A Phase 2, Safety and Dose-Finding Study in Treatment-Naive, Pre-Pubertal, Growth Hormone-Deficient Children

Study Overview
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Brief Summary
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The purpose of this study is to support selection of an appropriate safe and efficacious dose for study in further development.

Official Title
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A Phase 2, Randomized, Open-Label, Safety and Dose-Finding Study Comparing 3 Different Doses of Weekly TV-1106 and Daily Recombinant Human Growth Hormone Therapy in Treatment-Naive, Pre-Pubertal, Growth Hormone-Deficient Children

Conditions
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Growth Hormone-Deficiency

Intervention / Treatment
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* Drug: TV-1106
* Drug: somatropin


Participation Criteria
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Eligibility Criteria
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Criteria for Inclusion: Pre-pubertal boys ≥ 3 years to ≤ 11 years and pre-pubertal girls ≥ 3 years to ≤ 10 years growth hormone (GH) insufficiency Diagnosis criteria consistent with growth hormone research society consensus guidelines Patients with a previously treated pituitary tumor must have no tumor progression for at least the past year Physician determined rate of change in height less than 2 standard deviations per age group. Written Informed Consent Parent or legal guardian who is capable and willing to administer the study drug. Other criteria apply, please contact the investigator for more information Criteria for Exclusion: Any clinically significant medical condition as determined by the investigator, that is likely to affect growth Contraindications to rhGH treatment; History of or currently active malignancy, including pituitary tumors; Bone age, greater than chronological age or greater than 9 for girls or greater than 10 for boys within 3 months of screening. Patients with known diagnosis of diabetes or pre-diabetes Growth altering medications Allergies to the study medication components; Participation in another investigational study within 30 days of screening Any medical condition as judged by the investigator to interfere with patient participation or the objectives of the study Other criteria apply, please contact the investigator for more information

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: TV-1106 0.554 mg<br> | Drug: TV-1106<br>* TV-1106 0.554mg, TV-1106 0.924 mg, TV-1106 1.20 mg<br>* Other names: Human Growth Hormone Therapy;|
| Experimental: TV-1106 0.924 mg/kg<br> | Drug: TV-1106<br>* TV-1106 0.554mg, TV-1106 0.924 mg, TV-1106 1.20 mg<br>* Other names: Human Growth Hormone Therapy;|
| Experimental: TV-1106 1.20 mg/kg<br> | Drug: TV-1106<br>* TV-1106 0.554mg, TV-1106 0.924 mg, TV-1106 1.20 mg<br>* Other names: Human Growth Hormone Therapy;|
| Active Comparator: somatropin 0.033 mg/kg/day<br>Dosages may be adjusted according to findings and as necessary | Drug: somatropin<br>* Dose may be adjusted as required<br>* Other names: Human Growth Hormone Therapy;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Height velocity (HV) |  | Month 6 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Height velocity standard deviation score (HV-SDS) |  | Months 6 and 12 | 
| Height standard deviation score (H-SDS) |  | Months 6 and 12 | 
| Number of participants with adverse events |  | up to 24 months | 
| Height velocity (HV) |  | Month 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Growth Hormone-Deficient, recombinant human growth hormone