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

NCT02299297 | Completed Phase 2 Results FDA Drug

• 1 other identifier: AAAO1502
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Alopecia Areata

Keywords

Alopecia Areata

Outcome Measures

Primary Outcomes (1)

• Total Number of Responders

  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.

  Baseline up to between 24 and 72 weeks

Secondary Outcomes (4)

• Total Number of Responders Maintaining Response During the Post-Treatment Follow Up Period

  Week 24

• Total Number of Responders With Change in PHYSICIAN Global Assessment Score

  Up to 24 weeks

• Percentage of Regrowth

  Baseline up to between 24 and 72 weeks

• Dermatology Life Quality Index (DLQI) Score

  Up to 24 weeks

Study Arms (1)

Tofacitinib

EXPERIMENTAL

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.

Drug: Tofacitinib

Interventions

Tofacitinib DRUG

Dosage/Frequency: 5mg - 10mg, oral, twice daily

Also known as: XELJANZ

Tofacitinib

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients between 18 and 65 years of age.
• Patients with a diagnosis of moderate to severe patch type alopecia areata.
• Patients with alopecia totalis or universalis may be enrolled.
• Patients have \>30% and \<95% total scalp hair loss at baseline as measured using the SALT score to qualify as moderate to severe patch type AA; and 95-100% scalp hair loss to qualify as alopecia totalis or universalis.
• Duration of hair loss greater than 3 months without an upper limit of duration as long as there is reason to believe that regrowth is possible in the opinion of the investigator.
• No evidence of significant active ongoing regrowth present at baseline.
• Patients with a history of alopecia totalis/universalis can be included as long as the current episode of hair loss meets the criteria of 30 to 95% hair loss (i.e. they are not currently AT or AU), and as long as in the opinion of the investigator there does appear to be potential for regrowth. Patients with current episodes of alopecia totalis/universalis may be included in this study.
• Vaccinations should be up to date in agreement with current immunization guidelines prior to start of tofacitinib. The patient will be asked to obtain verbal verification from their primary care provider that this is the case.
• Patients may be naïve to treatment or unresponsive to intralesional (IL) steroids or other treatments for alopecia areata.
• Women of childbearing potential (WOCBP) must use highly effective methods of birth control \[for at least 12 weeks after the last dose of investigational product\] to minimize the risk of pregnancy. WOCBP must follow instructions for birth control for the entire duration of the study including a minimum of 90 days after dosing has been completed.
• Acceptable methods of highly effective birth control include:
• Condom with spermicide
• Diaphragm and spermicide
• Cervical cap and spermicide The use of intrauterine devices, (IUDs) shall be at the discretion of the investigator.
• Women must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) within 24 hours prior to the start of investigational product.

You may not qualify if:

• Sex and Reproductive Status
• WOCBP who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for up to 12 weeks after the last dose of study drug.
• WOCBP using a prohibited contraceptive method.
• Women who are pregnant or breastfeeding.
• Women with a positive pregnancy test on enrollment or before administration of tofacitinib.
• Sexually active fertile men not using effective birth control if their partners are WOCBP.
• Target Disease Exceptions
• Patients with current alopecia totalis/universalis (i.e. 100% scalp or 100% scalp and body loss, respectively) may be enrolled.
• Patients with a history of or active skin disease on the scalp such as psoriasis or seborrheic dermatitis.
• Patients in whom the diagnosis of alopecia areata is in question or in whom the pattern of hair loss is such that quantification of hair loss and assessment of regrowth is difficult. E.g patients with diffuse alopecia areata. This assessment is at the investigator's discretion.
• Patients with active medical conditions or malignancies (except adequately treated basal or squamous cell carcinoma) that in the opinion of the investigator would increase the risks associated with study participation, including patients with a history of recurrent infections.
• Patients with hemoglobin levels \<9 g/dL, lymphocyte count \<500 cells/mm3, absolute neutrophil count (ANC) \<1000 cells/mm3, at baseline.
• Patients known to be HIV or hepatitis B or C positive.
• Patients with history or evidence of moderate or severe hepatic and/or renal impairment.
• Patients with history of immunosuppression or history of recurrent serious infections.

Sponsors & Collaborators

• Columbia University: lead
• Locks of Love: collaborator

Study Sites (1)

Columbia University Medical Center

New York, New York, 10032, United States

Location

Related Publications (1)

• Jabbari A, Sansaricq F, Cerise J, Chen JC, Bitterman A, Ulerio G, Borbon J, Clynes R, Christiano AM, Mackay-Wiggan J. An Open-Label Pilot Study to Evaluate the Efficacy of Tofacitinib in Moderate to Severe Patch-Type Alopecia Areata, Totalis, and Universalis. J Invest Dermatol. 2018 Jul;138(7):1539-1545. doi: 10.1016/j.jid.2018.01.032. Epub 2018 Feb 13.

  PMID: 29452121 DERIVED

MeSH Terms

Conditions

Alopecia Areata

Interventions

tofacitinib

Condition Hierarchy (Ancestors)

Alopecia; Hypotrichosis; Hair Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Grace Ulerio, CCRC
• Organization: Columbia University Department of Dermatology Clinical Research Unit

gu2102@cumc.columbia.edu

212-305-6593

Study Officials

• Julian Mackay-Wiggan, MD, MS

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2014
• First Posted: November 24, 2014
• Study Start: January 1, 2015
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: May 7, 2019
• Results First Posted: May 7, 2019

Record last verified: 2019-04

Data Sharing

• IPD Sharing: Will share
• Time Frame: At the end of the study.
• Access Criteria: The data is available in the publication for this study.

Locations

US (1)