Congenital HypothyroidismPhase 4NCT05228184

Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)

A Phase 4 study testing Tirosint®-SOL and Levothyroxine Sodium in people with Congenital Hypothyroidism, run by IBSA Institut Biochimique SA at 10 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed with CH. Subjects will be randomized in a 2:1 ratio to Treatment (Tirosint®-SOL) or Control (conventional therapy with levothyroxine sodium crushed tablets). Read more about this study

Who is it for?: Ages 1 day to 9 months
Conditions the study is for: Congenital Hypothyroidism
What is being tested?: Tirosint®-SOL and Levothyroxine Sodium
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 34 participants
Where: 10 sitesUnited States
Study dates: 21 Jan 2022to 27 Mar 2025
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 1 day to 9 months
All sexes
No healthy volunteers
Child

Who may be able to join

Male and female patient aged 0 to 9 months
Primary CH diagnosis with elevated TSH and low or normal FT4, requiring treatment with LT4, under either of the following conditions:
Neonates newly diagnosed with primary CH and needing to initiate LT4 therapy, or
Infants previously diagnosed with primary CH and who are already on LT4 therapy for at least 3 weeks;
Provide and comply with the informed consent.

Who cannot join

Preterm neonates with a gestational age < 37 weeks;
Low birth weight (LBW) or very low birth weight (VLBW) neonates (weight < 2.5 kg) or VLBW infants (weight < 1.5 kg);
Neonates in neonatal intensive care units or requiring admission to NICU or neonates/infants hospitalized or requiring hospitalization or in fragile health conditions (e.g. with serious health problems or complications);
Neonates with CH diagnosis > 4 weeks after delivery;
Diagnosis of primary gastrointestinal disease:
Gastroesophageal reflux requiring medical therapy (beyond thickening of formula or position);

Show all 18Show fewer

Anatomic defects (e.g. intestinal atresia, malrotation, tracheoesophageal fistula, pyloric stenosis, Hirschsprung's disease, gastroschisis);
Dietary allergy (e.g. cow's milk protein allergy);
Malabsorption related to cystic fibrosis, celiac disease and others;
Necrotizing enterocolitis requiring surgical resection;
Known or suspected adrenal insufficiency (e.g. congenital adrenal hyperplasia, hypopituitarism);
Diagnosis of congenital cardiac disease, cardiac insufficiency or risk for cardiac failure;
Diagnosis of chromosomopathy;
Diagnosis of central hypothyroidism;
Hypersensitivity to glycerol;
Concomitant anticonvulsant medications, liothyronine, combination of LT4 and liothyronine, thyroid extracts and/or chronic or long-term use of systemic glucocorticoids
History of nonadherence with medication or medical visit schedule; or
Any condition for which, participation would not be in the best interest of the patient or that could limit protocol specified assessments.

Trial locations and contacts

10 sites in 1 country.

City, postcode or hospital

10 sites in 1 country

United States10 sites

Children's Hospital of Atlanta
Atlanta, Georgia, 30329
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Cook Children's Health Care Systems
Fort Worth, Texas, 76104
Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64111
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Yale University
New Haven, Connecticut, 06511
CHOC Children's Hospital
Orange, California, 92868
University of California San Francisco
San Francisco, California, 94143

Other trials in United States

About this study

Official title: A Randomized Comparative Study Between Liquid (Tirosint®-SOL) and Tablet Formulations of Levothyroxine in Neonates and Infants With Congenital Hypothyroidism

This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed with CH.

Subjects will be randomized in a 2:1 ratio to Treatment (Tirosint®-SOL) or Control (conventional therapy with levothyroxine sodium crushed tablets).

Read the detailed description

Newly diagnosed neonates will be randomly assigned to start therapy with LT4 at the initial dose recommended by the Standard of Care (SOC). Infants already on LT4 therapy will continue at the same daily dose within the randomly assigned treatment group (dose adjustments are allowed, if needed based on laboratory parameters and clinical response). Once enrolled, subjects will be treated and followed for 12 months (±1.5 months), participating in 7-8 study visits, consisting of 6-7 inclinic and 1-2 (or more if follow-up visits are required) telemedicine (TM) visits. The total number of visits depends on the age at inclusion.

Conditions: Congenital Hypothyroidism
Being tested: Tirosint®-SOL, Levothyroxine Sodium
Medical subject headings: Congenital Hypothyroidism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Tirosint®-SOLDrug
Also called levothyroxine sodium oral solution (liquid)
Dosage will be according to the USPI and Standard of Care.
Levothyroxine SodiumDrug
Also called levothyroxine sodium tablets
Tablets will be crushed and dissolved in solution. Dosage will be according to the USPI and Standard of Care.

Study groups in NCT05228184
Study group	Role	What they receive
Treatment	Experimental	Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg. Receives: Drug: Tirosint®-SOL
Control	Active comparator	Crushed levothyroxine sodium tablets Receives: Drug: Levothyroxine Sodium

What is being measured

The primary outcome is the main question the study is designed to answer.

LT4 Dose Required to Maintain TSH in Target Range (Unit: mcg/kg/Day)
The LT4 dose is calculated based on the daily LT4 dose (mcg) used in the time period preceding the visit (or the average daily dose on a weekly basis if more than one strength is used over the course of the week) and the body weight (kg) measured during the visit
Measured: 1 year of age

Secondary outcomes (12)

Hormonal Profile for TSH
TSH (unit mIU/L)
Measured: 1 year of age
Hormonal Profile for FT4
FT4 (unit pmol/L)
Measured: 1 year of age
Frequency of Dose Adjustments
Percent number of subjects (%) who need a dose adjustment in the long-term follow up phase
Measured: Up to 22 months based on age group
Number of Events of TSH Values Above 4.5 mU/L
TSH values above 4.5 mU/L
Measured: Up to 22 months based on age group
Number of Events of FT4 Values Below the Middle of the Laboratory Normal Range
FT4 values below the middle of the laboratory normal range
Measured: Up to 22 months based on age group
Growth Pattern for Length of Body
Subject growth measurements in Length (cm) of body
Measured: 1 year of age
Growth Pattern for Body Weight
Subject growth measurements in Body weight (kg)
Measured: 1 year of age
Growth Pattern for Head Circumference
Subject growth measurements in Head circumference (cm)
Measured: 1 year of age
Parent/Caregiver Reports of Satisfaction and Ease of Administration
Parents Satisfaction Questionnaire is a study-specific tool measuring coping and mental well-being and satisfaction in 20 questions.
12 of these questions were scored from 1 to 5, with higher score meaning better outcome.
Subject scores were calculated for each subject by visit as mean of the LN(single question score) [min=0; max=1.61].
Measured: 3 weeks after treatment start
Subject Acceptance of the Treatment (CareCAT)
Caregiver Administered Children's Acceptance Tool (CareCAT) is a 5-point nominal scale used to assess the acceptance of oral medicines in infants and toddlers who are unable to verbally give their opinion about a medicine
Measured: first treatment week
Signs and Symptoms of Hypothyroidism
Total number of subjects experiencing hypothyroidism's signs and symptoms
Measured: Up to 22 months based on age group
Signs and Symptoms of Hyperthyroidism
Total number of subjects experiencing hyperthyroidism's signs and symptoms
Measured: Up to 22 months based on age group

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

21 Jan 202227 Mar 2025

First posted8 Feb 2022
Study start21 Jan 2022
Primary completion27 Mar 2025
Study completion27 Mar 2025

Why it stopped: Inability to recruit subjects

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Congenital HypothyroidismStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 34 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05228184
Sponsor's protocol ID: 20US-T414
Status last verified: Jun 2026
First submitted: 21 Dec 2021
Last updated: 26 Jun 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Clinical and Basic Investigations Into Known and Suspected Congenital Disorders of Glycosylation
Congenital Disorders of Glycosylation
RecruitingObservational study
Understanding the Subnormal Rescue ofNeurological Development in Congenital Hypothyroidism (2022E5SZEM)
Congenital Hypothyroidism, Neurodevelopmental Disorders
Recruiting
Prospective Evaluation of OptiThyDose
Thyroid Diseases, Congenital Hypothyroidism
RecruitingObservational study
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

All Congenital Hypothyroidism trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized Comparative Study Between Liquid (Tirosint®-SOL) and Tablet Formulations of Levothyroxine in Neonates and Infants With Congenital Hypothyroidism
ClinicalTrials.gov ID: NCT05228184
Acronym / sponsor ID: 20US-T414
Conditions: Congenital Hypothyroidism
Phase: Phase 4
Study type: Interventional
Recruitment status: TerminatedReason stopped: Inability to recruit subjects
Enrolment: 34 participants (actual)
Lead sponsor: IBSA Institut Biochimique SAIndustry (pharma, biotech, devices)
Collaborators: Cromsource
Study start: 21 Jan 2022 (actual)
Primary completion: 27 Mar 2025 (actual)
Study completion: 27 Mar 2025 (actual)
First posted: 8 Feb 2022 (actual)
Results: Posted 29 May 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed with CH.

Subjects will be randomized in a 2:1 ratio to Treatment (Tirosint®-SOL) or Control (conventional therapy with levothyroxine sodium crushed tablets).

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Treatment	Experimental	Drug: Tirosint®-SOLTirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
Control	Active comparator	Drug: Levothyroxine SodiumCrushed levothyroxine sodium tablets

Who may qualify

Ages: 1 day to 9 months
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

Male and female patient aged 0 to 9 months
Primary CH diagnosis with elevated TSH and low or normal FT4, requiring treatment with LT4, under either of the following conditions:
Provide and comply with the informed consent.

Key exclusion criteria (18 listed)

Preterm neonates with a gestational age < 37 weeks;
Low birth weight (LBW) or very low birth weight (VLBW) neonates (weight < 2.5 kg) or VLBW infants (weight < 1.5 kg);
Neonates in neonatal intensive care units or requiring admission to NICU or neonates/infants hospitalized or requiring hospitalization or in fragile health conditions (e.g. with serious health problems or complications);
Neonates with CH diagnosis > 4 weeks after delivery;
Diagnosis of primary gastrointestinal disease:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Tirosint®-SOLAlso: levothyroxine sodium oral solution (liquid)Dosage will be according to the USPI and Standard of Care.	Drug	Treatment
Levothyroxine SodiumAlso: levothyroxine sodium tabletsTablets will be crushed and dissolved in solution. Dosage will be according to the USPI and Standard of Care.	Drug	Control

Primary outcome

LT4 Dose Required to Maintain TSH in Target Range (Unit: mcg/kg/Day)
Time frame: 1 year of age
The LT4 dose is calculated based on the daily LT4 dose (mcg) used in the time period preceding the visit (or the average daily dose on a weekly basis if more than one strength is used over the course of the week) and the body weight (kg) measured during the visit

12 secondary outcomes

Hormonal Profile for TSH
Time frame: 1 year of age
Hormonal Profile for FT4
Time frame: 1 year of age
Frequency of Dose Adjustments
Time frame: Up to 22 months based on age group
Number of Events of TSH Values Above 4.5 mU/L
Time frame: Up to 22 months based on age group
Number of Events of FT4 Values Below the Middle of the Laboratory Normal Range
Time frame: Up to 22 months based on age group
Growth Pattern for Length of Body
Time frame: 1 year of age
Growth Pattern for Body Weight
Time frame: 1 year of age
Growth Pattern for Head Circumference
Time frame: 1 year of age
Parent/Caregiver Reports of Satisfaction and Ease of Administration
Time frame: 3 weeks after treatment start
Subject Acceptance of the Treatment (CareCAT)
Time frame: first treatment week
Signs and Symptoms of Hypothyroidism
Time frame: Up to 22 months based on age group
Signs and Symptoms of Hyperthyroidism
Time frame: Up to 22 months based on age group

Results and safety

Results posted to the registry on 29 May 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Treatment	20	16
Control	11	10

Primary outcome: LT4 Dose Required to Maintain TSH in Target Range (Unit: mcg/kg/Day)

Time frame: 1 year of age. Mean (Standard Deviation). Unit: mcg/kg/day

Measure	Treatment n = 17	Control n = 10
Value	3.73 (1.469)	5.06 (1.127)

Treatment vs Control: p = 0.0044, Wilcoxon (Mann-Whitney)

Population analysed: intent-to-treat (total number of subjects with data at the visit of 1 year of age = 27)

12 secondary outcomes are reported in the full results.

Adverse events

Time frame: from enrolment until end of follow-up, up to 1 year of treatment. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Treatment	2 of 20 (10%)	15 of 20 (75%)	0 of 20
Control	1 of 11 (9.1%)	8 of 11 (72.7%)	0 of 11

Limitations and caveats (from the sponsor): The study was planned to enroll 126 subjects. Despite extension of timelines, enrollment was terminated with 31 randomized subjects for inability to recruit. Due to the very limited number of subjects randomized, the real effect of the intervention cannot be reliably established. Moreover, it was not possible to perform the analysis on the per-protocol population, nor exploratory analyses in neonates (time to normalize TSH and FT4 in neonates).

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Congenital Hypothyroidism

RecruitingObservational study
Clinical and Basic Investigations Into Known and Suspected Congenital Disorders of Glycosylation
Congenital Disorders of Glycosylation
RecruitingObservational study
Understanding the Subnormal Rescue ofNeurological Development in Congenital Hypothyroidism (2022E5SZEM)
Congenital Hypothyroidism, Neurodevelopmental Disorders
Recruiting
Prospective Evaluation of OptiThyDose
Thyroid Diseases, Congenital Hypothyroidism
RecruitingObservational study
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Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Giuseppe Mautone study director

IBSA Head of R&D Scientific Affairs

10 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Tirosint®-SOL and Levothyroxine Sodium in people with Congenital Hypothyroidism, run by IBSA Institut Biochimique SA at 10 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 26 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 May 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

LT4 Dose Required to Maintain TSH in Target Range (Unit: mcg/kg/Day) (time frame: 1 year of age). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05228184.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2026.