CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy

Gunnar M. Buyse; Nathalie Goemans; Erik Henricson; Alejandro Jara; Marleen van den Hauwe; Robert Leshner; Julaine M. Florence; Jill E. Mayhew; Diana M. Escolar

doi:10.1016/j.ejpn.2007.02.009

CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy

Gunnar M. Buyse
, Nathalie Goemans
, Erik Henricson
, Alejandro Jara
, Marleen van den Hauwe
, Robert Leshner
, Julaine M. Florence
, Jill E. Mayhew
, Diana M. Escolar

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

14 Scopus citations

Abstract

The authors report a pilot open-label two-center therapeutic trial of oxatomide in 14 steroid-naive DMD boys aged 5-10 years. Comparison of linear evolutions between 3 months medication-free lead-in periods and 6 months treatment periods showed no significant differences in quantitative (QMT) and manual (MMT) measurements of muscle strength and timed functional tests. A modest mitigation of strength deterioration over time cannot be excluded.

Original language	English (US)
Pages (from-to)	337-340
Number of pages	4
Journal	European Journal of Paediatric Neurology
Volume	11
Issue number	6
DOIs	https://doi.org/10.1016/j.ejpn.2007.02.009
State	Published - Nov 2007
Externally published	Yes

Keywords

Clinical trial
Duchenne
Muscle
Muscular dystrophy
Oxatomide
Treatment

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Clinical Neurology

Access to Document

10.1016/j.ejpn.2007.02.009

Cite this

@article{d277a835100c4801992e4c56422b37ec,

title = "CINRG pilot trial of oxatomide in steroid-na{\"i}ve Duchenne muscular dystrophy",

abstract = "The authors report a pilot open-label two-center therapeutic trial of oxatomide in 14 steroid-naive DMD boys aged 5-10 years. Comparison of linear evolutions between 3 months medication-free lead-in periods and 6 months treatment periods showed no significant differences in quantitative (QMT) and manual (MMT) measurements of muscle strength and timed functional tests. A modest mitigation of strength deterioration over time cannot be excluded.",

keywords = "Clinical trial, Duchenne, Muscle, Muscular dystrophy, Oxatomide, Treatment",

author = "Buyse, \{Gunnar M.\} and Nathalie Goemans and Erik Henricson and Alejandro Jara and \{van den Hauwe\}, Marleen and Robert Leshner and Florence, \{Julaine M.\} and Mayhew, \{Jill E.\} and Escolar, \{Diana M.\}",

note = "Funding Information: The authors respectfully and gratefully commemorate the late Dr. Kantilal Patel. This study was supported by a grant from the Muscular Dystrophy Association USA and from the Association Fran{\c c}aise contre les Myopathies. The authors also thank Janssen-Cilag for providing the study drug (Tinset {\textregistered} ), all patients and parents who participated in the study, and Drs. Paula Clemens, Jean Mah and Yoram Nevo for useful discussions. ",

year = "2007",

month = nov,

doi = "10.1016/j.ejpn.2007.02.009",

language = "English (US)",

volume = "11",

pages = "337--340",

journal = "European Journal of Paediatric Neurology",

issn = "1090-3798",

publisher = "W.B. Saunders Ltd",

number = "6",

}

TY - JOUR

T1 - CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy

AU - Buyse, Gunnar M.

AU - Goemans, Nathalie

AU - Henricson, Erik

AU - Jara, Alejandro

AU - van den Hauwe, Marleen

AU - Leshner, Robert

AU - Florence, Julaine M.

AU - Mayhew, Jill E.

AU - Escolar, Diana M.

N1 - Funding Information: The authors respectfully and gratefully commemorate the late Dr. Kantilal Patel. This study was supported by a grant from the Muscular Dystrophy Association USA and from the Association Française contre les Myopathies. The authors also thank Janssen-Cilag for providing the study drug (Tinset ® ), all patients and parents who participated in the study, and Drs. Paula Clemens, Jean Mah and Yoram Nevo for useful discussions.

PY - 2007/11

Y1 - 2007/11

N2 - The authors report a pilot open-label two-center therapeutic trial of oxatomide in 14 steroid-naive DMD boys aged 5-10 years. Comparison of linear evolutions between 3 months medication-free lead-in periods and 6 months treatment periods showed no significant differences in quantitative (QMT) and manual (MMT) measurements of muscle strength and timed functional tests. A modest mitigation of strength deterioration over time cannot be excluded.

AB - The authors report a pilot open-label two-center therapeutic trial of oxatomide in 14 steroid-naive DMD boys aged 5-10 years. Comparison of linear evolutions between 3 months medication-free lead-in periods and 6 months treatment periods showed no significant differences in quantitative (QMT) and manual (MMT) measurements of muscle strength and timed functional tests. A modest mitigation of strength deterioration over time cannot be excluded.

KW - Clinical trial

KW - Duchenne

KW - Muscle

KW - Muscular dystrophy

KW - Oxatomide

KW - Treatment

UR - https://www.scopus.com/pages/publications/35148852342

UR - https://www.scopus.com/pages/publications/35148852342#tab=citedBy

U2 - 10.1016/j.ejpn.2007.02.009

DO - 10.1016/j.ejpn.2007.02.009

M3 - Article

C2 - 17459739

AN - SCOPUS:35148852342

SN - 1090-3798

VL - 11

SP - 337

EP - 340

JO - European Journal of Paediatric Neurology

JF - European Journal of Paediatric Neurology

IS - 6

ER -

CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy

Abstract

Keywords

ASJC Scopus subject areas

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