Hypothyroidism, Trisomy 21
Conditions
Keywords
Tolerability, Adherence
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.
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.
Interventions
DRUGLiquid stable levothyroxine (L-T4) Tirosint-SOL
Investigation of tolerability, palatability, and adherence of liquid levothyroxine (L-T4) for 8 weeks.
DRUGOral tablet levothyroxine (L-T4)
Investigation of tolerability, palatability, and adherence of maintained oral tablet levothyroxine (L-T4) for 8 weeks
Sponsors
Children's Mercy Hospital Kansas City
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
OTHER
Masking
NONE
Intervention model description
Phase 4, single-center, pilot, randomized, open-label, cross-over study
Eligibility
Sex/Gender
ALL
Age
2 Months to 4 Years
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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. | 2 |
| 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. | 4 |
| Total | 6 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Study terminated | 0 | 1 |
Baseline characteristics
| Characteristic | 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 | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 2 Participants | 4 Participants | 6 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) White | 1 Participants | 2 Participants | 3 Participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 2 Participants | 4 Participants | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 4 |
| other Total, other adverse events | 0 / 2 | 0 / 4 |
| serious Total, serious adverse events | 0 / 2 | 0 / 4 |
Outcome results
Primary
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.
Time frame: 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.
Secondary
Adherence to Taking Study Medication
Evaluate differences in compliance with liquid stable L-T4 compared to L-T4 tablet. Evaluated in percentage of days compliant.
Time frame: 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.