Phase 3 Randomized Study of Ritlecitinib Tosilate Efficacy and Safety in Alopecia Areata Patients Aged 12 and Older with Dose Adjustment for Non-Responders
- Trial ID
- 2024-519370-40-00
- Protocol
- B7981094
- Sponsor
- Pfizer Inc.
Trial statistics
Diseases & Conditions
Objectives
The primary objective of this study is to evaluate the **efficacy** of ritlecitinib 100 mg once daily (QD) compared to placebo in promoting the regrowth of scalp hair in individuals with **alopecia areata**. This objective is clinically relevant as it aims to address the significant hair loss associated with alopecia areata, which can have profound psychological and social impacts on affected individuals.
Secondary objectives include:
- Comparing the efficacy of ritlecitinib 50 mg QD versus placebo on the regrowth of scalp hair.
- Assessing the effect of ritlecitinib 100 mg QD versus placebo and ritlecitinib 50 mg QD versus placebo on patient-centered outcomes.
- Comparing the efficacy of ritlecitinib 100 mg QD versus ritlecitinib 50 mg QD on the regrowth of scalp hair.
Participants
The clinical trial involves a total of **476 participants** diagnosed with **alopecia areata**, a condition characterized by hair loss. The study population includes both male and female subjects, with an age range starting from 12 years, provided local regulations allow, up to adults. Participants are required to have at least 50% hair loss on the scalp, as measured by the SALT score, with no evidence of terminal hair regrowth in the previous six months. The trial includes a vulnerable population, indicating that special considerations are in place for the protection of these participants. The selection process ensures that only individuals with a clinical diagnosis of alopecia areata, without other causes of hair loss, are included. Lifestyle factors such as diet and physical activity are not specified as part of the selection criteria. The trial aims to assess the efficacy of ritlecitinib 100 mg QD compared to a placebo in promoting scalp hair regrowth.
Plans and Procedures
The clinical trial is designed as a **randomized**, double-blind, placebo-controlled study to evaluate the safety and efficacy of **Ritlecitinib Tosilate** in participants aged 12 years and older with **alopecia areata**. The trial will involve two dosage groups, 50 mg and 100 mg, administered orally once daily, with a placebo group serving as a control. The study is structured to include an initial screening visit, followed by a series of study visits over a 48-week period, culminating in an end-of-study visit. The primary endpoint is the proportion of participants achieving a SALT ≤20 response at Week 24, with secondary endpoints including the PGI-C response and change from baseline in SALT score at Week 24.
Participants will be screened to ensure they meet the inclusion criteria, which require a clinical diagnosis of **alopecia areata** with at least 50% scalp hair loss, as measured by the SALT score, and no evidence of terminal hair regrowth within the previous six months. Adolescents aged 12 to 17 may participate if permitted by local regulations. The trial will exclude individuals who do not meet these criteria or have other etiologies of hair loss. The expected duration of participant involvement is approximately 48 weeks, with the possibility of early termination if significant adverse events occur or if the participant withdraws consent.
Study visits will be scheduled at regular intervals to monitor safety, efficacy, and compliance with the treatment regimen. The initial screening visit will confirm eligibility, followed by baseline assessments. Subsequent visits will include evaluations of hair regrowth, safety assessments, and collection of data for primary and secondary endpoints. The end-of-study visit will provide a comprehensive assessment of the participant's response to treatment and any adverse events experienced during the trial. The trial is anticipated to conclude by April 2027, with recruitment starting in August 2025.
Treatment
The clinical trial involves the administration of **Ritlecitinib Tosilate**, a chemical compound developed by Pfizer Inc. This experimental medication is provided in two pharmaceutical forms: a **capsule** and a **hard capsule**. The dosage for the capsule form is 100 mg, with a maximum daily dose of 100 mg and a total maximum dose of 33,600 mg over a treatment period of 48 weeks. The hard capsule form is administered at a dosage of 50 mg, with a maximum daily dose of 50 mg and a total maximum dose of 16,800 mg over the same treatment period. Both forms are administered orally once daily. Participant compliance with the dosing schedule is monitored throughout the study to ensure adherence to the treatment regimen.
The study also includes the use of a **placebo** as a comparator treatment. The placebo is designed to match the experimental medication in appearance and is available in two forms: a capsule corresponding to the 100 mg dose and another corresponding to the 50 mg dose. The placebo is administered orally once daily, following the same schedule as the active treatment. The use of a placebo allows for the assessment of the efficacy of Ritlecitinib Tosilate by providing a control group for comparison. The trial is designed to evaluate the safety and efficacy of Ritlecitinib Tosilate in adult and adolescent participants aged 12 years and older with **alopecia areata**.
Efficacy
The efficacy of the investigational product, **Ritlecitinib Tosilate**, will be assessed in a Phase 3 clinical trial involving adult and adolescent participants aged 12 years and older with **Alopecia Areata**. The primary endpoint for evaluating efficacy is the SALT (Severity of Alopecia Tool) ≤20 response at Week 24. Secondary endpoints include the SALT ≤20 response at Week 24, PGI-C (Patient Global Impression of Change) response defined as a score of "moderately improved" or "greatly improved" at Week 24, and the change from baseline (CFB) in SALT score at Week 24.
Measurements will be conducted at specified timepoints, with the primary and secondary endpoints being assessed at Week 24. The SALT score will be used to quantify the extent of scalp hair loss, and photographs taken at screening will be submitted for verification of the SALT score and hair loss due to Alopecia Areata. The PGI-C will be utilized to capture patient-reported outcomes regarding perceived improvement. These assessments will provide a comprehensive evaluation of the efficacy of Ritlecitinib Tosilate in promoting hair regrowth in the target population.
Inclusion and Exclusion Criteria
Inclusion Criteria
- 18 years or older (or the minimum age of consent in accordance with local regulations) at screening. Adolescents (12 to <18 years of age at screening) are also eligible for this study, but only if permitted by the local IRB/EC and local regulatory health authority (if applicable). Where these approvals have not been granted, only participants 18 years of age and older at screening will be enrolled.
- Must meet the following AA criteria at both Screening and Baseline: a. Have a clinical diagnosis of AA with no other etiology of hair loss. b. ≥50% hair loss of the scalp, as measured by SALT, without evidence of terminal hair regrowth within the previous 6 months. •Photographs taken at Screening must be submitted to the Sponsor’s designee for verification of SALT score ≥50 and hair loss due to AA. Participants must not be randomized until verification has been confirmed. c. Current episode of hair loss ≤10 years.
Exclusion Criteria
- Diseases or conditions affecting hair loss
- History of severe allergic or anaphylactoid reaction to any kinase inhibitor or a known allergy/hypersensitivity to any component (including excipients) of the study intervention.
- Any psychiatric condition including recent or active suicidal ideation or behavior that meets any of the following criteria: •Suicidal ideation associated with actual intent and a method or plan in the past year: “Yes” answers on items 4 or 5 of the C-SSRS administered at the Screening visit (see Section 8.3.10). •Previous history of suicidal behaviors in the past 5 years: “Yes” answer (for events that occurred in the past 5 years) to any of the suicidal behavior items of the C-SSRS. •For adults, any lifetime history of serious suicidal behavior or recurrent suicidal behavior. For adolescents, any previous lifetime history of suicidal behavior.
- General Infection History: •Having a history of systemic infection requiring hospitalization or parenteral therapy (antimicrobial, antiviral, antiparasitic, antiprotozoal, or antifungal), or as otherwise judged clinically significant by the investigator, within 3 months prior to Day 1.•Have active acute or chronic infection requiring treatment with oral antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 4 weeks prior to Day 1. •Evidence or history of untreated, currently treated or inadequately treated active or latent infection with Mycobacterium TB.
- Specific Viral Infection History: • History (single episode) of disseminated herpes zoster or disseminated herpes simplex, or a recurrent (more than one episode of) localized, dermatomal herpes zoster. • Infected with hepatitis B or hepatitis C viruses: all participants will undergo screening for hepatitis B and C for eligibility. • Have a known immunodeficiency disorder (including positive serology for HIV at Screening) or a first-degree relative with a hereditary immunodeficiency (unless known negative carrier status).
- Other Medical Conditions: •Have hearing loss with progression over the previous 5 years, sudden hearing loss, or middle or inner ear disease such as otitis media, cholesteatoma, Meniere’s disease, labyrinthitis, or other auditory condition that is considered acute, fluctuating or progressive. •Abnormal findings on the screening chest imaging (eg, chest x-ray) including, but not limited to, presence of active TB or other infections, cardiomyopathy, or malignancy. Chest imaging may be performed up to 12 weeks prior to Screening. Documentation of the official reading must be located and available in the source documentation. •Have any malignancies or have a history of malignancies with the exception of adequately treated or excised nonmetastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ. •Have a history of any lymphoproliferative disorder such as EBV-related lymphoproliferative disorder, history of lymphoma, history of leukemia, or signs and symptoms suggestive of current lymphatic or lymphoid disease. •Significant trauma or major surgery within 1 month of the first dose of study drug or considered in imminent need for surgery. Participants with elective surgery scheduled to occur during the study can only be enrolled with approval of the sponsor.
- Adolescent participants 12 to <18 years of age without one of the following: •Documented evidence from a health professional of having received varicella vaccination (2 doses); or •Evidence of prior exposure to varicella zoster virus (VZV) based on serological testing (ie, a positive VZV IgG Ab result) at Screening. Note: Serological testing must be performed for VZV immunoglobulin G (IgG) antibody (Ab) only in the absence of documented evidence from a health professional of having received varicella vaccination (2 doses). If serological testing is performed in the presence of documented evidence of having received varicella vaccination (2 doses), participants are eligible to enter the study regardless of the result of serological testing.
- Any medical or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study.
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 Czechia | Not Recruiting | 04 Aug 2025 | 17 |
 Poland | Not Recruiting | 04 Aug 2025 | 55 |
 Spain | Not Recruiting | 04 Aug 2025 | 2 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
PLACEBO FOR PF-06651600-15 CAPSULE, 100 MG | Placebo | N/A | — | — | — | N/A |
Ritlecitinib Tosilate | Test | CAPSULE | ORAL | 100 | 48 | PRD10739137 |
PLACEBO FOR PF-06651600-15 CAPSULE, 50 MG | Placebo | N/A | — | — | — | N/A |
Ritlecitinib tosilate | Test | CAPSULE, HARD | ORAL | 50 | 48 | PRD9906097 |