Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients

NCT04747275 | Terminated Phase 4 Results FDA Drug

• 1 other identifier: STUDY00001385
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Hypothyroidism; Trisomy 21

Keywords

Tolerability; Adherence

Outcome Measures

Primary Outcomes (1)

• Tolerability as Assessed by the CareCAT Tool

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

  4 months

Secondary Outcomes (1)

• Adherence to Taking Study Medication

  4 months

Study Arms (2)

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

OTHER

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.

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

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

OTHER

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.

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

Interventions

Liquid stable levothyroxine (L-T4) Tirosint-SOL DRUG

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

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

Oral tablet levothyroxine (L-T4) DRUG

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

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

Eligibility Criteria

• Age: 2 Months - 4 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Mercy Hospital Kansas City: lead

Study Sites (1)

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

MeSH Terms

Conditions

Hypothyroidism; Down Syndrome

Interventions

Thyroxine

Condition Hierarchy (Ancestors)

Thyroid Diseases; Endocrine System Diseases; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Thyroid Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Max Feldt
• Organization: Children's Mercy

mmfeldt@cmh.edu

816-960-8892

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physician, DO

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

Study Record Dates

• First Submitted: February 3, 2021
• First Posted: February 10, 2021
• Study Start: January 18, 2021
• Primary Completion: November 1, 2021
• Study Completion: November 1, 2021
• Last Updated: September 13, 2023
• Results First Posted: April 10, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)