Dual JAK1/TYK2 Inhibitor for Cicatricial Alopecia

NCT05076006 | Completed Phase 2 FDA Drug

• 1 other identifier: GCO 21-0434
• 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 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.

Conditions

Cicatricial Alopecia

Keywords

Alopecia; Scarring Alopecia; Hair Loss

Outcome Measures

Primary Outcomes (3)

• Incidence and Severity of 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.

  Week 48

• 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 12 and week 24. Changes are characterized by differences in Ct values from a specific time point (week 12 or week 24) to baseline.

  Week 12 and Week 24

• Changes from baseline in CXCR3 gene expression level in response to PF-06700841

  mRNA Levels of CXCR3 gene expression in skin biopsies quantified by normalized Ct values obtained by quantitative real-time PCR assay, measured at baseline, week 12 and week 24. Changes are characterized by differences in Ct values from a specific time point (week 12 or week 24) to baseline.

  Week 12 and Week 24

Secondary Outcomes (23)

• Changes from baseline in IFN-γ gene expression level in response to PF-06700841

  Week 12, Week 24 and Week 48

• Changes from baseline in CXCL9 gene expression level in response to PF-06700841

  Week 12, Week 24 and Week 48

• Changes from baseline in CXCL10 gene expression level in response to PF-06700841

  Week 12, Week 24 and Week 48

• Changes from baseline in IL-12RB1 gene expression level in response to PF-06700841

  Week 12, Week 24 and Week 48

• Changes from baseline in STAT1 gene expression level in response to PF-06700841

  Week 12, Week 24 and Week 48

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Tablets without active ingredients

Drug: Placebo

PF-06700841

EXPERIMENTAL

tablets containing active drug (a combined TYK/JAK inhibitor)

Drug: PF-06700841

Interventions

PF-06700841 DRUG

Active study drug

Also known as: Brepocitinib

PF-06700841

Placebo DRUG

placebo comparator

Placebo

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 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.

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.
• 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.
• 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.

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

Interventions

PF-06700841

Condition Hierarchy (Ancestors)

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

Study Officials

• Emma Guttman, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Only designated pharmacist will have knowledge of treatment assignment
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 9, 2021
• First Posted: October 13, 2021
• Study Start: May 19, 2021
• Primary Completion: September 30, 2023
• Study Completion: September 30, 2023
• Last Updated: July 15, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)