alexa Design Considerations for Clinical Trials Using the Cutaneous Dermatomyositis Disease Area and Severity Index as a Primary Endpoint
ISSN: 2155-9554

Journal of Clinical & Experimental Dermatology Research
Open Access

Like us on:
OMICS International organises 3000+ Global Conferenceseries Events every year across USA, Europe & Asia with support from 1000 more scientific Societies and Publishes 700+ Open Access Journals which contains over 50000 eminent personalities, reputed scientists as editorial board members.

Open Access Journals gaining more Readers and Citations

700 Journals and 15,000,000 Readers Each Journal is getting 25,000+ Readers

This Readership is 10 times more when compared to other Subscription Journals (Source: Google Analytics)

Research Article

Design Considerations for Clinical Trials Using the Cutaneous Dermatomyositis Disease Area and Severity Index as a Primary Endpoint

Julie A. Brevard1*, Mark Hurtt1, Joanna Horobin1, Olga Polyanskaya1, James Baker1, Victoria P. Werth2,3 and David F. Fiorentino4

1Idera Pharmaceuticals, Inc., Cambridge, MA, U.S.A

2Corporate Michael J. Crescenz (Philadelphia) Veterans Affairs Medical Center, Philadelphia, PA, U.S.A

3Department of Dermatology, University of Pennsylvania School of Medicine, Philadelphia, PA, U.S.A

4Department of Dermatology, Stanford University School of Medicine, Stanford, CA, U.S.A

*Corresponding Author:
Julie A. Brevard
Idera Pharmaceuticals, Inc., Cambridge, MA, U.S.A
Tel: 6176795567
E-mail: [email protected]

Received date: May 26, 2017; Accepted date: June 19, 2017; Published date: June 22, 2017

Citation: Brevard JA, Hurtt M, Horobin J, Polyanskaya O, Baker J, et al. (2017) Design Considerations for Clinical Trials Using the Cutaneous Dermatomyositis Disease Area and Severity Index as a Primary Endpoint. J Clin Exp Dermatol Res 8:401. doi: 10.4172/2155-9554.1000401

Copyright: © 2017 Brevard JA, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.



Objective: Skin inflammation is a universal characteristic of the multisystem inflammatory myopathy dermatomyositis (DM). About 35% of DM patients have a polycyclic or chronic disease course, usually due to active skin disease, despite standard of care therapy, demonstrating a significant unmet need for novel treatment options. To test the efficacy of novel therapeutics, feasible prospective clinical trials must be designed that employ quantitative endpoints. The Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) is a validated, reliable, and responsive measurement of cutaneous DM disease activity and damage. The primary objectives were to identify requisite design parameters (e.g., target trial population, duration, frequency of assessments, treatment difference, sample size) for a proofofconcept clinical trial of a novel therapy in patients with DM.

Methods: Trial simulations were conducted to determine sample size and statistical power. Simulation inputs were obtained from analyses of CDASI natural history data obtained from 115 adult DM patients followed at Stanford University.

Results: A population with baseline CDASI Activity scores ≥ 15 was simulated to eliminate a potential floor effect in patients with milder disease and to mimic a clinical trial population with moderate to severe skin disease. Given a 24 weeks trial with monthly disease assessments, simulations demonstrated that 45 patients per group were required to detect a 5 point treatment difference and 12 patients per group were required to detect a 10 point treatment difference with 80% statistical power using a 2 sided test.

Conclusion: This study provides evidence that the CDASI is a practical and feasible primary efficacy endpoint for DM clinical trials and lays out a framework for a clinical trial using CDASI as a primary endpoint.


Share This Page

Additional Info

Loading Please wait..
Peer Reviewed Journals
Make the best use of Scientific Research and information from our 700 + peer reviewed, Open Access Journals
International Conferences 2017-18
Meet Inspiring Speakers and Experts at our 3000+ Global Annual Meetings

Contact Us

Agri, Food, Aqua and Veterinary Science Journals

Dr. Krish

[email protected]

1-702-714-7001 Extn: 9040

Clinical and Biochemistry Journals

Datta A

[email protected]

1-702-714-7001Extn: 9037

Business & Management Journals


[email protected]

1-702-714-7001Extn: 9042

Chemical Engineering and Chemistry Journals

Gabriel Shaw

[email protected]

1-702-714-7001 Extn: 9040

Earth & Environmental Sciences

Katie Wilson

[email protected]

1-702-714-7001Extn: 9042

Engineering Journals

James Franklin

[email protected]

1-702-714-7001Extn: 9042

General Science and Health care Journals

Andrea Jason

[email protected]

1-702-714-7001Extn: 9043

Genetics and Molecular Biology Journals

Anna Melissa

[email protected]

1-702-714-7001 Extn: 9006

Immunology & Microbiology Journals

David Gorantl

[email protected]

1-702-714-7001Extn: 9014

Informatics Journals

Stephanie Skinner

[email protected]

1-702-714-7001Extn: 9039

Material Sciences Journals

Rachle Green

[email protected]

1-702-714-7001Extn: 9039

Mathematics and Physics Journals

Jim Willison

[email protected]

1-702-714-7001 Extn: 9042

Medical Journals

Nimmi Anna

[email protected]

1-702-714-7001 Extn: 9038

Neuroscience & Psychology Journals

Nathan T

[email protected]

1-702-714-7001Extn: 9041

Pharmaceutical Sciences Journals

John Behannon

[email protected]

1-702-714-7001Extn: 9007

Social & Political Science Journals

Steve Harry

[email protected]

1-702-714-7001 Extn: 9042

© 2008-2017 OMICS International - Open Access Publisher. Best viewed in Mozilla Firefox | Google Chrome | Above IE 7.0 version