disease
Ascorbic Acid
Placebos
Isometric Contraction
Electrophysiology
Pain Measurement
Therapeutics
Transgenic Mice
Walking
Fatigue
Leg
Arm
Quality of Life
Clinical Trials
Biopsy
Skin

Keywords

  • Ascorbic acid
  • Charcot-Marie-Tooth disease
  • Genetic disorders
  • Neuropathy
  • PMP22
  • Randomized controlled trial

ASJC Scopus subject areas

  • Pharmacology

Cite this

A multicenter, randomized, double-blind, placebo-controlled trial of long-term ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL) : The study protocol [EudraCT no.: 2006-000032-27]. / Pareyson, Davide; Schenone, Angelo; Fabrizi, Gian Maria; Santoro, Lucio; Padua, Luca; Quattrone, Aldo; Vita, Giuseppe; Gemignani, Franco; Visioli, Francesco; Solari, Alessandra; Salsano, E.; Scaioli, V.; Ciano, C.; Rimoldi, M.; Lauria, G.; Rizzetto, E.; Camozzi, F.; Grandis, M.; Narciso, E.; Nobbio, L.; Benedetti, L.; Rizzuto, N.; Cavallaro, T.; Bertolasi, L.; Casano, A.; Manganelli, F.; Nolano, M.; Pazzaglia, C.; Valentino, P.; Nisticò, R.; Pirritano, D.; Lucisano, A.; Canino, M.; Mazzeo, A.; Aguennouz, M.; Di Leo, R.; Girlanda, P.; Majorana, G.; Ciranni, A.; Lanzano, N.; Brindani, F.; Lettieri, C.; Bogani, P.; Hughes, R. A C; Mancardi, G. L.; Cavaletti, G.; Galimberti, S.; Radice, D.; Calabrese, D.; Ferrari, G.; Rimoldi., M.; Scaioli, V.; Lauria, G.

In: Pharmacological Research, Vol. 54, No. 6, 01.12.2006, p. 436-441.

Research output: Contribution to journalArticle

Pareyson, D, Schenone, A, Fabrizi, GM, Santoro, L, Padua, L, Quattrone, A, Vita, G, Gemignani, F, Visioli, F, Solari, A, Salsano, E, Scaioli, V, Ciano, C, Rimoldi, M, Lauria, G, Rizzetto, E, Camozzi, F, Grandis, M, Narciso, E, Nobbio, L, Benedetti, L, Rizzuto, N, Cavallaro, T, Bertolasi, L, Casano, A, Manganelli, F, Nolano, M, Pazzaglia, C, Valentino, P, Nisticò, R, Pirritano, D, Lucisano, A, Canino, M, Mazzeo, A, Aguennouz, M, Di Leo, R, Girlanda, P, Majorana, G, Ciranni, A, Lanzano, N, Brindani, F, Lettieri, C, Bogani, P, Hughes, RAC, Mancardi, GL, Cavaletti, G, Galimberti, S, Radice, D, Calabrese, D, Ferrari, G, Rimoldi., M, Scaioli, V & Lauria, G 2006, 'A multicenter, randomized, double-blind, placebo-controlled trial of long-term ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL): The study protocol [EudraCT no.: 2006-000032-27]', Pharmacological Research, vol. 54, no. 6, pp. 436-441. https://doi.org/10.1016/j.phrs.2006.09.001
Pareyson, Davide ; Schenone, Angelo ; Fabrizi, Gian Maria ; Santoro, Lucio ; Padua, Luca ; Quattrone, Aldo ; Vita, Giuseppe ; Gemignani, Franco ; Visioli, Francesco ; Solari, Alessandra ; Salsano, E. ; Scaioli, V. ; Ciano, C. ; Rimoldi, M. ; Lauria, G. ; Rizzetto, E. ; Camozzi, F. ; Grandis, M. ; Narciso, E. ; Nobbio, L. ; Benedetti, L. ; Rizzuto, N. ; Cavallaro, T. ; Bertolasi, L. ; Casano, A. ; Manganelli, F. ; Nolano, M. ; Pazzaglia, C. ; Valentino, P. ; Nisticò, R. ; Pirritano, D. ; Lucisano, A. ; Canino, M. ; Mazzeo, A. ; Aguennouz, M. ; Di Leo, R. ; Girlanda, P. ; Majorana, G. ; Ciranni, A. ; Lanzano, N. ; Brindani, F. ; Lettieri, C. ; Bogani, P. ; Hughes, R. A C ; Mancardi, G. L. ; Cavaletti, G. ; Galimberti, S. ; Radice, D. ; Calabrese, D. ; Ferrari, G. ; Rimoldi., M. ; Scaioli, V. ; Lauria, G. / A multicenter, randomized, double-blind, placebo-controlled trial of long-term ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL) : The study protocol [EudraCT no.: 2006-000032-27]. In: Pharmacological Research. 2006 ; Vol. 54, No. 6. pp. 436-441.
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abstract = "There is no treatment for Charcot-Marie-Tooth disease 1A (CMT1A), but ascorbic acid (AA) is efficacious in the transgenic mouse model. Thus, a clinical trial of AA in CMT1A is warranted. The CMT-TRIAAL is a phase III randomized, double-blind, placebo-controlled study involving 222 CMT1A adults from eight Italian centers. Eligible for the study are symptomatic adults with genetically confirmed CMT1A. Treatment consists of 2-year oral AA (1500 mg/day) or placebo. The primary trial endpoint is an improvement in CMT Neuropathy Score. Secondary efficacy endpoints are changes in distal arm and leg maximum voluntary isometric contraction; 10 m timed walking; 9-hole-peg test; overall neuropathy limitations scale; pain and fatigue visual analog scales; health-related quality of life (SF-36); and electrophysiology. Clinical-electrophysiological assessments are performed at baseline and every 6 months thereafter. In consenting patients from three centers, skin biopsy is performed to evaluate PMP22 expression. The study will last 34 months, starting from March 2006.",
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author = "Davide Pareyson and Angelo Schenone and Fabrizi, {Gian Maria} and Lucio Santoro and Luca Padua and Aldo Quattrone and Giuseppe Vita and Franco Gemignani and Francesco Visioli and Alessandra Solari and E. Salsano and V. Scaioli and C. Ciano and M. Rimoldi and G. Lauria and E. Rizzetto and F. Camozzi and M. Grandis and E. Narciso and L. Nobbio and L. Benedetti and N. Rizzuto and T. Cavallaro and L. Bertolasi and A. Casano and F. Manganelli and M. Nolano and C. Pazzaglia and P. Valentino and R. Nistic{\`o} and D. Pirritano and A. Lucisano and M. Canino and A. Mazzeo and M. Aguennouz and {Di Leo}, R. and P. Girlanda and G. Majorana and A. Ciranni and N. Lanzano and F. Brindani and C. Lettieri and P. Bogani and Hughes, {R. A C} and Mancardi, {G. L.} and G. Cavaletti and S. Galimberti and D. Radice and D. Calabrese and G. Ferrari and M. Rimoldi. and V. Scaioli and G. Lauria",
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T1 - A multicenter, randomized, double-blind, placebo-controlled trial of long-term ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL)

T2 - The study protocol [EudraCT no.: 2006-000032-27]

AU - Pareyson, Davide

AU - Schenone, Angelo

AU - Fabrizi, Gian Maria

AU - Santoro, Lucio

AU - Padua, Luca

AU - Quattrone, Aldo

AU - Vita, Giuseppe

AU - Gemignani, Franco

AU - Visioli, Francesco

AU - Solari, Alessandra

AU - Salsano, E.

AU - Scaioli, V.

AU - Ciano, C.

AU - Rimoldi, M.

AU - Lauria, G.

AU - Rizzetto, E.

AU - Camozzi, F.

AU - Grandis, M.

AU - Narciso, E.

AU - Nobbio, L.

AU - Benedetti, L.

AU - Rizzuto, N.

AU - Cavallaro, T.

AU - Bertolasi, L.

AU - Casano, A.

AU - Manganelli, F.

AU - Nolano, M.

AU - Pazzaglia, C.

AU - Valentino, P.

AU - Nisticò, R.

AU - Pirritano, D.

AU - Lucisano, A.

AU - Canino, M.

AU - Mazzeo, A.

AU - Aguennouz, M.

AU - Di Leo, R.

AU - Girlanda, P.

AU - Majorana, G.

AU - Ciranni, A.

AU - Lanzano, N.

AU - Brindani, F.

AU - Lettieri, C.

AU - Bogani, P.

AU - Hughes, R. A C

AU - Mancardi, G. L.

AU - Cavaletti, G.

AU - Galimberti, S.

AU - Radice, D.

AU - Calabrese, D.

AU - Ferrari, G.

AU - Rimoldi., M.

AU - Scaioli, V.

AU - Lauria, G.

PY - 2006/12/1

Y1 - 2006/12/1

N2 - There is no treatment for Charcot-Marie-Tooth disease 1A (CMT1A), but ascorbic acid (AA) is efficacious in the transgenic mouse model. Thus, a clinical trial of AA in CMT1A is warranted. The CMT-TRIAAL is a phase III randomized, double-blind, placebo-controlled study involving 222 CMT1A adults from eight Italian centers. Eligible for the study are symptomatic adults with genetically confirmed CMT1A. Treatment consists of 2-year oral AA (1500 mg/day) or placebo. The primary trial endpoint is an improvement in CMT Neuropathy Score. Secondary efficacy endpoints are changes in distal arm and leg maximum voluntary isometric contraction; 10 m timed walking; 9-hole-peg test; overall neuropathy limitations scale; pain and fatigue visual analog scales; health-related quality of life (SF-36); and electrophysiology. Clinical-electrophysiological assessments are performed at baseline and every 6 months thereafter. In consenting patients from three centers, skin biopsy is performed to evaluate PMP22 expression. The study will last 34 months, starting from March 2006.

AB - There is no treatment for Charcot-Marie-Tooth disease 1A (CMT1A), but ascorbic acid (AA) is efficacious in the transgenic mouse model. Thus, a clinical trial of AA in CMT1A is warranted. The CMT-TRIAAL is a phase III randomized, double-blind, placebo-controlled study involving 222 CMT1A adults from eight Italian centers. Eligible for the study are symptomatic adults with genetically confirmed CMT1A. Treatment consists of 2-year oral AA (1500 mg/day) or placebo. The primary trial endpoint is an improvement in CMT Neuropathy Score. Secondary efficacy endpoints are changes in distal arm and leg maximum voluntary isometric contraction; 10 m timed walking; 9-hole-peg test; overall neuropathy limitations scale; pain and fatigue visual analog scales; health-related quality of life (SF-36); and electrophysiology. Clinical-electrophysiological assessments are performed at baseline and every 6 months thereafter. In consenting patients from three centers, skin biopsy is performed to evaluate PMP22 expression. The study will last 34 months, starting from March 2006.

KW - Ascorbic acid

KW - Charcot-Marie-Tooth disease

KW - Genetic disorders

KW - Neuropathy

KW - PMP22

KW - Randomized controlled trial

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U2 - 10.1016/j.phrs.2006.09.001

DO - 10.1016/j.phrs.2006.09.001

M3 - Article

VL - 54

SP - 436

EP - 441

JO - Pharmacological Research

JF - Pharmacological Research

SN - 1043-6618

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