Tirosint-SOL for Congenital Hypothyroidism

Recruiting at 11 trial locations

Overseen By Claudia Scarsi Perler

Age: < 18

Sex: Any

Trial Phase: Phase 4

Sponsor: IBSA Institut Biochimique SA

Must be taking: Levothyroxine

Must not be taking: Anticonvulsants, Liothyronine, Glucocorticoids

Disqualifiers: Preterm birth, Low birth weight, NICU, Gastrointestinal disease, Cardiac disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

Duration: 12 months

34 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking anticonvulsant medications, liothyronine, a combination of LT4 and liothyronine, thyroid extracts, or long-term systemic glucocorticoids.

What data supports the effectiveness of the drug Tirosint-SOL for treating congenital hypothyroidism?

Research shows that liquid L-thyroxine, like Tirosint-SOL, is effective in treating congenital hypothyroidism, especially in newborns, by normalizing thyroid hormone levels. It is particularly beneficial for patients with absorption issues and is easier to handle, making it a promising option for common therapy. ^{1 2 3 4 5}

Is Tirosint-SOL (Levothyroxine Sodium) generally safe for humans?

Levothyroxine Sodium, including Tirosint-SOL, is generally considered safe for human use as it is a commonly prescribed medication for thyroid hormone replacement. However, like any medication, it can have side effects, and its safety profile has been well-studied in various conditions, although specific safety data for congenital hypothyroidism is not detailed in the provided research. ^{6 7 8 9 10}

How is the drug Tirosint-SOL different from other treatments for congenital hypothyroidism?

Tirosint-SOL is a liquid form of levothyroxine (a hormone replacement) that is alcohol-free, making it easier to absorb, especially for patients with absorption issues. It offers better hormonal control compared to tablet forms and is convenient for those who need to take it with or without food. ^{1 2 11 12 13}

Research Team

Giuseppe Mautone

Principal Investigator

IBSA Head of R&D Scientific Affairs

Eligibility Criteria

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.

Inclusion Criteria

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

Provide and comply with the informed consent

I am a baby aged 9 months or younger.

Exclusion Criteria

My newborn needs or is in intensive care.

My newborn weighs less than 2.5 kg.

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Tirosint®-SOL or crushed levothyroxine sodium tablets based on randomization

12 months

6-7 in-clinic visits, 1-2 telemedicine visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 22 months

Treatment Details

Interventions

• Levothyroxine Sodium
• Tirosint®-SOL

Trial Overview 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.

Participant Groups

Group I: Treatment Experimental Treatment 1 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: Control Active Control 1 Intervention

Crushed levothyroxine sodium tablets

Who Is Running the Clinical Trial?

IBSA Institut Biochimique SA

Lead Sponsor

Trials

42

Recruited

10,300+

Cromsource

Industry Sponsor

Trials

18

Recruited

3,200+

Findings from Research

Liquid levothyroxine (LT4) formulations, particularly Tirosint SOL, are more effective than tablet forms for patients with malabsorption issues, leading to better hormonal control and less frequent TSH measurements.

The liquid LT4 is safe and effective for various populations, including newborns and pregnant women, but caution is needed in neonates due to a higher risk of TSH suppression compared to tablet LT4.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Expert opinion on liquid L-thyroxine usage in hypothyroid patients and new liquid thyroxine formulation - Tirosint SOL [Opinia ekspertów dotycząca stosowania płynnej postaci lewotyroksyny oraz nowego preparatu Tirosint SOL u chorych na niedoczynność tarczycy]. Gietka-Czernel, M., Hubalewska-Dydejczyk, A., Kos-Kudła, B., et al.[2021]

References

1. Poland pubmed.ncbi.nlm.nih.gov

Expert opinion on liquid L-thyroxine usage in hypothyroid patients and new liquid thyroxine formulation - Tirosint SOL [Opinia ekspertów dotycząca stosowania płynnej postaci lewotyroksyny oraz nowego preparatu Tirosint SOL u chorych na niedoczynność tarczycy]. [2021]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Comparison Among Two Liquid Formulations of L-thyroxine in the Treatment of Congenital Hypothyroidism in the First Month of Life: A Pilot Study. [2022]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Congenital Hypothyroidism: 8-Year Experience Using 2 Newborn Screens in Alabama. [2020]

4. Germany pubmed.ncbi.nlm.nih.gov

Non-inferiority of liquid thyroxine in comparison to tablets formulation in the treatment of children with congenital hypothyroidism. [2022]

5. England pubmed.ncbi.nlm.nih.gov

A novel therapeutic paradigm to treat congenital hypothyroidism. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Evaluation of a panel of in vitro methods for assessing thyroid receptor β and transthyretin transporter disrupting activities. [2022]

7. Australia pubmed.ncbi.nlm.nih.gov

Effect of antithyroid drug on chick embryos during the last week of development: delayed hatching and decreased cerebellar acetylcholinesterase activity. [2013]

8. England pubmed.ncbi.nlm.nih.gov

Bexarotene-induced hypothyroidism: Characteristics and therapeutic strategies. [2020]

9. Netherlands pubmed.ncbi.nlm.nih.gov

Changes in thyroid hormone activity disrupt photomotor behavior of larval zebrafish. [2020]

10. England pubmed.ncbi.nlm.nih.gov

Towards a science-based testing strategy to identify maternal thyroid hormone imbalance and neurodevelopmental effects in the progeny - Part IV: the ECETOC and CLE Proposal for a Thyroid Function-Related Neurodevelopmental Toxicity Testing and Assessment Scheme (Thyroid-NDT-TAS). [2023]

11. Italy pubmed.ncbi.nlm.nih.gov

[Neonatal screening for congenital hypothyroidism in a suburban hospital: assessment of eleven years' experience]. [2013]

12. United States pubmed.ncbi.nlm.nih.gov

Diagnosis and treatment of hypothyroidism in children. [2013]

13. Egypt pubmed.ncbi.nlm.nih.gov

Serological Characteristics, Etiological Analysis, and Treatment Prognosis of Children with Congenital Hypothyroidism. [2023]