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November 22, 2004

Functional outcome measures as clinical trial endpoints in ALS

B. J. Traynor, MD, MRCPI, H. Zhang, PhD, J. M. Shefner, MD, PhD, D. Schoenfeld, PhD, M. E. Cudkowicz, MD, MSc, and on behalf of the NEALS ConsortiumAuthors Info & Affiliations
November 23, 2004 issue
63 (10) 1933-1935
https://doi.org/10.1212/01.WNL.0000144345.49510.4E
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Abstract

The topiramate study was a 12-month randomized placebo-controlled trial in patients with ALS. Follow-up evaluation of the placebo group (n = 97) constituted a well-described cohort of patients with ALS, in whom multiple outcome measures were assessed at 3-month intervals. During the 12-month study period, the decline of forced vital capacity (FVC%) and ALS functional rating scale (ALSFRS) was linear, whereas the decline of maximum voluntary isometric contraction–arm (MVIC-arm) and MVIC-grip Z scores was curvilinear. Rates of FVC% and ALFRS decline, but not of MVIC-arm or MVIC-grip, were independent predictors of survival.

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Information & Authors

Information

Published In

Neurology®
Volume 63 • Number 10 • November 23, 2004
Pages: 1933-1935

Copyright

© 2004.

Publication History

Received: February 3, 2004
Accepted: July 29, 2004
Published online: November 22, 2004
Published in print: November 23, 2004

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Authors

Affiliations & Disclosures

B. J. Traynor, MD, MRCPI
From the Neurology Clinical Trials Unit (Drs. Traynor and Cudkowicz), Department of Neurology, Massachusetts General Hospital, Boston, MA; Biostatistical Center (Drs. Zhang and Schoenfeld), Department of Medicine, Massachusetts General Hospital, Boston, MA; Department of Neurology (Dr. Shefner), Upstate Medical University, Syracuse, NY; and Clinical Investigator Training Program (Dr. Traynor), Harvard/MIT Health Sciences and Technology–Beth Israel Deaconess Medical Center, Boston, MA.
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H. Zhang, PhD
From the Neurology Clinical Trials Unit (Drs. Traynor and Cudkowicz), Department of Neurology, Massachusetts General Hospital, Boston, MA; Biostatistical Center (Drs. Zhang and Schoenfeld), Department of Medicine, Massachusetts General Hospital, Boston, MA; Department of Neurology (Dr. Shefner), Upstate Medical University, Syracuse, NY; and Clinical Investigator Training Program (Dr. Traynor), Harvard/MIT Health Sciences and Technology–Beth Israel Deaconess Medical Center, Boston, MA.
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J. M. Shefner, MD, PhD
From the Neurology Clinical Trials Unit (Drs. Traynor and Cudkowicz), Department of Neurology, Massachusetts General Hospital, Boston, MA; Biostatistical Center (Drs. Zhang and Schoenfeld), Department of Medicine, Massachusetts General Hospital, Boston, MA; Department of Neurology (Dr. Shefner), Upstate Medical University, Syracuse, NY; and Clinical Investigator Training Program (Dr. Traynor), Harvard/MIT Health Sciences and Technology–Beth Israel Deaconess Medical Center, Boston, MA.
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D. Schoenfeld, PhD
From the Neurology Clinical Trials Unit (Drs. Traynor and Cudkowicz), Department of Neurology, Massachusetts General Hospital, Boston, MA; Biostatistical Center (Drs. Zhang and Schoenfeld), Department of Medicine, Massachusetts General Hospital, Boston, MA; Department of Neurology (Dr. Shefner), Upstate Medical University, Syracuse, NY; and Clinical Investigator Training Program (Dr. Traynor), Harvard/MIT Health Sciences and Technology–Beth Israel Deaconess Medical Center, Boston, MA.
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M. E. Cudkowicz, MD, MSc
From the Neurology Clinical Trials Unit (Drs. Traynor and Cudkowicz), Department of Neurology, Massachusetts General Hospital, Boston, MA; Biostatistical Center (Drs. Zhang and Schoenfeld), Department of Medicine, Massachusetts General Hospital, Boston, MA; Department of Neurology (Dr. Shefner), Upstate Medical University, Syracuse, NY; and Clinical Investigator Training Program (Dr. Traynor), Harvard/MIT Health Sciences and Technology–Beth Israel Deaconess Medical Center, Boston, MA.
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on behalf of the NEALS Consortium
From the Neurology Clinical Trials Unit (Drs. Traynor and Cudkowicz), Department of Neurology, Massachusetts General Hospital, Boston, MA; Biostatistical Center (Drs. Zhang and Schoenfeld), Department of Medicine, Massachusetts General Hospital, Boston, MA; Department of Neurology (Dr. Shefner), Upstate Medical University, Syracuse, NY; and Clinical Investigator Training Program (Dr. Traynor), Harvard/MIT Health Sciences and Technology–Beth Israel Deaconess Medical Center, Boston, MA.

Notes

Address correspondence and reprint requests to Dr. Bryan J. Traynor, Massachusetts General Hospital–East, Day Laboratory for Neuromuscular Research, Building 114, Room 3125, 16th Street, Charlestown, MA 02129; e-mail: [email protected]

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