Clinical trial NCT06531109: Characterization And Clinical Outcomes of AA Patients Treated With Ritlecitinib
Official title: PRESTO - Characterization and Clinical Outcomes of Alopecia Aerata (AA) Patients Treated With Ritlecitinib in a Real-world (RW) Cohort: A Multinational, Prospective Observational Study.
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Alopecia Areata
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2024-07-31
Enrollment: 850
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population eligible for enrollment includes any adult and adolescent patients aged ≥12 years diagnosed with AA who receive at least one dose of ritlecitinib and satisfy the inclusion and exclusion criteria. The study will be open for enrollment for approximately 12 months after the first patient has been enrolled. Depending on the observed enrollment rate, the enrollment period and number of sites may be reassessed and revised during the study. It will be allowed to enroll patients with previous use of topical JAK inhibitors only if there was at least a 4-week wash-out period prior to Baseline Visit

Brief summary: Alopecia areata (AA) is a chronic relapsing autoimmune disease characterized by nonscarring hair loss affecting children, adolescents, and adults across all ages, races, and genders. AA primarily affects the scalp; however, it also can affect nails, eyelashes, eyebrows, and other hair follicles on the patient's body. The 3 main types of AA are:

* Patchy alopecia (PA), as seen in 90% of clinical diagnoses
* Alopecia totalis (AT), that affects all scalp hair
* Alopecia universalis (AU), involving all scalp, face, and body hair Dermatologist preferences for utility and order of skin-directed therapies to treat AA vary widely, with treatment choices based on various factors such as patients' age, disease duration and severity (Meah et al., 2020).

Ritlecitinib is a bioavailable small molecule that irreversibly binds to Janus kinase-3 (JAK3) and Tyrosine kinase Expressed in the hepatocellular Carcinoma kinase family (TEC). Ritlecitinib 50 mg once daily was approved by the FDA 23 June 2023 and EMA 20 July 2023 for the treatment of severe alopecia areata in adults and adolescents 12 years of age and older. In Japan, ritlecitinib was approved on 26 June 2023 for the treatment of alopecia areata (limited to intractable cases involving widespread hair loss). Additional countries have since approved ritlecitinib. Those approvals are based on the results of the ritlecitinib pivotal phase 2b/3 study (ALLEGRO 2b/3) which examined efficacy and safety of ritlecitinib in AA patients globally.

Despite positive results from the ALLEGRO program, there is still lack of evidence on ritlecitinib patients' characteristics and clinical outcomes in routine clinical practice. The investigators will evaluate patient and disease characteristics, treatment patterns, and clinical and patient-reported outcomes among patients with AA who are receiving ritlecitinib.

The aim of this study is to measure effectiveness of ritlecitinib in a real-world setting. Ritlecitinib will be prescribed to patients according to the approved product label. Treatment will be guided by clinical judgement of the treating physician ie, study investigators, according to standard of care, independently of this study.


Eligibility criteria: Inclusion Criteria:

1. Male and female patients aged \>12 years at baseline.
2. Patients with diagnosis of alopecia areata confirmed by a certified dermatologist, who are prescribed ritlecitinib as per the product label independently of the decision to enroll a patient in this study.
3. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study. Following receipt of oral and written information about the trial, the adolescent (depending on local institutional review board/independent ethics committee requirements) must provide assent, and one or both (according to local regulations) parents or guardians of the child must provide signed informed consent before any study-related activity is carried out.
4. Patients, who in the opinion of the investigator, are willing and able to comply with regular clinic visits as per standard practice at the site and agree to complete PRO questionnaires and other patient completed questions.

Exclusion Criteria:

1. Diagnosed with other types of alopecia or other diseases that can cause hair loss (including, but not limited to known androgenetic alopecia, traction and scarring alopecia, telogen effluvium).
2. Diagnosed with other scalp diseases that may impact AA assessment (e.g., scalp psoriasis, dermatitis, etc.) or other active systemic diseases that may cause hair loss (e.g., lupus erythematosus, thyroiditis, systemic sclerosis, lichen planus, etc.). that could interfere with assessment of hair loss/regrowth.
3. Patients previously treated with ritlecitinib or other JAK inhibitors.
4. Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family.

Primary outcomes:
measure: Percentage change from baseline in Severity of Alopecia Tool (SALT) score at week 24
timeFrame: 24 weeks from initiation of the treatment
Secondary outcomes:
measure: Demographic characteristic of participants: Age, sex, race, ethnicity, socio-economic status, area of residence, smoking status
timeFrame: Baseline
measure: Demographic Characteristic of Participants: Date of Initial AA diagnosis
timeFrame: Baseline
measure: Demographic Characteristics of participants: Duration of AA since diagnosis
timeFrame: Baseline
measure: Demographic characteristics of participants: Duration of current AA Episode
timeFrame: Baseline
measure: Demographic Characteristic of Participants: Predominant AA phenotype
timeFrame: Baseline
measure: Clinical Characteristics of participants: Severity of Alopecia Tool (SALT) Score
timeFrame: Baseline
measure: Clinical characteristics of participants: Eyelash Assessment (ELA) score
timeFrame: Baseline
measure: Clinical Characteristics of participants: Eyebrow Assessment (EBA) Score
timeFrame: Baseline
measure: Clinical Characteristics of participants: number of affected nails
timeFrame: Baseline
measure: Clinical Characteristics of participants: Presence of Beard involvement
timeFrame: Baseline
measure: AA treatment history of participants: number of patients with prior AA treatments
timeFrame: Baseline
measure: AA treatment history of participants: prior AA treatment class and route
timeFrame: Baseline
measure: AA treatment history of participants: duration of prior therapy / frequency
timeFrame: baseline
measure: AA treatment history of the participants: total number of prior AA treatments
timeFrame: Baseline
measure: RW effectiveness of ritlecitinib: proportion of patients with <20 in SALT score
timeFrame: week 24, 48, 72 and 96
measure: RW effectiveness of ritlecitinib: Proportion of patients with <10
timeFrame: week 24, 48, 72 and 96
measure: RW effectiveness of ritlecitinib: Change from baseline in SALT score
timeFrame: week 24, 48, 72 and 96
measure: RW effectiveness of ritlecitinib: proportion of patient with improvement from abnormal baseline in Eyebrow Assessment score (EBA)
timeFrame: Week 24, 48, 72 and 96
measure: RW effectiveness of ritlecitinib: proportion of patient with improvement from abnormal baseline in Eyelashes Assessment Score (ELA)
timeFrame: Week 24, 48, 72 and 96
measure: RW effectiveness of ritlecitinib: proportion of patients with improvement from abnormal baseline in beard assessment and nail involvement
timeFrame: week 24, 48, 72 and 96
measure: Patient reported outcome: Evaluate ritlecitinib treatment satisfaction
timeFrame: week 24, 48, 72 and 96
measure: Patient reported outcome: Evaluate impression of hair growth
timeFrame: week 24, 48, 72 and 96
measure: Evaluate impact of ritlecitinib: Patient reported quality of life
timeFrame: week 24, 48, 72 and 96
measure: Evaluate impact of ritlecitinib: Patient reported quality of life
timeFrame: week 24, 48, 72 and 96
Other outcomes:
Locations:
city: Montgomery
country: United States
facility: C2 Research Center
state: Alabama
city: Fremont
country: United States
facility: CENTER FOR DERMATOLOGY CLINICAL RESEARCH, Inc
state: California
city: Fresno
country: United States
facility: Rao Dermatology
state: California
city: Oxnard
country: United States
facility: Cura Clinical Research
state: California
city: Santa Ana
country: United States
facility: Southern California Clinical Research
state: California
city: Boca Raton
country: United States
facility: Dermatology of Boca
state: Florida
city: Coral Gables
country: United States
facility: Pediatric Skin Research,LLC
state: Florida
city: Jacksonville
country: United States
facility: Suncoast Skin Solutions
state: Florida
city: Miami
country: United States
facility: Skin Research of South Florida LLC
state: Florida
city: Marriottsville
country: United States
facility: Kindred Hair and Skin Center
state: Maryland
city: Auburn Hills
country: United States
facility: Oakland Hills Dermatology
state: Michigan
city: Waterford
country: United States
facility: Michigan Dermatology Institute
state: Michigan
city: Minneapolis
country: United States
facility: Twin Cities Dermatology Center
state: Minnesota
city: Portland
country: United States
facility: NW Dermatology Institute
state: Oregon
city: Southlake
country: United States
facility: Epiphany Dermatology
state: Texas
city: Mill Creek
country: United States
facility: Frontier Dermatology
state: Washington
city: Hamamatsu
country: Japan
facility: Hamamatsu University Hospital
state: Shizuoka
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: