Trial Outcomes & Findings for Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis (NCT NCT02299297)

Last Updated: 2019-05-07

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Baseline up to between 24 and 72 weeks

Results posted on

2019-05-07

Participant Flow

Participant milestones

Participant milestones
Measure	Tofacitinib 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Overall Study STARTED	12
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tofacitinib n=12 Participants 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline up to between 24 and 72 weeks

Outcome measures

Outcome measures
Measure	Tofacitinib n=12 Participants 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Total Number of Responders	8 Participants

SECONDARY outcome

Timeframe: Week 24

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. Durability of response was measured by comparing SALT scores from baseline to 24 weeks after treatment.

Outcome measures

Outcome measures
Measure	Tofacitinib n=8 Participants 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Total Number of Responders Maintaining Response During the Post-Treatment Follow Up Period	3 Participants

SECONDARY outcome

Timeframe: Up to 24 weeks

A physician's assessment of the severity of disease based on a 6-point scale (score of 0 = clear and 5 = very severe). Responders are defined by participants who exhibited regrowth.

Outcome measures

Outcome measures
Measure	Tofacitinib n=8 Participants 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Total Number of Responders With Change in PHYSICIAN Global Assessment Score	8 Participants

SECONDARY outcome

Timeframe: Baseline up to between 24 and 72 weeks

Percentage of regrowth was measured by comparing the SALT score at the beginning and end of treatment.

Outcome measures

Outcome measures
Measure	Tofacitinib n=12 Participants 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Percentage of Regrowth	56.8 percentage of regrowth Interval 12.1 to 100.0

SECONDARY outcome

Timeframe: Up to 24 weeks

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.

Outcome measures

Outcome measures
Measure	Tofacitinib n=12 Participants 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Dermatology Life Quality Index (DLQI) Score Baseline	6.5 score on a scale Standard Deviation 5.0
Dermatology Life Quality Index (DLQI) Score 3 months	5.2 score on a scale Standard Deviation 6.7
Dermatology Life Quality Index (DLQI) Score 6 months	6 score on a scale Standard Deviation 6.9

Adverse Events

Tofacitinib

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Tofacitinib n=12 participants at risk 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. Tofacitinib: Dosage/Frequency: 5mg - 10mg, oral, twice daily
Infections and infestations Upper respiratory infection	91.7% 11/12 • Number of events 11 • Up to 18 months from the start of treatment
Renal and urinary disorders increased bowel movement frequency	33.3% 4/12 • Number of events 4 • Up to 18 months from the start of treatment
Blood and lymphatic system disorders Blood on urinalysis	33.3% 4/12 • Number of events 4 • Up to 18 months from the start of treatment
Metabolism and nutrition disorders Loose stools	25.0% 3/12 • Number of events 3 • Up to 18 months from the start of treatment
General disorders Mild acne	25.0% 3/12 • Number of events 3 • Up to 18 months from the start of treatment
Metabolism and nutrition disorders Weight gain	16.7% 2/12 • Number of events 2 • Up to 18 months from the start of treatment
Infections and infestations Asymptomatic bacteriuria	16.7% 2/12 • Number of events 2 • Up to 18 months from the start of treatment
Vascular disorders Hypertensive urgency	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
General disorders Bloating	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
Metabolism and nutrition disorders Constipation	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
General disorders Dizziness	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
General disorders Headache	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
Nervous system disorders Neuropathic pain	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
Reproductive system and breast disorders Vaginal spotting (post menopause)	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
Eye disorders Conjunctivitis	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
Renal and urinary disorders Urinary retention	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment
Hepatobiliary disorders Transaminitis	8.3% 1/12 • Number of events 1 • Up to 18 months from the start of treatment

Additional Information

Grace Ulerio, CCRC

Columbia University Department of Dermatology Clinical Research Unit

Phone: 212-305-6593

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place