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Cicatricial AlopeciaPhase 2NCT05076006

Dual JAK1/TYK2 Inhibitor for Cicatricial Alopecia

A Phase 2 study testing PF-06700841 in people with Cicatricial Alopecia, run by Emma Guttman at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Alopecia could be subdivided into two main groups of diseases: non-scarring alopecia, such as male pattern baldness, or alopecia areata (AA), in which hair follicles are preserved, yet quiescent, and scarring alopecia, also known as cicatricial alopecia (CA), in which hair follicles are irreversibly destroyed. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cicatricial Alopecia
What is being tested?
PF-06700841
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
51 participants
Where
1 siteUnited States
Study dates
19 May 2021to 30 Sep 2023
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Subjects of any gender, age 18 years or older, at the time of informed consent at Screening
  • Subjects who are willing and able to adhere to the study visit schedule and comply with protocol requirements.
  • Subject self-reports active CA (LPP/FFA or CCCA). for at least 6 months from screening visit. Diagnosis will be confirmed clinically at screening visit.
  • Subject has a negative Tuberculin purified protein derivative (PPD) or QuantiFERON TB-Gold test (QFT) at screening.
  • Subject is judged to be in otherwise good overall health following a detailed medical and medication history, physical examination, and laboratory testing.

Who cannot join

  • Subject's cause of hair loss is indeterminable and/or they have concomitant causes of alopecia, such pregnancy-related, drug-induced, telogen effluvium, or advanced androgenetic alopecia.
  • Subject has a history of CA for ≥ 7 years since their disease onset, severe fibrosing disease, or very rapid hair loss.
  • Subject has a history of moderate to severe keloids on the scalp, as determined by clinical examination at screening.
  • Other scalp disease that may impact assessment (eg, scalp psoriasis, dermatitis, etc).
  • Subject is pregnant or breastfeeding. Female subjects of childbearing potential must agree to use two effective methods (one of which is a highly effective method) of contraception throughout the study and for at least 28 days after the last dose of investigational product. Women of childbearing potential must test negative for pregnancy prior to enrollment in this study.
  • Participation in other studies involving investigational drug(s) within 8 weeks or within 5 half-lives (if known), whichever is longer, prior to study entry and/or during study participation.
Show all 31Show fewer
  • Active systemic diseases that may cause hair loss (eg, systemic lupus erythematosus, thyroiditis, systemic sclerosis, lichen planus, etc).
  • Any Psychiatric condition in the opinion of the investigator precludes participation in the study .
  • Current or recent history of clinically significant severe, progressive, or uncontrolled renal (including but not limited to active renal disease or recent kidney stones), hepatic, hematological, gastrointestinal, metabolic, endocrine (particularly thyroid disease which can be associated with hair loss), pulmonary, cardiovascular, psychiatric, immunologic/rheumatologic or neurologic disease; or have any other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration, or interfere with the interpretation of study results; or in the opinion of the investigator, the subject is inappropriate for entry into this study, or unwilling/unable to comply with STUDY PROCEDURES.
  • Any present malignancies or history of malignancies with the exception of adequately treated or excised non-metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ.
  • History of any lymphoproliferative disorder such as Epstein Barr Virus (EBV) related lymphoproliferative disorder, history of lymphoma, history of leukemia, or signs and symptoms suggestive of current lymphatic or lymphoid disease.
  • History (single episode) of disseminated herpes zoster or disseminated herpes simplex, or a recurrent (more than one episode of) localized, dermatomal herpes zoster.
  • History of systemic infection requiring hospitalization, parenteral antimicrobial therapy, or as otherwise judged clinically significant by the investigator within 6 months prior to Day 0.
  • Active acute or chronic infection requiring treatment with oral antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 4 weeks prior to Day 0 or superficial skin infection within 1 week prior to Day 0.
  • Significant trauma or major surgery within 1 month of signing informed consent.
  • Considered in imminent need for surgery or with elective surgery scheduled to occur during the study.
  • Active hepatitis B, hepatitis C, human immunodeficiency virus (HIV), or positive HIV serology at the time of screening for subjects determined by the investigators to be at high-risk for this disease.
  • Have an active history of alcohol or substance abuse within 1 year prior to Day 0.
  • Donation of blood in excess of 500 mL within 8 weeks prior to Day 0.
  • Subject has received a live attenuated vaccine ≤ 30 days prior to study screening.
  • Subject has any uncertain or clinically significant laboratory abnormalities that may affect interpretation of study data or endpoints, at determined by the PI.
  • History of adverse systemic or allergic reactions to components of study drug.
  • Use of systemic immunosuppressive medications, including, but not limited to, cyclosporine, systemic or intralesional corticosteroids, mycophenolate mofetil, azathioprine, methotrexate, within 8 weeks prior to baseline visit.
  • Use of other systemic agent for CA, including, 5α-reductase inhibitors, hydroxychloroquine, or retinoids, within 4 weeks prior to baseline visit.
  • Use of an intralesional corticosteroids or oral JAK inhibitor (tofacitinib, ruxolitinib) within 4 weeks prior to the baseline visit.
  • Subject has used topical corticosteroids, and/or tacrolimus, and/or pimecrolimus within 1 week before the baseline visit.
  • Subject has been previously treated with biological drugs in the last 12 weeks for other indications.
  • Subjects previously tested with a positive or indeterminable PPD or QFT result, including subjects that completed standard tuberculosis therapy.
  • Screening 12-lead ECG that demonstrates clinically significant abnormalities requiring treatment, e.g acute myocardial infarction, serious tachy or brady arrhythmias or that are indicative of serious underlying heart disease (e.g. cardiomyopathy, major congenital heart disease, low voltage in all leads, Wolff-Parkinson-White syndrome and other clinically relevant abnormalities which may affect participant safety or interpretation of study results. A history of additional risk factors for Torsades de Pointes (TdP) (e.g. heart failure, hypokalemia, family history of long QT syndrome).
  • If QTcF exceeds 450 mS, or QRS exceeds 120 mS, the ECG should be repeated 2 more times and the average of 3 QTc or QRS values should be used to determine the participants' eligibility. Participants with average screening value QTcF > 450 mS should be excluded. Concomitant use of medications that prolong the QT/QTcF interval is exclusionary.
  • Use of concomitant drugs that prolong the QT interval at the judgment of the PI

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

Other trials in United States

About this study

Official title: A Pilot Study to Assess Safety and Biomarker Responses of the Dual JAK1/TYK2 Inhibitor for Cicatricial Alopecia

Alopecia could be subdivided into two main groups of diseases: non-scarring alopecia, such as male pattern baldness, or alopecia areata (AA), in which hair follicles are preserved, yet quiescent, and scarring alopecia, also known as cicatricial alopecia (CA), in which hair follicles are irreversibly destroyed. CA leads to scarred areas, most commonly on the scalp, that cannot re-grow hair. Despite being a long-term condition, that often has significant impact on patients' well-being, available effective treatments for these diseases are lacking. In addition, the molecular abnormalities causing CA are largely unknown. The study team's research involves administrating patients a new investigational drug (a combined TYK/JAK inhibitor) which has been shown to be safe and well tolerated in clinical studies to date, and is being investigated in other conditions, such as AA. CA patients will be asked to provide small samples of skin and blood throughout the treatment period, to find out how they respond to the drug, and to attempt to better understand these diseases.

Read the detailed description

JAK inhibitors are a group of small molecules, recently emerging as an appealing class of immune modifiers in dermatology. These are antagonists of the various members of the JAK enzymes family, which consists of JAK1, JAK2, JAK3, and tyrosine kinase-2 (TYK2). JAKs enable the binding and activation of the transducer and activator of transcription (STAT), by phosphorylating the cytoplasmic domain of multiple cytokine receptors. This results in translocation of the STAT into the nucleus, which greatly affects transcription. JAK antagonism therefore blocks this signaling through STAT activation, targeting Th1/IFN-γ as well as common γc cytokines (shared between IL-2, IL-4, IL-9, IL-7, IL-15 and IL-21), and TYK2 also adds an IL-23 capability. Therefore PF-06700841, a dual inhibitor of JAK1 and TYK2, currently being investigated for a number of indications including psoriasis, Crohn's disease, ulcerative colitis, psoriatic arthritis, atopic dermatitis, psoriasis, systemic lupus erythematosus and AA, and which has been shown to be safe and well tolerated, with good safety profile, was chosen for this protocol.

The study team will evaluate scalp and blood markers of inflammation, hair keratins and fibrosis, and our ultimate goal would be to elucidate the relations between inflammation and tissue scarring. While the study design is specifically powered to detect mechanistic tissue effects of PF-06700841, drug safety and tolerability in this patient population will also be closely monitored.

The study team's research proposal is novel in that the team proposes to investigate the immune profile of CA patients in skin and blood, at baseline, as well as during treatment with PF-06700841. In addition to the much-needed, prospective investigation of a new treatment modality for these diseases, the study team also aims to better characterize these diseases molecularly, and attempt to determine the effects of the inflammatory process on the resultant fibrosis. These findings will help to identify new treatment targets as well as direct further investigation for the development of new therapies for these disfiguring diseases.

Being tested
PF-06700841, Placebo
Keywords
Alopecia, Scarring Alopecia, Hair Loss
Medical subject headings
Alopecia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PF-06700841Drug

    Also called Brepocitinib

    Active study drug

  • PlaceboDrug

    placebo comparator

Study groups in NCT05076006
Study groupRoleWhat they receive
PlaceboPlacebo comparatorTablets without active ingredients
Receives: Drug: Placebo
PF-06700841Experimentaltablets containing active drug (a combined TYK/JAK inhibitor)
Receives: Drug: PF-06700841

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Treatment-Emergent Adverse Events

    The adverse event will be described and categorized as Treatment-emergent, Serious, abnormal in vital signals, and abnormal in laboratory parameters.

    Incidence and Severity of

    Measured: Week 48

  2. Changes From Baseline in CCL5 Gene Expression Level in Response to PF-06700841

    mRNA Levels of CCL5 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at baseline, week 24 and week 48. Changes are characterized by differences in Ct values from a specific time point (week 24 or week 48) to baseline.

    Measured: Baseline, Week 24 and Week 48

  3. Changes From Baseline in CXCR3(TGFB1) Gene Expression Level in Response to PF-06700841

    mRNA Levels of CXCR3(TGFB1) gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at baseline, week 24 and week 48. Changes are characterized by differences in Ct values from a specific time point (week 24 or week 48) to baseline.

    Measured: Baseline, Week 24 and Week 48

Secondary outcomes (10)

  1. Changes From Baseline in IFN-γ Gene Expression Level in Response to PF-06700841

    mRNA Levels of IFN-γ gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at week 24 and week 48. Changes are characterized by differences in Ct values from a specific time point (week 24 or week 48) to baseline.

    Measured: Baseline, Week 24 and Week 48

  2. Changes From Baseline in CXCL9 Gene Expression Level in Response to PF-06700841

    mRNA Levels of CXCL9 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at week 24 and week 48. Changes are characterized by differences in Ct values from a specific time point (week 24 or week 48) to baseline.

    Measured: Baseline, Week 24 and Week 48

  3. Changes From Baseline in CXCL10 Gene Expression Level in Response to PF-06700841

    mRNA Levels of CXCL10 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at week 24 and week 48. Changes are characterized by differences in Ct values from a specific time point (week 24 or week 48) to baseline.

    Measured: Baseline, Week 24 and Week 48

  4. Changes From Baseline in IL-12RB1 Gene Expression Level in Response to PF-06700841

    mRNA Levels of IL-12RB1 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at week 24 and week 48. Changes are characterized by differences in Ct values from a specific time point (week 24 or week 48) to baseline.

    Measured: Baseline, Week 24 and Week 48

  5. Changes From Baseline in STAT1 Gene Expression Level in Response to PF-06700841

    mRNA Levels of STAT1 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at week 24 and week 48. Changes are characterized by differences in Ct values from a specific time point (week 24 or week 48) to baseline.

    Measured: Baseline, Week 24 and Week 48

  6. Mean Change in The Frontal Fibrosis Alopecia Severity Index (FFASI) as Compared to Baseline

    Mean Change in The Frontal Fibrosis Alopecia Severity Index (FFASI) at Week 12, Week 24 and Week 48 as compared to baseline

    The Frontal Fibrosis Alopecia Severity Index utilizes clinical images of the entire hairline divided into 4 sections. The scalp/head section is scored 0-84 with the second section (all other areas) scored 0-16. The sum of the scalp and other areas results in a total score 0-100, with higher score indicating more severity.

    Measured: Baseline, Week 12, Week 24, Week 48

  7. Mean Percent Change in The Lichen Planopilaris Activity Index (LPPAI) as Compared to Baseline

    Mean Percent Change in The Lichen Planopilaris Activity Index (LPPAI) at Week 12, Week 24 and Week 48 as compared to baseline

    The Lichen Planopilaris Activity Index is a numeric composite index that aggregates symptoms and signs of pruritus, pain, burning, scalp erythema, perifollicular erythema, perifollicular scale, pull test and spreading. Symptoms and signs are measured in a 4-point scale (0-absent, 1-mild, 2-moderate and 3-severe). Full range from 0-10, higher score indicates more severity.

    Measured: Baseline, Week 12, Week 24, Week 48

  8. Change in The Central Hair Loss Grade (CHLG)

    The Central Hair Loss Grade is a clinical measure of severity that uses a 6 points scale (0 no central scalp hair loss , 1 - minimal central scalp hair loss , 2 - clinically evident central scalp hair loss, 3-5 advanced central scalp hair loss). Change in CHLG for participants with CCCA at Week 48 as compared to baseline

    Measured: Baseline and Week 48

  9. Change in Physician Global Assessment of Improvement (PGA-I)

    The PGA-I ranges from -4(significant worsening) to 4(significant improvement).

    Measured: Week 24 and Week 28

  10. Absolute Change in the Dermatology Quality of Life Index (DLQI)

    DLQI is a questionnaire with quality of life indicators related to the health of the skin. This questionnaire has 10 items related to skin problems, each one with 4 possible answers: Very Much, A Lot, A Little, and Not at All. The sum of items scores will generate a score of how much the skin problem affects the personal life. Full score range from 0 to 30, with higher score indicating poorer health outcomes. Change at Week 48 as compared to baseline

    Measured: Baseline and Week 48

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Oct 2021
  2. Study start19 May 2021
  3. Primary completion30 Sep 2023
  4. Study completion30 Sep 2023

Resources

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
Randomised
Intervention model
Crossover
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
51 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05076006
Sponsor's protocol ID
GCO 21-0434
Status last verified
May 2026
First submitted
9 Aug 2021
Last updated
19 May 2026
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • David E, Shokrian N, Del Duca E, Meariman M, Dubin C, Hawkins K, Andrews E, Sikand S, Singer G, Oemar B, Estrada Y, Bose S, Pulsinelli J, Mahling P, Da Rosa JC, Ungar B, Peeva E, Guttman-Yassky E. A phase 2a trial of brepocitinib for cicatricial alopecia. J Am Acad Dermatol. 2025 Mar;92(3):427-434. doi: 10.1016/j.jaad.2024.09.073. Epub 2024 Oct 24.

    PubMed 39461505

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PF-06700841 in people with Cicatricial Alopecia, run by Emma Guttman 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 5 inclusion and 31 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 19 May 2026. 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. Some participants in this study may receive a placebo. 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 19 May 2026). 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?

Number of Participants With Treatment-Emergent Adverse Events (time frame: Week 48). The study lists 3 primary outcomes in all. 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 NCT05076006, 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 19 May 2026.

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