What side effects are associated with this type of intervention?

The most common treatments for Congenital Hypothyroidism involve synthetic thyroxine (T4) replacement, such as Tirosint®-SOL and conventional levothyroxine tablets. Known side effects of these treatments can include symptoms of hyperthyroidism if the dose is too high, such as increased heart rate, sweating, weight loss, and anxiety. Other potential side effects include allergic reactions, gastrointestinal issues, and, in rare cases, hair loss. It is crucial to monitor thyroid hormone levels regularly to adjust the dosage and minimize side effects.

What prior FDA approvals exist?

The FDA has approved several synthetic thyroxine (T4) replacement therapies for the treatment of Congenital Hypothyroidism, with levothyroxine being the standard. Approved formulations include tablets, capsules, and liquid solutions such as Tirosint®-SOL. These treatments are designed to normalize thyroid hormone levels, supporting proper growth and development in affected individuals.

What other types of research exist?

Promising alternatives to Tirosint®-SOL for congenital hypothyroidism patients include the new liquid levothyroxine formulation developed by a Greek pharmaceutical company and other liquid or soft gel preparations of levothyroxine, which have shown improved absorption and therapeutic equivalence to traditional tablet forms.

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Thyroid Hormone Replacement

Tirosint-SOL for Congenital Hypothyroidism

Q: What is the mechanism of action of this treatment?

The primary treatment for Congenital Hypothyroidism is synthetic thyroxine (T4) replacement, such as Tirosint®-SOL. This treatment works by providing the body with the hormone thyroxine, which is deficient in patients with Congenital Hypothyroidism. Thyroxine is crucial for normal growth, brain development, and metabolism. By supplementing this hormone, the treatment helps normalize thyroid function, ensuring proper physical and cognitive development in affected infants. Accurate and consistent dosing, as seen with liquid formulations like Tirosint®-SOL, is particularly important to maintain stable thyroid hormone levels and optimize developmental outcomes.

Phase 4

Waitlist Available

Research Sponsored by IBSA Institut Biochimique SA

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Primary CH diagnosis with elevated TSH and low T4, 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 4 weeks

Male and female patient aged 0 to 9 months

Must not have

Neonates in neonatal intensive care units or requiring admission to NICU or neonates/infants hospitalized or requiring hospitalization

Low birth weight and very low birth weight neonates (weight < 2.5 kg)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 22 months based on age group

Awards & highlights

Approved for 5 Other Conditions

All Individual Drugs Already Approved

Pivotal Trial

Drug Has Already Been Approved

No Placebo-Only Group

Summary

This trial is testing whether a liquid thyroid hormone medication (Tirosint®-SOL) is better than crushed tablets for infants with congenital hypothyroidism. The treatment provides the necessary thyroid hormone that these infants need for proper growth and development. Tirosint®-SOL is a liquid formulation of thyroid hormone, which has been shown to provide better thyroid hormone control and improved quality of life compared to tablet forms.

Who is the study for?

This trial is for babies aged 0 to 9 months with Congenital Hypothyroidism. It's open to those newly diagnosed or already on treatment for at least 4 weeks, but not for preterm infants, those in intensive care, with primary gastrointestinal diseases, severe heart conditions, chromosomal disorders, certain medication use or allergies.

What is being tested?

The study compares two forms of thyroid hormone replacement: Tirosint®-SOL and the traditional levothyroxine sodium tablets. Babies will be randomly assigned to either the new liquid solution (2 out of every 3) or the standard crushed tablet form.

What are the potential side effects?

Potential side effects from both treatments are similar since they contain the same active ingredient and may include symptoms like rapid heartbeat, sweating, restlessness or sleep disturbances due to excessive thyroid hormone levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have primary congenital hypothyroidism and need or am on LT4 therapy.

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I am a baby aged 9 months or younger.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My newborn needs or is in intensive care.

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My newborn weighs less than 2.5 kg.

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If you have certain digestive, heart, or hormone-related conditions, or if taking certain medications, participating in this study might not be safe for you.

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My baby was born before 37 weeks of pregnancy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 22 months based on age group

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 22 months based on age group

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 22 months based on age group for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

LT4 dose required to maintain TSH in target range (unit: mcg/kg/day)

Secondary study objectives

Frequency of dose adjustments

Growth pattern for body weight

Growth pattern for head circumference

+9 more

Other study objectives

Time to normalize FT4 in neonates

Time to normalize TSH in neonates

Awards & Highlights

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: TreatmentExperimental Treatment1 Intervention

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.

Group II: ControlActive Control1 Intervention

Crushed levothyroxine sodium tablets

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The primary treatment for Congenital Hypothyroidism is synthetic thyroxine (T4) replacement, such as Tirosint®-SOL. This treatment works by providing the body with the hormone thyroxine, which is deficient in patients with Congenital Hypothyroidism. Thyroxine is crucial for normal growth, brain development, and metabolism. By supplementing this hormone, the treatment helps normalize thyroid function, ensuring proper physical and cognitive development in affected infants. Accurate and consistent dosing, as seen with liquid formulations like Tirosint®-SOL, is particularly important to maintain stable thyroid hormone levels and optimize developmental outcomes.

Delayed Diagnosis of Congenital Hypothyroidism in a Child with Trisomy 21 and Biotinidase Deficiency and Successful Use of Levothyroxine Sodium Oral Solution.