Ritlecitinib for Cicatricial Alopecia

NCT05549934 | Completed Phase 2 FDA Drug

• 1 other identifier: GCO 22-1234
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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. 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 research team will be administering a new investigational drug (a JAK3/TEC inhibitor), ritlecitinib, which has shown statistically significant improvement in scalp hair loss for AA patients in a proof of concept and phase 2b/3 studies (B7981015 AA study). This is an open-label clinical trial. 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.

Conditions

Cicatricial Alopecia

Keywords

frontal fibrosing alopecia; FFA; lichen planopilaris; LPP; central centrifugal; scarring alopecia; CCCA

Outcome Measures

Primary Outcomes (4)

• Number of Treatment-Emergent Adverse Events

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

  Week 48

• Severity of Treatment-Emergent Adverse Events

  The adverse event will be categorized according to CTCAE guidelines when applicable

  Week 48

• Change in Ct values of mRNA levels of CCL5 gene

  Changes in mRNA Levels of CCL5 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at baseline and week 24. The unit of the outcome is called Ct value and it represents the number of amplification cycles to reach a level of fluorescence in the experiment. The Ct value is directly associated to the level of expression of a gene

  Baseline to Week 24

• Change in Ct values of mRNA levels of CCL5 gene

  Changes in mRNA Levels of CCL5 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at baseline and week 48. The unit of the outcome is called Ct value and it represents the number of amplification cycles to reach a level of fluorescence in the experiment. The Ct value is directly associated to the level of expression of a gene.

  Baseline to Week 48

Secondary Outcomes (7)

• Change in Ct values of mRNA levels of CXCR3

  Baseline to Week 24

• Ct value of mRNA levels of CXCR3

  Baseline to Week 48

• The Frontal Fibrosis Alopecia Severity Index (FFASI)

  Baseline

• The Frontal Fibrosis Alopecia Severity Index (FFASI)

  Week 24

• The Frontal Fibrosis Alopecia Severity Index (FFASI)

  Week 48

Study Arms (1)

PF-06651600 (Ritlecitinib)

EXPERIMENTAL

200 mg once-daily for 8 weeks and then 100 mg once daily for the remaining 40 weeks

Drug: PF-06651600

Interventions

PF-06651600 DRUG

tablets containing active drug (a combined JAK3/TEC inhibitor)

Also known as: Ritlecitinib

PF-06651600 (Ritlecitinib)

Eligibility Criteria

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

You may qualify if:

• 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 a history of at least 6 months of CA (LPP/FFA or CCCA). Diagnosis will be made clinically (according to the LPPAI37, FFASI36 and/or CHLG38).
• Subject has a negative Tuberculin purified protein derivative (PPD) or QuantiFERON TB-Gold test (QFT) at screening or within the last 12 months.
• A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
• Is not a woman of childbearing potential (WOCBP) OR;
• Is a WOCBP (all female participants, regardless of whether or not they have experienced/reported menarche, are considered WOCBP unless they are permanently sterile or confirmed infertile). A WOCBP who is sexually active must use a contraceptive method that is highly effective, with a failure rate of \<1%, during the intervention period and for at least 28 days after the last dose of study intervention.
• And if a WOCBP, must have a negative highly sensitive serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline performed before the first dose of study intervention.
• Subject is judged to be in otherwise good overall health following a detailed medical and medication history, physical examination, and laboratory testing.

You may not qualify if:

• 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 at screening (de novo patients only).
• 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 (e.g., scalp psoriasis, dermatitis, etc.).
• Subject is pregnant or breastfeeding.
• Participation in other studies involving investigational drug(s) within 4 weeks or within 5 half-lives (if known), whichever is longer, prior to study entry and/or during study participation (de novo patients only).
• Active systemic diseases that may cause hair loss (e.g., systemic lupus erythematosus, thyroiditis, systemic sclerosis, etc.).
• Any Psychiatric condition in the opinion of the investigator precludes participation in the study.
• Subjects with a Columbia Suicide Severity Rating Scale (C-SSRS) score of "yes" on questions 4 and/or 5 at Visit 2 (Baseline).
• 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.
• History of thromboembolic events including DVT and PE or history of inherited coagulopathies.
• 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.

Sponsors & Collaborators

• Emma Guttman: lead
• Pfizer: collaborator

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

MeSH Terms

Conditions

Alopecia; Lichen Planus

Interventions

PF-06651600

Condition Hierarchy (Ancestors)

Hypotrichosis; Hair Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Lichenoid Eruptions; Skin Diseases, Papulosquamous

Study Officials

• Emma Guttman-Yassky, MD, PhD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: The Waldman Professor and System Chair

Study Record Dates

• First Submitted: September 19, 2022
• First Posted: September 22, 2022
• Study Start: December 15, 2022
• Primary Completion: March 20, 2025
• Study Completion: March 20, 2025
• Last Updated: April 6, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)