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Transitioned to multi site study with sponsor
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Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption. For an age group unable to swallow tablets whole by mouth, tablets must be crushed and suspended in water, breast milk or formula for administration in order to treat children with hypothyroidism. For this age group, ease of administration may have a significant impact on compliance and ability to remain euthyroid. We propose that Tirosint-SOL® will be more favorably received due to ease of administration, improved tolerability and palatability, therefore leading to improved adherence when compared to L-T4 tablets.
Hypothyroidism is a common medical disorder in children with Trisomy 21 (T21). Treatment with oral tablet levothyroxine (L-T4) is primarily used to treat this condition. However, children with T21 have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption. For an age group unable to swallow tablets whole by mouth, tablets must be crushed and suspended in water, breast milk or formula for administration in order to treat children with hypothyroidism. For this age group, ease of administration may have a significant impact on compliance and ability to remain euthyroid. Until recently, there was no liquid preparation of L-T4 in the United States for use in children. Tirosint-SOL® (liquid stable L-T4) is now FDA-approved for use in children. The Investigator proposes that Tirosint-SOL® will be more favorably received due to ease of administration, improved tolerability and palatability, therefore leading to improved adherence when compared to L-T4 tablets.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Liquid Stable Levothyroxine (l-T4) TirosintSOL first, then Oral Tablet Levothyroxine (L-T4) | Other | Participants start on Liquid Tirosint®-SOL for 8 weeks using continued same daily dose of LT4. Dose adjustments are allowed as needed, based on laboratory results and clinical response. Then Participants will crossover to conventional therapy of LT4 tablets for 8 weeks. Participants total participation will be 16 weeks total. |
|
| Oral Tablet Levothyroxine (L-T4) first, then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL | Other | Participants continue conventional therapy of LT4 tablets for 8 weeks at current dose, after 8 weeks will crossover to receive Liquid Stable Levothyroxine (L-T4) Tirosint-SOL for 8 weeks. Participants total participation will be 16 weeks total. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Liquid stable levothyroxine (L-T4) Tirosint-SOL | Drug | Investigation of tolerability, palatability, and adherence of liquid levothyroxine (L-T4) for 8 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Tolerability as Assessed by the CareCAT Tool | Utilize medication tolerability questionnaire, Care Children's Acceptance Tool (CAT), to understand parental perceptions of liquid stable L-T4. | 4 months |
| Measure | Description | Time Frame |
|---|---|---|
| Adherence to Taking Study Medication | Evaluate differences in compliance with liquid stable L-T4 compared to L-T4 tablet. Evaluated in percentage of days compliant. | 4 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Liquid Stable Levothyroxine (l-T4) TirosintSOL First, Then Oral Tablet Levothyroxine (L-T4) | Participants start on Liquid Tirosint®-SOL for 8 weeks using continued same daily dose of LT4. Dose adjustments are allowed as needed, based on laboratory results and clinical response. Then Participants will crossover to conventional therapy of LT4 tablets for 8 weeks. Participants total participation will be 16 weeks total. |
| FG001 | Oral Tablet Levothyroxine (L-T4) First, Then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL | Participants continue conventional therapy of LT4 tablets for 8 weeks at current dose, after 8 weeks will crossover to receive Liquid Stable Levothyroxine (L-T4) Tirosint-SOL for 8 weeks. Participants total participation will be 16 weeks total. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
6 subjects were enrolled and completed the study, however, due to concerns with data collection and integrity, we elected to not use those subjects data moving forward. No further subjects were recruited prior to the study closing
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| ID | Title | Description |
|---|---|---|
| BG000 | Liquid Stable Levothyroxine (l-T4) TirosintSOL First, Then Oral Tablet Levothyroxine (L-T4) | Participants start on Liquid Tirosint®-SOL for 8 weeks using continued same daily dose of LT4. Dose adjustments are allowed as needed, based on laboratory results and clinical response. Then Participants will crossover to conventional therapy of LT4 tablets for 8 weeks. Participants total participation will be 16 weeks total. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Tolerability as Assessed by the CareCAT Tool | Utilize medication tolerability questionnaire, Care Children's Acceptance Tool (CAT), to understand parental perceptions of liquid stable L-T4. | 6 subjects were enrolled and 5 completed the study, however, due to concerns with data collection and integrity, the overseeing Institutional Review Board determined that results for these subjects should not be analyzed. No further subjects were recruited prior to the study closing. | Posted | 4 months |
|
112 Days, Duration of Study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Liquid Stable Levothyroxine (l-T4) TirosintSOL First, Then Oral Tablet Levothyroxine (L-T4) | Participants start on Liquid Tirosint®-SOL for 8 weeks using continued same daily dose of LT4. Dose adjustments are allowed as needed, based on laboratory results and clinical response. Then Participants will crossover to conventional therapy of LT4 tablets for 8 weeks. Participants total participation will be 16 weeks total. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Max Feldt | Children's Mercy | 816-960-8892 | mmfeldt@cmh.edu |
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 22, 2022 | Dec 1, 2022 | Prot_002.pdf |
| ICF | No | No | Yes | Informed Consent Form | Apr 4, 2022 | Oct 17, 2022 | ICF_001.pdf |
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| ID | Term |
|---|---|
| D007037 | Hypothyroidism |
| D004314 | Down Syndrome |
| ID | Term |
|---|---|
| D013959 | Thyroid Diseases |
| D004700 | Endocrine System Diseases |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
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| ID | Term |
|---|---|
| D013974 | Thyroxine |
| ID | Term |
|---|---|
| D013963 | Thyroid Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D024322 | Amino Acids, Aromatic |
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Phase 4, single-center, pilot, randomized, open-label, cross-over study
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| Oral tablet levothyroxine (L-T4) | Drug | Investigation of tolerability, palatability, and adherence of maintained oral tablet levothyroxine (L-T4) for 8 weeks |
|
| BG001 | Oral Tablet Levothyroxine (L-T4) First, Then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL | Participants continue conventional therapy of LT4 tablets for 8 weeks at current dose, after 8 weeks will crossover to receive Liquid Stable Levothyroxine (L-T4) Tirosint-SOL for 8 weeks. Participants total participation will be 16 weeks total. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
Participants continue conventional therapy of LT4 tablets for 8 weeks at current dose, after 8 weeks will crossover to receive the treatment arm (Tirosint-SOL) for 8 weeks. Participants will be treated for 16 weeks total. |
|
| Secondary | Adherence to Taking Study Medication | Evaluate differences in compliance with liquid stable L-T4 compared to L-T4 tablet. Evaluated in percentage of days compliant. | 6 subjects were enrolled and 5 completed the study, however, due to concerns with data collection and integrity, the overseeing Institutional Review Board determined that results for these subjects should not be analyzed. No further subjects were recruited prior to the study closing. | Posted | 4 months |
|
|
| 0 |
| 2 |
| 0 |
| 2 |
| 0 |
| 2 |
| EG001 | Oral Tablet Levothyroxine (L-T4) First, Then Liquid Stable Levothyroxine (L-T4) Tirosint-SOL | Participants continue conventional therapy of LT4 tablets for 8 weeks at current dose, after 8 weeks will crossover to receive Liquid Stable Levothyroxine (L-T4) Tirosint-SOL for 8 weeks. Participants total participation will be 16 weeks total. | 0 | 4 | 0 | 4 | 0 | 4 |
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| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |