Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients

Children's Mercy Hospital Kansas City

Status and phase: Terminated; Phase 4

Conditions: Trisomy 21; Hypothyroidism

Treatments: Drug: Liquid stable levothyroxine (L-T4) Tirosint-SOL; Drug: Oral tablet levothyroxine (L-T4)

Study type: Interventional

Funder types: Other

Identifiers

NCT04747275

STUDY00001385

Details and patient eligibility

About

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.

Full 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.

Enrollment: 6 patients

Sex: All

Ages: 2 months to 4 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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6 participants in 2 patient groups

Liquid Stable Levothyroxine (l-T4) TirosintSOL first, then Oral Tablet Levothyroxine (L-T4)

Other group

Description:

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.

Treatment:

Drug: Oral tablet levothyroxine (L-T4)

Drug: Liquid stable levothyroxine (L-T4) Tirosint-SOL

Oral Tablet Levothyroxine (L-T4) first, then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL

Other group

Description:

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.

Treatment:

Drug: Oral tablet levothyroxine (L-T4)

Drug: Liquid stable levothyroxine (L-T4) Tirosint-SOL