Trial Outcomes & Findings for Targeted Levothyroxine Dosing in Infants With Congenital Hypothyroidism (NCT NCT02374593)

NCT ID: NCT02374593

Last Updated: 2017-10-09

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 24 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2017-10-09

Participant Flow

Participant milestones

Measure	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.	Historical Control Patients in this arm were treated with levothyroxine empirically with an initial dose between 10-15 mcg/kg/day.
Overall Study STARTED	24	44
Overall Study COMPLETED	22	44
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	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.	Historical Control Patients in this arm were treated with levothyroxine empirically with an initial dose between 10-15 mcg/kg/day.
Overall Study non-compliant	2	0

Baseline Characteristics

Targeted Levothyroxine Dosing in Infants With Congenital Hypothyroidism

Baseline characteristics by cohort

Measure	Targeted Dosing n=22 Participants 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.	Historical Control n=44 Participants Patients in this arm were treated with levothyroxine empirically with an initial dose between 10-15 mcg/kg/day.	Total n=66 Participants Total of all reporting groups
Age, Continuous	1 weeks n=5 Participants	1 weeks n=7 Participants	1 weeks n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	28 Participants n=7 Participants	42 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	16 Participants n=7 Participants	24 Participants n=5 Participants
Region of Enrollment United States	22 participants n=5 Participants	44 participants n=7 Participants	66 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	Targeted Dosing n=22 Participants 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.	Historical Control n=44 Participants Patients in this arm were treated with levothyroxine empirically with an initial dose between 10-15 mcg/kg/day.	Patients With Eutopic Thyroids patients with eutopic thyroids	Historical Controls see title
Dose Adjustments	2.5 Number of dose adjustments Standard Error 0.334	2.4 Number of dose adjustments Standard Error 0.211	—	—

SECONDARY outcome

Timeframe: 6 months

Whether the dose adjustment was made for overtreatment was noted as based on TSH and fT4/T4 results

Outcome measures

Measure	Targeted Dosing n=10 Participants 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.	Historical Control n=4 Participants Patients in this arm were treated with levothyroxine empirically with an initial dose between 10-15 mcg/kg/day.	Patients With Eutopic Thyroids n=8 Participants patients with eutopic thyroids	Historical Controls n=44 Participants see title
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	2 dose adjustments Standard Error 0.275	1 dose adjustments Standard Error 0.275	2 dose adjustments Standard Error 0.275

Adverse Events

Targeted Dosing

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Historical Control

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

Dr. Erica Eugster

Riley Hospital for Children

Phone: 317 944-3889

Email: eeugster@iu.edu

Results disclosure agreements

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