Saizen® Solution for Injection Adult Growth Hormone Deficiency (GHD) Immunogenicity Study

Study Overview
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Brief Summary
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To assess the immunogenicity of Saizen® solution for injection in adult subjects with documented Growth Hormone Deficiency (GHD).

Official Title
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A Phase IIIb, Open-label, Single-arm, Multicenter Study to Assess the Immunogenicity of the Recombinant-Human Growth Hormone (r-hGH) Liquid Multidose Formulation (Saizen® Solution for Injection) When Administered to Male and Female Adults With Documented Growth Hormone Deficiency (GHD)

Conditions
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Growth Hormone Deficiency (GHD)

Intervention / Treatment
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* Drug: Saizen®


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult male and female subjects, 18-60 years of age, inclusive, at the time the informed consent is signed Subjects who have confirmed adult GHD Subjects who are growth hormone (GH) treatment-naive or had received Saizen® (freeze-dried formulation) for pediatric GHD (PGHD), or are currently receiving Saizen® freeze-dried formulation for adult GHD (AGHD) Subjects who have binding antibody-negative (BAbs-) at screening Subjects who have no evidence of concomitant disease, intercurrent illness, or resultant therapy that would interfere with subject compliance, the evaluation of study results, or compromise the safety of the subject Female subjects of childbearing potential who have a negative serum pregnancy test at the screening visit (and at each scheduled visit during the study) Subjects who are willing to comply with the procedures of the study Subjects who are willing to sign an Independent Ethics Committee/Institutional Review Board approved informed consent form Other protocol-defined inclusion criteria may apply Exclusion Criteria: Subjects who are currently receiving or have previously received treatment for adult GHD or any other indication, including PGHD, with a commercial GH product other than Saizen® freeze-dried formulation Subjects who had a chronic underlying disease within 6 months prior to screening or concomitant medication(s) that in the opinion of the investigator would exclude the subject from the trial Subjects who have significant renal impairment Subjects who have diabetes mellitus Subjects who are immunosuppressed Subjects who have a current malignancy or a history of any malignancy (excluding fully-treated basal cell carcinoma) Subjects who have participated in another study and received an investigational drug within 30 days prior to screening visit Subjects who have clinically significant abnormal laboratory value(s) Subjects who have known hypersensitivity or allergy to exogenous human GH or any of the excipients or phenol, the bacteriostatic agent in the Saizen® solution for injection Other protocol-defined exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 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: Saizen®<br> | Drug: Saizen®<br>* Single dose of Saizen® (recombinant human growth hormone, r-hGH) solution for injection will be administered subcutaneously for 26 weeks. Dosage regimen will be in accordance with marketed formulation of Saizen® (freeze-dried formulation), based on locally approved product labeling.<br>* Other names: Somatropin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Who Developed Positive Binding Antibodies (BAbs+) to Saizen® | Binding antibodies (BAbs) are all antibodies which are capable of binding to the investigational drug molecule (Saizen®), irrespective of their binding site. | Baseline up to Week 26 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Who Developed Positive Neutralizing Antibodies (NAbs+) to Saizen® | Neutralizing antibodies (NAbs) are defined as a subgroup of BAbs which bind to the active sites of the investigational drug molecule (Saizen®) and therefore neutralize its potency. | Baseline up to Week 26 | 
| Insulin-like Growth Factor-I (IGF-1) Levels |  | Baseline, Week 2, Week 4, Week 8, Week 13, Week 18, Week 26 | 
| Insulin-like Growth Factor-I Standard Deviation Score (IGF-1 SDS) | Insulin-like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) was provided by the central laboratory; its calculation is based on the actual value of IGF-1 minus mean reference value of IGF-1 divided by reference standard deviation of IGF-1. | Baseline, Week 2, Week 4, Week 8, Week 13, Week 18, Week 26 | 
| Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) Levels |  | Baseline, Week 2, Week 4, Week 8, Week 13, Week 18, Week 26 | 
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Adverse events (AEs): Any untoward medical occurrence in the form of signs, clinically significant abnormalities in laboratory findings, diseases, symptoms, or worsening of complications. TEAEs: AEs occurring after the first administration of Saizen® solution for injection (on Day 1) up to the scheduled routine post treatment follow-up visit (4 weeks [28 days] after the final administration of Saizen® solution for injection). | Day 1 up to 28 days after last dose of study treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Adult Growth Hormone Deficiency (AGHD), immunogenicity, growth hormone, antibodies, GH biomarkers, IGF-I