Clinical trial NCT04747275: Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Official title: Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Hypothyroidism
Trisomy 21
Keywords: Tolerability
Adherence
Lead sponsor: Children's Mercy Hospital Kansas City
Sponsor class: OTHER
First posted: 2021-02-10
Enrollment: 6
Sex: ALL
Minimum age (years): 2
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: Phase 4, single-center, pilot, randomized, open-label, cross-over study
Primary purpose: OTHER

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Trisomy 21 children age 2 months to less than 5 years of age
* Prior confirmed diagnosis of congenital or acquired hypothyroidism

Exclusion Criteria:

* 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

Primary outcomes:
measure: Tolerability as Assessed by the CareCAT Tool
timeFrame: 4 months
Secondary outcomes:
measure: Adherence to Taking Study Medication
timeFrame: 4 months
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
Study officials:
References: