Clinical trial NCT05650333: A Study to Learn About the Study Medicine (Called Ritlecitinib) For the Potential Treatment of Severe Alopecia Areata (AA) In Children 6 To Less Than 12 Years of Age
Official title: AN INTERVENTIONAL PK, PD, PHASE 1, OPEN-LABEL STUDY TO INVESTIGATE PK AND PD OF MULTIPLE-DOSE RITLECITINIB IN CHILDREN 6 TO LESS THAN 12 YEARS OF AGE WITH SEVERE ALOPECIA AREATA
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Alopecia Areata
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-12-14
Enrollment: 15
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: The purpose of the study is to evaluate the pharmacokinetics (how the medicine is changed and eliminated from your body after you take it) and pharmacodynamics (effects of the medicine in the body) of the study medicine (called Ritlecitinib) in children of 6 to \<12 years of age with Alopecia Areata, a condition of scalp hair loss. 12 children with alopecia areata will be participating in this study. All participants will receive study medicine with a dose of 20 milligram (mg) orally once daily for 7 days. 5 blood samples will be collected on day 7 for pharmacokinetic evaluation and 2 blood samples each at screening and on Day 7 will be collected for pharmacodynamic evaluation. Participants will take part in the study for about 10 weeks.

Detailed description: This is an interventional, Pharmacokinetic (PK), Pharmacodynamic (PD), phase 1, open label study in children 6 to less than 12 years of age with ≥50% scalp hair loss due to severe alopecia areata. The purpose of the study is to collect data to support dose selection for subsequent studies in the same population.

Participants will be screened and, if all eligibility criteria are met, will receive the first dose of Investigational product within 28 days after the screening visit.

Participants will receive 20 mg ritlecitinib in one dose, daily, for 7 consecutive days. Blood samples for pharmacodynamic evaluation will be collected on screening and Day 7. Blood samples for pharmacokinetic evaluation will be collected on Day 7 at: 0 hr (pre-dose), 0.5 hr, 1 hr, 3 hrs, and 8 hrs after dosing.

At least 12 evaluable participants with respect to the primary endpoint will be enrolled in the study.

Participants and their parents/legal guardians will be required to visit the study site 3 times during the study (Screening, Day 1 and Day 7) A safety follow-up visit will be conducted by phone, 28 to 35 days after the last dose of ritlecitinib.

Eligibility criteria: Key Inclusion criteria:

1. Participants who are 6 to less than12 years old at the baseline visit.
2. A diagnosis of severe AA, including AT and AU, with ≥50% scalp hair loss due to AA (ie, a SALT score of ≥50) at both the Screening and Baseline visits, without evidence of terminal hair regrowth within the previous 12 months.

Key Exclusion Criteria:

1. A known congenital cause of AA, other systemic diseases that may cause hair loss (eg, lupus erythematosus, thyroiditis, systemic sclerosis, lichen planus, etc) or other etiology of hair loss (eg, telogen effluvium, androgenetic alopecia, etc).
2. Any present malignancies or history of malignancies, history of any lymphoproliferative disorder
3. History (one or more episodes) of CMV, varicella, herpes zoster (shingles) or disseminated herpes simplex.
4. Other medical or psychiatric condition (including recent \[within the past year\] or active suicidal ideation/behavior) that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
5. Not up to date with all age appropriate vaccines (including 2-dose vaccination for varicella) or vaccination with attenuated live vaccine within 6 weeks of first dose of study medicine.

Primary outcomes:
measure: Area Under the Plasma Concentration-Time Profile Over the Dosing Interval of 24 Hours, at Steady State (AUC24ss/AUCtau) of Ritlecitinib on Day 7
timeFrame: Day 7: 0 (pre-dose), 0.5, 1, 3, 8 and 24 hours [pre-dose concentration was used as an estimate for the concentration of 24 hours post dose]
Secondary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax) of Ritlecitinib
timeFrame: 0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
measure: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Ritlecitinib
timeFrame: 0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
measure: Apparent Oral Clearance (CL/F) of Ritlecitinib
timeFrame: 0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
measure: Apparent Volume of Distribution (Vz/F) of Ritlecitinib
timeFrame: 0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
measure: Elimination Half-Life (t1/2) of Ritlecitinib
timeFrame: 0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
measure: Change From Baseline in Interferon Gamma Induced Protein 10 (IP-10) on Day 7
timeFrame: Baseline and Day 7
measure: Change From Baseline in T Lymphocytes on Day 7
timeFrame: Baseline and Day 7
measure: Change From Baseline in B Lymphocytes on Day 7
timeFrame: Baseline and Day 7
measure: Change From Baseline in Natural Killer (NK) Cells on Day 7
timeFrame: Baseline and Day 7
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42)
measure: Number of Participants With Treatment Related AEs
timeFrame: Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42)
measure: Number of Participants With Serious AEs (SAEs)
timeFrame: Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42)
measure: Number of Participants With AEs Leading to Treatment Discontinuation
timeFrame: Day 1 up to Day 7
measure: Number of Participants With Clinically Significant Abnormalities in Vital Signs
timeFrame: Day 1 up to Day 7
measure: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Values
timeFrame: Day 1 up to Day 7
measure: Number of Participants as Per Score for Pediatric Taste Assessment Questionnaire
timeFrame: Day 1 and 7
Other outcomes:
Locations:
city: Encinitas
country: United States
facility: California Dermatology & Clinical Research Institute
state: California
city: Coral Gables
country: United States
facility: Pediatric Skin Research,LLC
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Indianapolis
country: United States
facility: Dawes Fretzin Clinical Research Group, LLC
state: Indiana
city: Albuquerque
country: United States
facility: University of New Mexico Health Sciences Center
state: New Mexico
city: Albuquerque
country: United States
facility: UNMH
state: New Mexico
city: Tulsa
country: United States
facility: Vital Prospects Clinical Research Institute, PC
state: Oklahoma
city: Portland
country: United States
facility: Northwest Dermatology Institute
state: Oregon
city: San Antonio
country: United States
facility: Texas Dermatology and Laser Specialists
state: Texas
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: