A Randomized Comparative Study Between Liquid (Tirosint®-SOL) and Tablet Formulations of Levothyroxine in Neonates and Infants With Congenital Hypothyroidism
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 34
- 试验地点
- 20
- 主要终点
- LT4 dose required to maintain TSH in target range (unit: mcg/kg/day)
研究概览
简要总结
This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed with CH.
Subjects will be randomized in a 2:1 ratio to Treatment (Tirosint®-SOL) or Control (conventional therapy with levothyroxine sodium crushed tablets).
详细描述
Newly diagnosed neonates will be randomly assigned to start therapy with LT4 at the initial dose recommended by the Standard of Care (SOC). Infants already on LT4 therapy will continue at the same daily dose within the randomly assigned treatment group (dose adjustments are allowed, if needed based on laboratory parameters and clinical response). Once enrolled, subjects will be treated and followed for 12 months (±1.5 months), participating in 7-8 study visits, consisting of 6-7 inclinic and 1-2 (or more if follow-up visits are required) telemedicine (TM) visits. The total number of visits depends on the age at inclusion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Day 至 9 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patient aged 0 to 9 months
- •Primary CH diagnosis with elevated TSH and low or normal FT4, requiring treatment with LT4, under either of the following conditions:
- •Neonates newly diagnosed with primary CH and needing to initiate LT4 therapy, or
- •Infants previously diagnosed with primary CH and who are already on LT4 therapy for at least 3 weeks;
- •Provide and comply with the informed consent.
排除标准
- •Preterm neonates with a gestational age < 37 weeks;
- •Low birth weight (LBW) or very low birth weight (VLBW) neonates (weight < 2.5 kg) or VLBW infants (weight < 1.5 kg);
- •Neonates in neonatal intensive care units or requiring admission to NICU or neonates/infants hospitalized or requiring hospitalization or in fragile health conditions (e.g. with serious health problems or complications);
- •Neonates with CH diagnosis > 4 weeks after delivery;
- •Diagnosis of primary gastrointestinal disease:
- •Gastroesophageal reflux requiring medical therapy (beyond thickening of formula or position);
- •Anatomic defects (e.g. intestinal atresia, malrotation, tracheoesophageal fistula, pyloric stenosis, Hirschsprung's disease, gastroschisis);
- •Dietary allergy (e.g. cow's milk protein allergy);
- •Malabsorption related to cystic fibrosis, celiac disease and others;
- •Necrotizing enterocolitis requiring surgical resection;
- •Known or suspected adrenal insufficiency (e.g. congenital adrenal hyperplasia, hypopituitarism);
- •Diagnosis of congenital cardiac disease, cardiac insufficiency or risk for cardiac failure;
- •Diagnosis of chromosomopathy;
- •Diagnosis of central hypothyroidism;
- •Hypersensitivity to glycerol;
- •Concomitant anticonvulsant medications, liothyronine, combination of LT4 and liothyronine, thyroid extracts and/or chronic or long-term use of systemic glucocorticoids
- •History of nonadherence with medication or medical visit schedule; or
- •Any condition for which, participation would not be in the best interest of the patient or that could limit protocol specified assessments.
研究组 & 干预措施
Treatment
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
干预措施: Tirosint®-SOL (Drug)
Control
Crushed levothyroxine sodium tablets
干预措施: Levothyroxine Sodium (Drug)
结局指标
主要结局
LT4 dose required to maintain TSH in target range (unit: mcg/kg/day)
时间窗: Up to 22 months based on age group
The LT4 dose is calculated based on the daily LT4 dose (mcg) used in the time period preceding the visit (or the average daily dose on a weekly basis if more than one strength is used over the course of the week) and the body weight (kg) measured during the visit.
次要结局
- Growth pattern for length of body(Up to 22 months based on age group)
- Frequency of dose adjustments(Up to 22 months based on age group)
- Number of events of FT4 values below the middle of the laboratory normal range(Up to 22 months based on age group)
- Hormonal profile for FT4(Up to 22 months based on age group)
- Hormonal profile for TSH(Up to 22 months based on age group)
- Parent/caregiver reports of satisfaction and ease of administration(Up to 22 months based on age group)
- Number of events of TSH values above 4.5 mU/L(Up to 22 months based on age group)
- Subject acceptance of the treatment (CareCAT)(Up to 22 months based on age group)
- Signs and symptoms of hypothyroidism(Up to 22 months based on age group)
- Growth pattern for body weight(Up to 22 months based on age group)
- Growth pattern for head circumference(Up to 22 months based on age group)
- Signs and symptoms of hyperthyroidism(Up to 22 months based on age group)