Alopecia AreataPhase 2NCT02299297
Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis
A Phase 2 study testing Tofacitinib in people with Alopecia Areata, run by Columbia University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Alopecia Areata
- What is being tested?
- Tofacitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2015to Dec 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
- Women must not be breastfeeding.
Who cannot join
- 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.
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- 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.
- Coexisting disease or concurrent medications
- Patients taking potent inhibitors of CYP3A4 (e.g.ketoconazole).
- Patients receiving one or more concomitant medications that result in both moderate inhibition of CYP3A4 and potent inhibition of CYP2C19 (e.g., fluconazole).
- Patients known to be HIV or hepatitis B or C positive.
- Patients with evidence of infection or active/untreated skin cancer.
- Patients who have been treated with intralesional steroids, systemic steroids, anthralin, squaric acid, diphenylcyclopropenone (DPCP), protopic, minoxidil or other medication which in the opinion of the investigator may affect hair regrowth within one month of the baseline visit.
- Patients who are impaired, incapacitated, or incapable of completing study-related assessments.
- Patients with current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematologic, gastrointestinal, pulmonary, cardiac, neurologic, or cerebral disease, which, in the opinion of the investigator, might place a subject at unacceptable risk for participation in the study.
- Female patients who have had a breast cancer screening that is suspicious for malignancy and in whom the possibility of malignancy cannot be reasonably excluded by additional clinical, laboratory, or other diagnostic evaluations.
- Patients with a history of cancer in the last 5 years, other than non-melanoma skin cell cancers cured by local resection or carcinoma in situ. Existing non-melanoma skin cell cancers should be removed, the lesion site healed, and residual cancer ruled out before administration of the study drug.
- Patients who currently abuse drugs or alcohol.
- Patients with evidence (as assessed by the investigator) of active or latent bacterial or viral infections at the time of potential enrollment, including subjects with evidence of human immunodeficiency virus (HIV) detected during screening.
- Patients with herpes zoster or cytomegalovirus (CMV) that resolved less than 2 months before the informed consent document was signed.
- Patients who have received any live vaccines within 3 months of the anticipated first dose of study medication. Subjects may not receive live vaccine concurrently with tofacitinib.
- Patients with a history or symptoms suggestive of gastrointestinal perforation or disorders that might increase the risk of GI perforation such as gastric ulcers or diverticulitis.
- Patients who take NSAIDs at high dose or on a frequent basis, which in the investigator's opinion might increase the risks of gastrointestinal perforation.
- Patients with any serious bacterial infection within the last 3 months, unless treated and resolved with antibiotics, or any chronic bacterial infection (e.g., chronic pyelonephritis, osteomyelitis, or bronchiectasis).
- Patients at risk for tuberculosis (TB). Specifically excluded from this study will be subjects with a history of active TB within the last 3 years, even if it was treated; a history of active TB greater than 3 years ago, unless there is documentation that the prior anti-TB treatment was appropriate in duration and type; current clinical, radiographic, or laboratory evidence of active TB; and latent TB that was not successfully treated (4 weeks).
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Medical Center
New York, New York, 10032
About this study
Official title: An Open-Label Pilot Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis
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.
Read the detailed description
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.
- Conditions
- Alopecia Areata
- Being tested
- Tofacitinib
- Keywords
- Alopecia Areata
- Medical subject headings
- Alopecia Areata
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TofacitinibDrug
Also called XELJANZ
Dosage/Frequency: 5mg - 10mg, oral, twice daily
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Tofacitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline up to between 24 and 72 weeks
Secondary outcomes (4)
Total Number of Responders Maintaining Response During the Post-Treatment Follow Up Period
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.
Measured: Week 24
Total Number of Responders With Change in PHYSICIAN Global Assessment Score
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.
Measured: Up to 24 weeks
Percentage of Regrowth
Percentage of regrowth was measured by comparing the SALT score at the beginning and end of treatment.
Measured: Baseline up to between 24 and 72 weeks
Dermatology Life Quality Index (DLQI) Score
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.
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2014 (estimated)
- Study startJan 2015
- Primary completionDec 2017
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alopecia AreataStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- Locks of Love
- Responsible party
- Sponsor
- Investigators
- Julian Mackay-Wiggan, MD, MS, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02299297
- Sponsor's protocol ID
- AAAO1502
- Status last verified
- Apr 2019
- First submitted
- 14 Nov 2014
- Last updated
- 7 May 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual data will be included in the publications, but will not be identifiable.
Publications
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.
PubMed 29452121
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Pilot Study To Evaluate The Efficacy Of Tofacitinib In Moderate To Severe Alopecia Areata, Totalis And Universalis
- ClinicalTrials.gov ID
- NCT02299297
- Acronym / sponsor ID
- AAAO1502
- Conditions
- Alopecia Areata
- Keywords
- Alopecia Areata
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- Locks of Love
- Study start
- Jan 2015 (actual)
- Primary completion
- Dec 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 24 Nov 2014 (estimated)
- Results
- Posted 7 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tofacitinib | Experimental | Drug: TofacitinibTofacitinib 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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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.
Key exclusion criteria (33 listed)
- Sex and Reproductive Status
- Target Disease Exceptions
- Coexisting disease or concurrent medications
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TofacitinibAlso: XELJANZDosage/Frequency: 5mg - 10mg, oral, twice daily | Drug | Tofacitinib |
Primary outcome
Total Number of Responders
Time frame: Baseline up to between 24 and 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.
4 secondary outcomes
Total Number of Responders Maintaining Response During the Post-Treatment Follow Up Period
Time frame: Week 24
Total Number of Responders With Change in PHYSICIAN Global Assessment Score
Time frame: Up to 24 weeks
Percentage of Regrowth
Time frame: Baseline up to between 24 and 72 weeks
Dermatology Life Quality Index (DLQI) Score
Time frame: Up to 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 29452121Jabbari 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.
- Individual participant dataSharing planned: Yes. Individual data will be included in the publications, but will not be identifiable.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Julian Mackay-Wiggan, MD, MS principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tofacitinib in people with Alopecia Areata, run by Columbia University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 33 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total Number of Responders (time frame: Baseline up to between 24 and 72 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02299297, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2019.