Congenital Hypothyroidism
Conditions
Brief summary
This is a clinical study comparing targeted levothyroxine dosing based on thyroid anatomy as visualized on ultrasound (normal vs. ectopic/sublingual vs. athyreosis) to empiric levothyroxine dosing in infants with congenital hypothyroidism. Patients enrolled in the study for targeted dosing will be compared to controls obtained by retrospective chart review. The main outcome is to determine if there is a difference in the frequency of over-treatment and under-treatment during the first 6 months of therapy.
Interventions
DRUGLevothyroxine
Levothyroxine dose will be adjusted at the first clinic visit based on thyroid anatomy on ultrasound.
DEVICEUltrasound
Sponsors
Erica Eugster
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 2 Weeks
Healthy volunteers
No
Inclusion criteria
* Newborns diagnosed with congenital hypothyroidism (CH). * Newborns with CH who are treated by a pediatric endocrinologist at Riley Hospital for Children or satellite location. * Newborns with CH who have thyroid imaging performed (thyroid ultrasound or technetium scan) at diagnosis.
Exclusion criteria
* Newborns who do not have CH. * Newborns with CH who have not had thyroid imaging performed at diagnosis.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Dose Adjustments | 6 months | Thyroid labs (TSH and fT4/T4) will be monitored per standard care: 2 weeks after initiation of levothyroxine and once monthly during the first 6 months of treatment. The number of dose adjustments required per participant during the first 6 months of treatment were recorded. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Adjustments Due to Overtreatment in Subjects With Athyreosis, Ectopic and Eutopic Thyroid Glands Compared With Controls | 6 months | Whether the dose adjustment was made for overtreatment was noted as based on TSH and fT4/T4 results |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Targeted Dosing Patients in this arm will receive targeted levothyroxine dosing based on thyroid anatomy on ultrasound as follows: 10 mcg/kg for normal gland, 12 mcg/kg for ectopic gland, 15 mcg/kg for athyreosis. | 22 |
| Historical Control Patients in this arm were treated with levothyroxine empirically with an initial dose between 10-15 mcg/kg/day. | 44 |
| Total | 66 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | non-compliant | 2 | 0 |
Baseline characteristics
| Characteristic | Targeted Dosing | Historical Control | Total |
|---|---|---|---|
| Age, Continuous | 1 weeks | 1 weeks | 1 weeks |
| Region of Enrollment United States | 22 participants | 44 participants | 66 participants |
| Sex: Female, Male Female | 14 Participants | 28 Participants | 42 Participants |
| Sex: Female, Male Male | 8 Participants | 16 Participants | 24 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 0 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 0 |
Outcome results
Primary
Dose Adjustments
Thyroid labs (TSH and fT4/T4) will be monitored per standard care: 2 weeks after initiation of levothyroxine and once monthly during the first 6 months of treatment. The number of dose adjustments required per participant during the first 6 months of treatment were recorded.
Time frame: 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Targeted Dosing | Dose Adjustments | 2.5 Number of dose adjustments | Standard Error 0.334 |
| Historical Control | Dose Adjustments | 2.4 Number of dose adjustments | Standard Error 0.211 |
Secondary
Number of Adjustments Due to Overtreatment in Subjects With Athyreosis, Ectopic and Eutopic Thyroid Glands Compared With Controls
Whether the dose adjustment was made for overtreatment was noted as based on TSH and fT4/T4 results
Time frame: 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Targeted Dosing | Number of Adjustments Due to Overtreatment in Subjects With Athyreosis, Ectopic and Eutopic Thyroid Glands Compared With Controls | 2.7 dose adjustments | Standard Error 0.275 |
| Historical Control | Number of Adjustments Due to Overtreatment in Subjects With Athyreosis, Ectopic and Eutopic Thyroid Glands Compared With Controls | 2 dose adjustments | Standard Error 0.275 |
| Patients With Eutopic Thyroids | Number of Adjustments Due to Overtreatment in Subjects With Athyreosis, Ectopic and Eutopic Thyroid Glands Compared With Controls | 1 dose adjustments | Standard Error 0.275 |
| Historical Controls | Number of Adjustments Due to Overtreatment in Subjects With Athyreosis, Ectopic and Eutopic Thyroid Glands Compared With Controls | 2 dose adjustments | Standard Error 0.275 |