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
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 15
- 试验地点
- 9
- 主要终点
- Area Under the Plasma Concentration-Time Profile Over the Dosing Interval of 24 Hours, at Steady State (AUC24ss/AUCtau) of Ritlecitinib on Day 7
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •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).
- •Any present malignancies or history of malignancies, history of any lymphoproliferative disorder
- •History (one or more episodes) of CMV, varicella, herpes zoster (shingles) or disseminated herpes simplex.
- •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.
- •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.
研究组 & 干预措施
Ritlecitinib 20 mg
Participants will receive Ritlecitinib 20 mg by mouth once daily (QD).
干预措施: Ritlecitinib 20 mg (Drug)
结局指标
主要结局
Area Under the Plasma Concentration-Time Profile Over the Dosing Interval of 24 Hours, at Steady State (AUC24ss/AUCtau) of Ritlecitinib on Day 7
时间窗: 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]
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.
次要结局
- Change From Baseline in B Lymphocytes on Day 7(Baseline and Day 7)
- 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(0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7)
- Apparent Volume of Distribution (Vz/F) of Ritlecitinib(0 (pre-dose), 0.5, 1, 3 and 8 hours post-dose on Day 7)
- Elimination Half-Life (t1/2) of Ritlecitinib(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(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)(Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42))
- Number of Participants With Treatment Related AEs(Day 1 of dosing up to 35 days after the last dose (maximum up to Day 42))
- Number of Participants With Serious AEs (SAEs)(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(Day 1 up to Day 7)
- Number of Participants With Clinically Significant Abnormalities in Vital Signs(Day 1 up to Day 7)
- Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Values(Day 1 up to Day 7)
- Number of Participants as Per Score for Pediatric Taste Assessment Questionnaire(Day 1 and 7)