Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis
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|ClinicalTrials.gov Identifier: NCT02299297|
Recruitment Status : Active, not recruiting
First Posted : November 24, 2014
Last Update Posted : August 1, 2017
|Condition or disease||Intervention/treatment||Phase|
|Alopecia Areata||Drug: Tofacitinib||Phase 2|
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 if there is delayed response. The safety of the medication, Tofacitinib, in patients with AA will also be evaluated. Blood work will be collected before medication is started, during the treatment period and after Tofacitinib is stopped in order to monitor for adverse effects of the medication. Small scalp biopsies and peripheral blood will be taken at the beginning of the study before treatment and at weeks 4 and 24. Additional, optional scalp biopsies and blood draws may be suggested at other important time points. The chemical analysis of these skin samples and blood will help us to understand how the disease happens, how the treatment works, and even guide us to better treatments in the future.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Pilot Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis|
|Actual Study Start Date :||January 2015|
|Estimated Primary Completion Date :||November 2017|
|Estimated Study Completion Date :||November 2017|
Tofacitinib will be self-administered for 6 months, with the option to extend treatment up to an additional 6 months at the discretion of the principal investigator. Patients will then be followed for 6 months off the drug to assess the incidence and timing of recurrence of disease or documentation of delayed response to treatment.
Dosage/Frequency: 5mg - 10mg, oral, twice daily
Other Name: XELJANZ
- Proportion of responders at the end of treatment period [ Time Frame: Baseline, during the first 24 to 72 weeks ]This is defined as 50% or greater hair re-growth from baseline as assessed by the Severity of ALopecia Tool (SALT) score after up to 24 weeks/6 months to 72 weeks/18 months of treatment. This is a relatively strict definition for defining responders and non-responders and was chosen to minimize the potential for spontaneous remission, in which fewer than 10% are expected to achieve this magnitude of hair regrowth spontaneously.
- Proportion of responders during the follow up period [ Time Frame: 8, 16, and 24 weeks post-treatment ]To assess the durability of responses, patients who achieve 50% regrowth from baseline during the first 6 to 18 months, will continue to be followed for an additional 6 months post-treatment or until it is determined that relapse has occurred.
- Change in PHYSICIAN Global Assessment Score [ Time Frame: Baseline, end of treatment (up to 72 weeks), up to 24 weeks post-treatment ]A physician's assessment of the severity of disease based on a 6-point scale (score of 0 = clear and 5 = very severe).
- Change in PATIENT Global Assessment Score [ Time Frame: Baseline, end of treatment (up to 72 weeks), up to 24 weeks post-treatment ]A widely used patient-reported outcomes (PROs) that reflect the patient's perspective of their overall health and medical condition.
- Change in Dermatology Life Quality Index (DLQI) Score [ Time Frame: Baseline, end of treatment (up to 72 weeks), up to 24 weeks post-treatment ]The first dermatology-specific Quality of Life instrument. It is a simple 10-question validated questionnaire used to evaluate patient's quality of life. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
- Change in Skindex-29 Questionnaire Score [ Time Frame: Baseline, end of treatment (up to 72 weeks), up to 24 weeks post-treatment ]a disease-specific questionnaire that comprehensively assesses the effects of skin diseases on patient's quality of life. it inquires about how often (Never, Rarely, Sometimes, Often, All the time) during the previous four weeks the patient experienced the effect described in each item. Seven items address the Symptoms domain, ten items the Emotional domain, and twelve items the Functioning domain. All responses are transformed to a linear scale of 100, varying from 0 (no effect) to 100 (effect experienced all the time). Scores are reported as three scale scores, corresponding to the three domains; a scale score is the average of a patient's responses to items in a given domain.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02299297
|United States, New York|
|Columbia University Medical Center|
|New York, New York, United States, 10032|
|Principal Investigator:||Julian Mackay-Wiggan, MD, MS||Columbia University|