Clinical Trial: Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: An Open-Label Pilot Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis

Brief Summary: This is an open-label pilot study of tofacitinib taken daily for 6 months in the treatment of moderate to severe AA, and alopecia totalis or universalis, followed by 6 months follow-up off drug to assess the incidence and timing of recurrence of disease or documentation of delayed response to treatment. There will be the option of increasing the treatment duration up to an additional 6 months beyond the initially scheduled 6 months of treatment, if clinically indicated, and at the discretion of the investigator.

Detailed Summary:

Alopecia areata (AA) is a common disease of the immune system, known as an "autoimmune" disease. In the disease, the immune system mistakenly destroys the hair follicle, causing hair to fall out. Despite many people having this disease, research into its cause and new, better ways to treat AA has lagged far behind other similar diseases of the immune system. Currently, there are no Federal Drug Administration approved drugs for AA.

Tofacitinib (made by Pfizer) is an intervention known to effectively treat a disease of the joints, known as rheumatoid arthritis. It is also being studied in the treatment of psoriasis, another "autoimmune" disease, by fighting inflammation. There are some genetic and chemical similarities between those with active rheumatoid arthritis, psoriasis, and AA,suggesting that treatment with the same drug is likely to be effective. In mice specially designed for testing drugs for the treatment of human alopecia, this medication worked to prevent the disease AA from starting in mice that would have otherwise developed the disease. To test Tofacitinib, we are going to treat 15 patients with moderate to severe AA for up to 6 months. This is an "open label" study, meaning that there will not be a placebo group; all patients enrolled in the study will receive the active medication. The effectiveness of the medication will be measured by changes in hair re-growth as determined by physical exam and photography, as well as by patient and physician scoring. After the treatment period of up to 6 months is completed, There will be the option of increasing the treatment duration up to an additional 6 months beyond the initially scheduled 6 months of treatment, if clinically indicated, and at the discretion of the investigator. Patients will be followed for another 6 months off of the drug to see if the effects of treatment last and
Sponsor: Julian M. Mackay-Wiggan

Current Primary Outcome: Change in SALT Score [ Time Frame: Baseline up to Week 48 ]

The primary efficacy endpoint will be the proportion of responders after 6 months of treatment, with response defined as 50% or greater hair re-growth from baseline as assessed by SALT score up to 6 months.


Original Primary Outcome: Change in SALT Score [ Time Frame: Baseline up to Week 48 ]

The study's primary efficacy endpoint will be the proportion of responders after 6 months of treatment, with response defined as 50% or greater hair re-growth from baseline as assessed by SALT score up to 6 months.


Current Secondary Outcome:

  • Change in Percentage of Hair Loss [ Time Frame: Baseline up to Week 48 ]
    As additional secondary endpoints, efficacy will be measured by changes in hair re-growth as a continuous variable as determined by physical exam and Canfield photography, as well as patient and physician global evaluation scores. To assess the durability of responses, patients will continue to be followed for an additional 6 months off treatment.
  • Change in Patient Global Assessment [ Time Frame: Weeks 12 and 24 ]
    Change in patient global assessment between baseline and 6 months
  • Change in Physician Global Assessment (PGA) Score [ Time Frame: Baseline up to Week 48 ]
    Change in PGA (Physician Global Assessment) based on live evaluations and evaluation of standardized photographs between baseline and month 6.
  • Patient Quality of Life Assessment Score [ Time Frame: Weeks 12 and 24 ]
    Change in patient quality of life assessment at weeks 12 and 24.
  • Incidence of Adverse Effects [ Time Frame: Baseline up to Week 48 ]
    Safety will be assessed by summarizing the incidence and type of adverse events. The proportion of patients who discontinued treatment will be summarized.


Original Secondary Outcome: Same as current

Information By: Columbia University

Dates:
Date Received: November 14, 2014
Date Started: January 2015
Date Completion: June 2017
Last Updated: February 14, 2017
Last Verified: February 2017