Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE4

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-01-18

Study Completion Date

2021-11-01

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hypothyroidism Trisomy 21

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Phase 4, single-center, pilot, randomized, open-label, cross-over study

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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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.

Group Type OTHER

Liquid stable levothyroxine (L-T4) Tirosint-SOL

Intervention Type DRUG

Investigation of tolerability, palatability, and adherence of liquid levothyroxine (L-T4) for 8 weeks.

Oral tablet levothyroxine (L-T4)

Intervention Type DRUG

Investigation of tolerability, palatability, and adherence of maintained oral tablet levothyroxine (L-T4) for 8 weeks

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.

Group Type OTHER

Liquid stable levothyroxine (L-T4) Tirosint-SOL

Intervention Type DRUG

Investigation of tolerability, palatability, and adherence of liquid levothyroxine (L-T4) for 8 weeks.

Oral tablet levothyroxine (L-T4)

Intervention Type DRUG

Investigation of tolerability, palatability, and adherence of maintained oral tablet levothyroxine (L-T4) for 8 weeks

Interventions

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Liquid stable levothyroxine (L-T4) Tirosint-SOL

Investigation of tolerability, palatability, and adherence of liquid levothyroxine (L-T4) for 8 weeks.

Intervention Type DRUG

Oral tablet levothyroxine (L-T4)

Investigation of tolerability, palatability, and adherence of maintained oral tablet levothyroxine (L-T4) for 8 weeks

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

2 Months

Maximum Eligible Age

4 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No