Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 150
- 试验地点
- 2
- 主要终点
- Serum Free Thyroxine (FT4) value
研究概览
简要总结
The aim of this multicentric, randomised, two-arms and single-blinded clinical trial is to prospectively evaluate OptiThyDose for Congenital hypothyroidism (CH) and Graves' disease (GD).
详细描述
Thyroid diseases can affect people from birth to adulthood, ith some being present at birth (congenital) and others developing later in life (acquired). These diseases need to be treated quickly and properly because if left untreated, they can impact brain development, thinking abilities, growth, puberty, and other important body functions. However, treating thyroid diseases in children can be challenging, as it's important to avoid both under- and overdosing.
Algorithms that help determine the best individual dose for children with thyroid diseases could reduce the risk of long-term problems, like impaired thinking and growth. This is especially important because cases of thyroid diseases in children are increasing worldwide.
OptiThyDose is a new mathematical model developed to help doctors find the right dose for children with thyroid diseases.
The primary goal of this multicentric, randomised, two-arms and single-blinded study is to test how well OptiThyDose works for children with two types of thyroid diseases: Congenital Hypothyroidism (CH) and Graves' Disease (GD).
If proven effective, OptiThyDose could help ensure more accurate dosing of thyroid medications, leading to better hormone control, fewer side effects, and improved health outcomes in children with Congenital Hypothyroidism (CH) and Graves' Disease (GD).
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
- Single (Participant)
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Congenital hypothyroidism (CH)
- •Newborns with pathological neonatal screening and confirmation of an increased Thyrotropin (TSH) level in an independent venous blood sample
- •Graves' disease (GD)
- •Children until 18 years with new diagnosis of GD, recurrence of GD, or insufficiently controlled GD under CMZ/MMZ during follow-up according to:
- •Pathological lab values (suppressed TSH, increased thyroid hormone levels, positive Anti-TSH-receptor antibodies)
- •Typical clinical picture, if present (goitre, tachycardia, palpitations, weight loss, hyperphagia, altered mood)
- •The study participant must be accessible for scheduled visits, treatment and follow-up.
- •Signed Informed Consent form (ICF) obtained prior to any study related procedure. Written IC for study participation must be signed and dated by the patient and/or his/her legal representative(s) in accordance with national legal requirements
排除标准
- •Exclusion of newborns from mothers with GD
- •Exclusion of patients in case of a life-threatening event
- •Exclusion of children with known other aetiologies of hyperthyroidism than GD without elevated Anti-TSH-receptor antibodies e.g.:
- •known toxic thyroid nodules proven by ultrasound/scintigraphy
- •known amiodarone induced hyperthyroidism
- •known McCune Albright syndrome (based on clinical, laboratory, and genetic diagnosis) associated hyperthyroidism
- •known genetically proven hyperthyroidism caused by activating mutations of the TSH receptor gene
研究组 & 干预措施
Control group w/ Congenital Hypothyroidism
Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
OptiThyDose w/ Congenital Hypothyroidism
Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
干预措施: OptiThyDose (Other)
Control group w/ Graves' Disease
Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
OptiThyDose w/ Graves' Disease
Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
干预措施: OptiThyDose (Other)
结局指标
主要结局
Serum Free Thyroxine (FT4) value
时间窗: 90 days post treatment start
The serum Free Thyroxine (FT4) values is evaluated. FT4, interpreted according to the age of patients, is used in clinical routine as marker of the adequacy of: * Thyroid hormone substitution with LT4 of insufficient thyroid function in patients with CH (low FT4 levels in case of under-dosing of LT4, high FT4 levels in case of over-dosing of LT4) * Suppression of overactive thyroid function with CMZ/MMZ in patients with GD (low FT4 levels in case of over-dosing of CMZ/MMZ, high FT4 levels in case of under-dosing of CMZ/MMZ)
次要结局
- Proportion of Thyroid Hormone Levels Within Target Range(90 days post treatment start and up to 1 year post treatment start)
- Deviations from Local Laboratory Reference Ranges for Thyroid Hormones(Up to 1 year post treatment start)
- Number of clinical visits(Up to 1 year post treatment start)
- Disease-related adverse events(Up to 1 year post treatment start)
- Average daily dose of administered drugs per kg(Up to 1 year post treatment start)