Guidelines for clinical trials in systemic sclerosis (scleroderma)

Barbara White, Eugene A. Bauer, Lowell A. Goldsmith, Marc C. Hochberg, Linda M. Katz, Joseph H. Korn, Peter A. Lachenbruch, E. Carwile Leroy, Margaret P. Mitrane, Harold E. Paulus, Arnold Postlethwaite, Virginia D. Steen

Research output: Contribution to journalArticle

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Abstract

Objective. To develop guidelines for therapeutic trials designed to improve the overall course of systemic sclerosis (SSc), that is, to reduce the development of significant organ damage or death. Methods. A committee developed general guidelines for patient inclusion and exclusion criteria, randomization, blinding of patients and physicians, controls, duration of the trial, investigator training, responses, samples size, study dropouts, statistical analyses, data management, and safety monitoring. Delphi and nominal group techniques were used. Results. Briefly, patients with diffuse cutaneous SSc of less than 24 months' duration should be included because they are at greatest risk for the development of severe organ damage and death. Patients should be excluded if they have other connective tissue diseases, SSc‐like illnesses related to exposures or ingestions, severe existing internal organ damage, an unacceptable risk of side effects, or concurrent therapies that might independently influence the outcome. Randomized, double‐blind, placebo‐controlled trials are preferred. The treatment and followup period must be long enough to permit observation of any disease modification, which is likely to require 18–36 months, unless an extraordinarily effective therapy is identified. Responses selected should be quantitative, consistently and accurately reflect activity of SSc in major target organs (not solely the skin), be sensitive to change, and be standardized, with limited variability. An example of a set of responses is given. Surrogate responses are desirable, but none have been validated as correlating with organ damage. Conclusion. Guidelines have been established for trials of disease‐modifying interventions in SSc. These guidelines will need to be altered as additional information becomes available. Any given protocol will be individualized based on the nature of the intervention and objectives of the study. Nonetheless, each study team should develop a protocol that meets the spirit of these guidelines.

Original languageEnglish (US)
Pages (from-to)351-360
Number of pages10
JournalArthritis & Rheumatism
Volume38
Issue number3
DOIs
StatePublished - Jan 1 1995

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Systemic Scleroderma
Clinical Trials
Guidelines
Safety Management
Diffuse Scleroderma
Statistical Data Interpretation
Connective Tissue Diseases
Therapeutics
Random Allocation
Sample Size
Eating
Research Personnel
Observation
Physicians
Skin

All Science Journal Classification (ASJC) codes

  • Immunology and Allergy
  • Rheumatology
  • Immunology
  • Pharmacology (medical)

Cite this

White, B., Bauer, E. A., Goldsmith, L. A., Hochberg, M. C., Katz, L. M., Korn, J. H., ... Steen, V. D. (1995). Guidelines for clinical trials in systemic sclerosis (scleroderma). Arthritis & Rheumatism, 38(3), 351-360. https://doi.org/10.1002/art.1780380309

Guidelines for clinical trials in systemic sclerosis (scleroderma). / White, Barbara; Bauer, Eugene A.; Goldsmith, Lowell A.; Hochberg, Marc C.; Katz, Linda M.; Korn, Joseph H.; Lachenbruch, Peter A.; Carwile Leroy, E.; Mitrane, Margaret P.; Paulus, Harold E.; Postlethwaite, Arnold; Steen, Virginia D.

In: Arthritis & Rheumatism, Vol. 38, No. 3, 01.01.1995, p. 351-360.

Research output: Contribution to journalArticle

White, B, Bauer, EA, Goldsmith, LA, Hochberg, MC, Katz, LM, Korn, JH, Lachenbruch, PA, Carwile Leroy, E, Mitrane, MP, Paulus, HE, Postlethwaite, A & Steen, VD 1995, 'Guidelines for clinical trials in systemic sclerosis (scleroderma)', Arthritis & Rheumatism, vol. 38, no. 3, pp. 351-360. https://doi.org/10.1002/art.1780380309
White B, Bauer EA, Goldsmith LA, Hochberg MC, Katz LM, Korn JH et al. Guidelines for clinical trials in systemic sclerosis (scleroderma). Arthritis & Rheumatism. 1995 Jan 1;38(3):351-360. https://doi.org/10.1002/art.1780380309
White, Barbara ; Bauer, Eugene A. ; Goldsmith, Lowell A. ; Hochberg, Marc C. ; Katz, Linda M. ; Korn, Joseph H. ; Lachenbruch, Peter A. ; Carwile Leroy, E. ; Mitrane, Margaret P. ; Paulus, Harold E. ; Postlethwaite, Arnold ; Steen, Virginia D. / Guidelines for clinical trials in systemic sclerosis (scleroderma). In: Arthritis & Rheumatism. 1995 ; Vol. 38, No. 3. pp. 351-360.
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abstract = "Objective. To develop guidelines for therapeutic trials designed to improve the overall course of systemic sclerosis (SSc), that is, to reduce the development of significant organ damage or death. Methods. A committee developed general guidelines for patient inclusion and exclusion criteria, randomization, blinding of patients and physicians, controls, duration of the trial, investigator training, responses, samples size, study dropouts, statistical analyses, data management, and safety monitoring. Delphi and nominal group techniques were used. Results. Briefly, patients with diffuse cutaneous SSc of less than 24 months' duration should be included because they are at greatest risk for the development of severe organ damage and death. Patients should be excluded if they have other connective tissue diseases, SSc‐like illnesses related to exposures or ingestions, severe existing internal organ damage, an unacceptable risk of side effects, or concurrent therapies that might independently influence the outcome. Randomized, double‐blind, placebo‐controlled trials are preferred. The treatment and followup period must be long enough to permit observation of any disease modification, which is likely to require 18–36 months, unless an extraordinarily effective therapy is identified. Responses selected should be quantitative, consistently and accurately reflect activity of SSc in major target organs (not solely the skin), be sensitive to change, and be standardized, with limited variability. An example of a set of responses is given. Surrogate responses are desirable, but none have been validated as correlating with organ damage. Conclusion. Guidelines have been established for trials of disease‐modifying interventions in SSc. These guidelines will need to be altered as additional information becomes available. Any given protocol will be individualized based on the nature of the intervention and objectives of the study. Nonetheless, each study team should develop a protocol that meets the spirit of these guidelines.",
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