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Trial Outcomes & Findings for Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients (NCT NCT04747275)

NCT ID: NCT04747275

Last Updated: 2023-09-13

Results Overview

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

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

6 participants

Primary outcome timeframe

4 months

Results posted on

2023-09-13

Participant Flow

Participant milestones

Participant milestones
Measure
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.
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.
Overall Study
STARTED
2
4
Overall Study
COMPLETED
2
3
Overall Study
NOT COMPLETED
0
1

Reasons for withdrawal

Reasons for withdrawal
Measure
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.
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.
Overall Study
Study terminated
0
1

Baseline Characteristics

Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Liquid Stable Levothyroxine (l-T4) TirosintSOL First, Then Oral Tablet Levothyroxine (L-T4)
n=2 Participants
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.
Oral Tablet Levothyroxine (L-T4) First, Then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL
n=4 Participants
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.
Total
n=6 Participants
Total of all reporting groups
Age, Categorical
<=18 years
2 Participants
n=5 Participants
4 Participants
n=7 Participants
6 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Sex: Female, Male
Male
2 Participants
n=5 Participants
4 Participants
n=7 Participants
6 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
White
1 Participants
n=5 Participants
2 Participants
n=7 Participants
3 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
2 Participants
n=7 Participants
2 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 4 months

Population: 6 subjects were enrolled and 5 completed the study, however, due to concerns with data collection and integrity, the overseeing Institutional Review Board determined that results for these subjects should not be analyzed. No further subjects were recruited prior to the study closing.

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 4 months

Population: 6 subjects were enrolled and 5 completed the study, however, due to concerns with data collection and integrity, the overseeing Institutional Review Board determined that results for these subjects should not be analyzed. No further subjects were recruited prior to the study closing.

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

Outcome measures

Outcome data not reported

Adverse Events

Liquid Stable Levothyroxine (l-T4) TirosintSOL First, Then Oral Tablet Levothyroxine (L-T4)

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Oral Tablet Levothyroxine (L-T4) First, Then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Max Feldt

Children's Mercy

Phone: 816-960-8892

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place