Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Tolerability as Assessed by the CareCAT Tool
研究概览
简要总结
Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption. For an age group unable to swallow tablets whole by mouth, tablets must be crushed and suspended in water, breast milk or formula for administration in order to treat children with hypothyroidism. For this age group, ease of administration may have a significant impact on compliance and ability to remain euthyroid. We propose that Tirosint-SOL® will be more favorably received due to ease of administration, improved tolerability and palatability, therefore leading to improved adherence when compared to L-T4 tablets.
详细描述
Hypothyroidism is a common medical disorder in children with Trisomy 21 (T21). Treatment with oral tablet levothyroxine (L-T4) is primarily used to treat this condition. However, children with T21 have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption. For an age group unable to swallow tablets whole by mouth, tablets must be crushed and suspended in water, breast milk or formula for administration in order to treat children with hypothyroidism. For this age group, ease of administration may have a significant impact on compliance and ability to remain euthyroid. Until recently, there was no liquid preparation of L-T4 in the United States for use in children. Tirosint-SOL® (liquid stable L-T4) is now FDA-approved for use in children. The Investigator proposes that Tirosint-SOL® will be more favorably received due to ease of administration, improved tolerability and palatability, therefore leading to improved adherence when compared to L-T4 tablets.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 2 Months 至 4 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Trisomy 21 children age 2 months to less than 5 years of age
- •Prior confirmed diagnosis of congenital or acquired hypothyroidism
排除标准
- •Gestational age ≤ 35 weeks, concomitant anticonvulsant medications, history of nonadherence with medication or medical visit schedule
- •Subjects must be able to take oral medication, no G-tube or parental fed subjects
研究组 & 干预措施
Liquid Stable Levothyroxine (l-T4) TirosintSOL first, then Oral Tablet Levothyroxine (L-T4)
Participants start on Liquid Tirosint®-SOL for 8 weeks using continued same daily dose of LT4. Dose adjustments are allowed as needed, based on laboratory results and clinical response. Then Participants will crossover to conventional therapy of LT4 tablets for 8 weeks. Participants total participation will be 16 weeks total.
干预措施: Liquid stable levothyroxine (L-T4) Tirosint-SOL (Drug)
Liquid Stable Levothyroxine (l-T4) TirosintSOL first, then Oral Tablet Levothyroxine (L-T4)
Participants start on Liquid Tirosint®-SOL for 8 weeks using continued same daily dose of LT4. Dose adjustments are allowed as needed, based on laboratory results and clinical response. Then Participants will crossover to conventional therapy of LT4 tablets for 8 weeks. Participants total participation will be 16 weeks total.
干预措施: Oral tablet levothyroxine (L-T4) (Drug)
Oral Tablet Levothyroxine (L-T4) first, then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL
Participants continue conventional therapy of LT4 tablets for 8 weeks at current dose, after 8 weeks will crossover to receive Liquid Stable Levothyroxine (L-T4) Tirosint-SOL for 8 weeks. Participants total participation will be 16 weeks total.
干预措施: Liquid stable levothyroxine (L-T4) Tirosint-SOL (Drug)
Oral Tablet Levothyroxine (L-T4) first, then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL
Participants continue conventional therapy of LT4 tablets for 8 weeks at current dose, after 8 weeks will crossover to receive Liquid Stable Levothyroxine (L-T4) Tirosint-SOL for 8 weeks. Participants total participation will be 16 weeks total.
干预措施: Oral tablet levothyroxine (L-T4) (Drug)
结局指标
主要结局
Tolerability as Assessed by the CareCAT Tool
时间窗: 4 months
Utilize medication tolerability questionnaire, Care Children's Acceptance Tool (CAT), to understand parental perceptions of liquid stable L-T4.
次要结局
- Adherence to Taking Study Medication(4 months)
研究者
Matthew Feldt
Physician, DO
Children's Mercy Hospital Kansas City