Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients

• Sponsor: Children's Mercy Hospital Kansas City (Other)
• Overall Status: Terminated
• CT.gov ID: NCT04747275
• Collaborator: (none)
• Enrollment: 6
• Location: 1
• Arms: 2
• Actual Duration (Months): 9.4
• Patients Per Site Per Month: 0.6

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hypothyroidism; Trisomy 21	Drug: Oral tablet levothyroxine (L-T4); Drug: Liquid stable levothyroxine (L-T4) Tirosint-SOL	Phase 4

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 6 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Intervention Model Description: Phase 4, single-center, pilot, randomized, open-label, cross-over studyPhase 4, single-center, pilot, randomized, open-label, cross-over study
• Masking: None (Open Label)
• Primary Purpose: Other
• Official Title: Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
• Actual Study Start Date: Jan 18, 2021
• Actual Primary Completion Date: Nov 1, 2021
• Actual Study Completion Date: Nov 1, 2021

Arms and Interventions

Arm	Intervention/Treatment
Other: Maintain oral tablet levothyroxine (L-T4) Arm 1: This arm will include patients on tablet L-T4. In each arm of the study, patients will be given an 8 week supply of either tablet L-T4 or liquid stable L-T4 Tirosint-SOL® . After 8 weeks patients will cross over to the second arm of the study for additional 8 weeks.	Drug: Oral tablet levothyroxine (L-T4); Investigation of tolerability, palatability, and adherence of maintained oral tablet levothyroxine (L-T4)
Other: Receive liquid stable levothyroxine (L-T4) Tirosint-SOL® Arm 2: This arm will receive liquid stable L-T4 Tirosint-SOL® If currently on tablet L-T4, the dose will be the same for Tirosint-SOL® In each arm of the study, patients will be given an 8 week supply of either tablet L-T4 or liquid stable L-T4 Tirosint-SOL® . After 8 weeks patients will cross over to the first arm of the study for additional 8 weeks.	Drug: Liquid stable levothyroxine (L-T4) Tirosint-SOL; Investigation of tolerability, palatability, and adherence of liquid levothyroxine (L-T4)

Outcome Measures

Primary Outcome Measures

1. Tolerability as assessed by the CareCAT Tool [4 months]

   Utilize medication tolerability questionnaire, Care Children's Acceptance Tool (CAT), to understand parental perceptions of liquid stable L-T4.

Secondary Outcome Measures

1. Adherence to taking study medication [4 months]

   Evaluate differences in compliance with liquid stable L-T4 compared to L-T4 tablet. Evaluated in percentage of days compliant.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Months to 4 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Mercy Hospital	Kansas City	Missouri	United States	64108

Sponsors and Collaborators

• Children's Mercy Hospital Kansas City

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Children's Mercy Hospital Kansas City
• ClinicalTrials.gov Identifier: NCT04747275
• Other Study ID Numbers: STUDY00001385
• First Posted: Feb 10, 2021
• Last Update Posted: Jul 1, 2022
• Last Verified: Jun 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No
• Keywords provided by Children's Mercy Hospital Kansas City: Tolerability; Adherence
• Additional relevant MeSH terms: Down Syndrome; Hypothyroidism; Trisomy

Study Results

No Results Posted as of Jul 1, 2022