Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

Study Overview
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Brief Summary
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Protocol CP-4-004-EXT is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.

Official Title
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Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily R-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children

Conditions
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Growth Hormone Deficiency (GHD)

Intervention / Treatment
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* Drug: MOD-4023


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study. Exclusion Criteria: Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast). History of radiation therapy or chemotherapy. Malnourished children defined as: Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory; Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory; BMI < -2 Standard Deviation for age and sex; Children with psychosocial dwarfism. Children born small for gestational age (SGA - birth weight and/or birth length < -2 SD for gestational age). Presence of anti-hGH antibodies at screening. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc. Patients with diabetes mellitus. Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or 6.1 mmol/l) after repeated blood analysis. Chromosomal abnormalities and medical syndromes (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, short stature homeobox-containing gene (SHOX) mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia. Closed epiphyses. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies (thyroxine, hydrocortisone, desmopressin (DDAVP)) Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year. Major medical conditions and/or presence of contraindication to r-hGH treatment. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis. Drug, substance, or alcohol abuse. Known hypersensitivity to the components of study medication. Other causes of short stature such as coeliac disease, hypothyroidism and rickets. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct. Participation in any other trial of an investigational agent within 30 days prior to Screening.

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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MOD-4023<br>Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN). | Drug: MOD-4023<br>* Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annual Height Velocity |  | 8 years | 
| Delta height SDS every 12 months |  | 8 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Absolute IGF-I levels on day 4 after MOD-4023 dosing |  | 8 years | 
| IGF-I SDS on day 4 after MOD-4023 dosing |  | 8 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Growth hormone deficiency (GHD), r-hGH, Long-acting