Tirosint-SOL for Congenital Hypothyroidism

No longer 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new liquid form of the thyroid hormone treatment, Tirosint-SOL, for babies with congenital hypothyroidism (CH), a condition where the thyroid gland doesn't produce enough hormones. The study compares Tirosint-SOL to the usual crushed tablet form of the same hormone. It includes babies diagnosed with CH who are either just starting treatment or have been on similar therapy for at least three weeks. This trial may suit infants diagnosed with CH who are currently on or need thyroid hormone treatment. As a Phase 4 trial, this research involves an FDA-approved treatment and aims to understand how it can benefit more patients.

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 is the safety track record for these treatments?

Research has shown that Tirosint-SOL, a liquid form of levothyroxine sodium, is safe in various situations, including during pregnancy. No reports of increased major birth defects have emerged, indicating it is well-tolerated by vulnerable groups. Additionally, studies on children taking Tirosint-SOL demonstrate it is generally well-tolerated. This safety information reassures those considering joining a trial with Tirosint-SOL, suggesting the treatment is safe for people. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Unlike traditional levothyroxine sodium tablets for congenital hypothyroidism, Tirosint-SOL is an oral solution, which can be a game-changer, especially for infants and young children who may have difficulty swallowing pills. This liquid formulation ensures more precise dosing and potentially better absorption, which can lead to more stable thyroid hormone levels. Researchers are excited about Tirosint-SOL because it offers flexibility in dosing and administration, catering to individual patient needs more effectively than crushed tablets.

What evidence suggests that Tirosint-SOL might be an effective treatment for congenital hypothyroidism?

Research has shown that Tirosint-SOL, a liquid form of levothyroxine, effectively treats congenital hypothyroidism (CH) in infants. This trial will compare Tirosint-SOL with crushed levothyroxine sodium tablets. Tirosint-SOL improves thyroid function, crucial for normal growth and development. It has been proven safe and effective, providing a reliable treatment for infants with CH. Liquid forms like Tirosint-SOL are often easier for infants to take than crushed tablets. Experts agree that levothyroxine solutions benefit CH treatment by supporting healthy development in children. ^{5 6 7 8 9}

Who Is on the Research Team?

Giuseppe Mautone

Principal Investigator

IBSA Head of R&D Scientific Affairs

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11430788/

Comparison between Liquid and Tablet Formulations in ...

Comparison between liquid and tablet formulations in the treatment of congenital hypothyroidism up to 3 years of age: the first Italian study.

2.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/pediatric/92_206977%20levothyroxine%20clinical%20prea.pdf

Clinical Review Tirosint-SOL - accessdata.fda.gov

Congenital hypothyroidism causes growth failure ... Tmax for baseline-corrected levothyroxine for levothyroxine sodium oral solution with.

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11856982/

Efficacy of levothyroxine on growth and development in ...

L-T4 is an effective drug for the treatment with CH in children, which significantly promote their growth and development while improving thyroid function.

4.

tirosintsol.com

tirosintsol.com/tirosint-sol-in-the-news/

Tirosint®-SOL in the news

TIROSINT is not for use in people with temporary hypothyroidism caused by thyroiditis, or inflammation of the thyroid gland. Important Safety Information.

5.

biospace.com

biospace.com/new-guidelines-cite-oral-levothyroxine-solution-as-an-option-for-treating-congenital-hypothyroidism-ch

New Guidelines Cite Oral Levothyroxine Solution as an ...

The availability of a levothyroxine oral solutions like Tirosint®-SOL, which has been demonstrated to be effective and safe for infants with CH, ...

6.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2016/206977s000lbl.pdf

TIROSINT-SOL (levothyroxine sodium) oral solution

Experience with levothyroxine use in pregnant women, including data from post-marketing studies, have not reported increased rates of major birth defects or ...

7.

fda.gov

fda.gov/media/181669/download

Tirosint Pediatric Postmarketing Safety review

This review evaluates FDA Adverse Event Reporting System (FAERS) reports for Tirosint-SOL. (levothyroxine sodium) oral solution in pediatric ...

8.

tirosintsol.com

tirosintsol.com/

Tirosint-SOL Liquid Levothyroxine Solution - Hypothyroidism ...

TIROSINT is not for use in people with temporary hypothyroidism caused by thyroiditis, or inflammation of the thyroid gland. Important Safety Information.

9.

tirosint.com

tirosint.com/hcp/resources/

Resources | TIROSINT® (levothyroxine sodium) Capsules

Not indicated for treatment of transient hypothyroidism during the recovery phase of subacute thyroiditis. IMPORTANT SAFETY INFORMATION. WARNING: NOT FOR THE ...