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

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

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.

Study Overview

• Status: Completed
• Conditions: Alopecia Areata
• Intervention / Treatment: Drug: Ritlecitinib 20 mg

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.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Encinitas, California, United States, 92024
      • California Dermatology & Clinical Research Institute
  • Florida
    • Coral Gables, Florida, United States, 33146
      • Pediatric Skin Research,LLC
    • Miami, Florida, United States, 33155
      • Nicklaus Children's Hospital
  • Indiana
    • Indianapolis, Indiana, United States, 46250
      • Dawes Fretzin Clinical Research Group, LLC
  • New Mexico
    • Albuquerque, New Mexico, United States, 87102
      • University of New Mexico Health Sciences Center
    • Albuquerque, New Mexico, United States, 87106
      • UNMH
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
      • Vital Prospects Clinical Research Institute, PC
  • Oregon
    • Portland, Oregon, United States, 97210
      • Northwest Dermatology Institute
  • Texas
    • San Antonio, Texas, United States, 78218
      • Texas Dermatology and Laser Specialists

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 11 years (Child)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ritlecitinib 20 mg; Participants will receive Ritlecitinib 20 mg by mouth once daily (QD).	Drug: Ritlecitinib 20 mg; orally administered, Ritlecitinib 20 mg once daily (QD)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Area Under the Plasma Concentration-Time Profile Over the Dosing Interval of 24 Hours, at Steady State (AUC24ss/AUCtau) of Ritlecitinib on Day 7	Linear-log trapezoidal method was used for evaluation. For the calculation of AUCtau, pre-dose concentration of Day 7 was used as an estimate for the concentration of 24 hours post-dose on Day 7.	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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Observed Plasma Concentration (Cmax) of Ritlecitinib		0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Ritlecitinib		0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
Apparent Oral Clearance (CL/F) of Ritlecitinib	Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological process. Clearance obtained after oral dose (apparent oral clearance) is influenced by the fraction of the dose absorbed.	0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
Apparent Volume of Distribution (Vz/F) of Ritlecitinib	Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vz/F is influenced by the fraction absorbed.	0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
Elimination Half-Life (t1/2) of Ritlecitinib	Elimination half-life (t1/2) is the time measured for the plasma concentration to decrease by one half at the elimination phase.	0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7
Change From Baseline in Interferon Gamma Induced Protein 10 (IP-10) on Day 7		Baseline and Day 7
Change From Baseline in T Lymphocytes on Day 7	T lymphocytes included CD3 cells, CD4 T helper lymphocytes and CD8 T cytotoxic lymphocytes.	Baseline and Day 7
Change From Baseline in B Lymphocytes on Day 7	B lymphocytes included CD19 cells.	Baseline and Day 7
Change From Baseline in Natural Killer (NK) Cells on Day 7		Baseline and Day 7
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	An AE is any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment-emergent were events between first dose to 35 days after last dose, that were absent before treatment or that worsened relative to pretreatment state.	Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42)
Number of Participants With Treatment Related AEs	An AE is any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. Relatedness was judged by investigator.	Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42)
Number of Participants With Serious AEs (SAEs)	An AE is any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.	Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42)
Number of Participants With AEs Leading to Treatment Discontinuation	An AE is any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.	Day 1 up to Day 7
Number of Participants With Clinically Significant Abnormalities in Vital Signs	Vital signs evaluation included blood pressure and heart rate measurements. Clinical significance of any vital sign abnormality was judged by investigator.	Day 1 up to Day 7
Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Values	Clinical laboratory parameters included haematology: haemoglobin, haematocrit, red blood cells count, platelet count, white blood cells count, total absolute: neutrophils, eosinophils, monocytes, basophils, lymphocytes; chemistry: urea and creatinine estimated creatinine clearance, glucose (fasting), sodium, potassium, chloride, aspartate aminotransferase (AT), alanine AT, total bilirubin, alkaline phosphatase, albumin, total protein; urinalysis: local dipstick: pH, qualitative: glucose, protein, albuminuria, blood, ketones, nitrites, leukocyte esterase and others. Clinical significance of any laboratory abnormality was judged by investigator.	Day 1 up to Day 7
Number of Participants as Per Score for Pediatric Taste Assessment Questionnaire	The pediatric taste questionnaire included 3 questions regarding: 1) Overall Taste (likeability in tase), 2) Overall Mouthfeel (how the medicine felt) and 3) Overall Volume of Medicine (likeability of the amount of medicine). Each question ranged from 1 (most favorable) to 5 (least favorable), higher scores indicates less liking to medicine. Ritlecitinib was provided as capsules; for administration, the capsules were dissolved in water and the contents of the capsule in water were taken according to the dosing administration instructions.	Day 1 and 7

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): March 2, 2023
• Primary Completion (Actual): August 11, 2023
• Study Completion (Actual): August 11, 2023

Study Registration Dates

• First Submitted: December 6, 2022
• First Submitted That Met QC Criteria: December 6, 2022
• First Posted (Actual): December 14, 2022

Study Record Updates

• Last Update Posted (Actual): October 8, 2024
• Last Update Submitted That Met QC Criteria: June 25, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Pathological Conditions, Anatomical; Hypotrichosis; Hair Diseases; Alopecia; Alopecia Areata
• Other Study ID Numbers: B7981031

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No