A Randomized, Placebo-Controlled Clinical Trial of Efficacy and Safety: Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis

Marina G. Silveira, Andrea A. Gossard, Alisha C. Stahler, Roberta A. Jorgensen, Janice L. Petz, Ahmad H. Ali, Keith Lindor

Research output: Contribution to journalArticle

14 Citations (Scopus)

Abstract

BACKGROUND AND AIMS: Fatigue is a common symptom of primary biliary cirrhosis (PBC), and is associated with an impaired quality of life.

STUDY QUESTION: No studies have assessed the use of modafinil in fatigue related to PBC in a controlled manner.

STUDY DESIGN, MEASURES, AND OUTCOMES: A randomized, double-blind, placebo-controlled study was conducted to determine the safety and efficacy of modafinil for the treatment of fatigue in PBC. Forty patients were randomized to modafinil (n = 20) or placebo (n = 20) for 12 weeks. A verbal report of fatigue for at least 6 months was required for enrollment. Modafinil was administered at 100 mg by mouth once daily; a change by 50 mg every 2 weeks (maximum: 200 mg once daily) was allowed, depending on the subject's response to treatment. The primary outcome was defined as a ≥50% improvement in fatigue severity [quantified by the Fisk Fatigue Impact Scale (FFIS)] after 12 weeks of treatment, compared with baseline values.

RESULTS: Thirty-three PBC patients completed the study. After 12 weeks of therapy, only 5 patients had a ≥50% reduction in FFIS scores: 3 patients (17.6%) in the modafinil arm and 2 (12.5%) in the placebo arm (P = 1.00). Change in median FFIS score was not statistically different between patients in the 2 treatment groups (P = 0.36). Modafinil was associated with minimal adverse events (headaches, diarrhea, and rash).

CONCLUSIONS: In patients with PBC who have fatigue, treatment with modafinil for 12 weeks was safe and fairly well tolerated; however, it did not result in beneficial effects on fatigue compared with patients treated with placebo (CONSORT Table 1). ClinicalTrials.gov identifier NCT00943176.

Original languageEnglish (US)
Pages (from-to)e167-e176
JournalAmerican Journal of Therapeutics
Volume24
Issue number2
DOIs
StatePublished - Mar 1 2017

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Biliary Liver Cirrhosis
Fatigue
Randomized Controlled Trials
Placebos
Safety
Therapeutics
Arm
modafinil
Exanthema
Headache
Mouth
Diarrhea
Quality of Life
Outcome Assessment (Health Care)

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

Cite this

A Randomized, Placebo-Controlled Clinical Trial of Efficacy and Safety : Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis. / Silveira, Marina G.; Gossard, Andrea A.; Stahler, Alisha C.; Jorgensen, Roberta A.; Petz, Janice L.; Ali, Ahmad H.; Lindor, Keith.

In: American Journal of Therapeutics, Vol. 24, No. 2, 01.03.2017, p. e167-e176.

Research output: Contribution to journalArticle

Silveira, Marina G. ; Gossard, Andrea A. ; Stahler, Alisha C. ; Jorgensen, Roberta A. ; Petz, Janice L. ; Ali, Ahmad H. ; Lindor, Keith. / A Randomized, Placebo-Controlled Clinical Trial of Efficacy and Safety : Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis. In: American Journal of Therapeutics. 2017 ; Vol. 24, No. 2. pp. e167-e176.
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abstract = "BACKGROUND AND AIMS: Fatigue is a common symptom of primary biliary cirrhosis (PBC), and is associated with an impaired quality of life.STUDY QUESTION: No studies have assessed the use of modafinil in fatigue related to PBC in a controlled manner.STUDY DESIGN, MEASURES, AND OUTCOMES: A randomized, double-blind, placebo-controlled study was conducted to determine the safety and efficacy of modafinil for the treatment of fatigue in PBC. Forty patients were randomized to modafinil (n = 20) or placebo (n = 20) for 12 weeks. A verbal report of fatigue for at least 6 months was required for enrollment. Modafinil was administered at 100 mg by mouth once daily; a change by 50 mg every 2 weeks (maximum: 200 mg once daily) was allowed, depending on the subject's response to treatment. The primary outcome was defined as a ≥50{\%} improvement in fatigue severity [quantified by the Fisk Fatigue Impact Scale (FFIS)] after 12 weeks of treatment, compared with baseline values.RESULTS: Thirty-three PBC patients completed the study. After 12 weeks of therapy, only 5 patients had a ≥50{\%} reduction in FFIS scores: 3 patients (17.6{\%}) in the modafinil arm and 2 (12.5{\%}) in the placebo arm (P = 1.00). Change in median FFIS score was not statistically different between patients in the 2 treatment groups (P = 0.36). Modafinil was associated with minimal adverse events (headaches, diarrhea, and rash).CONCLUSIONS: In patients with PBC who have fatigue, treatment with modafinil for 12 weeks was safe and fairly well tolerated; however, it did not result in beneficial effects on fatigue compared with patients treated with placebo (CONSORT Table 1). ClinicalTrials.gov identifier NCT00943176.",
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AU - Ali, Ahmad H.

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AB - BACKGROUND AND AIMS: Fatigue is a common symptom of primary biliary cirrhosis (PBC), and is associated with an impaired quality of life.STUDY QUESTION: No studies have assessed the use of modafinil in fatigue related to PBC in a controlled manner.STUDY DESIGN, MEASURES, AND OUTCOMES: A randomized, double-blind, placebo-controlled study was conducted to determine the safety and efficacy of modafinil for the treatment of fatigue in PBC. Forty patients were randomized to modafinil (n = 20) or placebo (n = 20) for 12 weeks. A verbal report of fatigue for at least 6 months was required for enrollment. Modafinil was administered at 100 mg by mouth once daily; a change by 50 mg every 2 weeks (maximum: 200 mg once daily) was allowed, depending on the subject's response to treatment. The primary outcome was defined as a ≥50% improvement in fatigue severity [quantified by the Fisk Fatigue Impact Scale (FFIS)] after 12 weeks of treatment, compared with baseline values.RESULTS: Thirty-three PBC patients completed the study. After 12 weeks of therapy, only 5 patients had a ≥50% reduction in FFIS scores: 3 patients (17.6%) in the modafinil arm and 2 (12.5%) in the placebo arm (P = 1.00). Change in median FFIS score was not statistically different between patients in the 2 treatment groups (P = 0.36). Modafinil was associated with minimal adverse events (headaches, diarrhea, and rash).CONCLUSIONS: In patients with PBC who have fatigue, treatment with modafinil for 12 weeks was safe and fairly well tolerated; however, it did not result in beneficial effects on fatigue compared with patients treated with placebo (CONSORT Table 1). ClinicalTrials.gov identifier NCT00943176.

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