Clinical Trial: An Open-label Phase 4 Study to Explore Immunogenicity of the Liquid Formulation of Saizen® in Subjects With Adult Growth Hormone Deficiency (AGHD)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Open-label, Single-arm, Phase IV, Multicenter Trial to Explore the Immunogenicity of the Liquid Formulation of Saizen® in Subjects With Adult Growth Hormone Deficiency

Brief Summary: This is an open-label, single-arm, multicenter, Phase 4 study to explore the immunogenicity of the liquid formulation of Saizen® in subjects with Adult Growth Hormone Deficiency (AGHD), who are growth hormone (GH) treatment-naïve or who had prior GH treatment for GHD which was stopped at least 1 month prior to Screening and have no contraindication to the use of GH.

Detailed Summary:
Sponsor: Merck KGaA

Current Primary Outcome: Percentage of Subjects Developing Binding Antibodies (BAbs) to Saizen® [ Time Frame: Baseline up to Week 39 ]

Original Primary Outcome: Percentage of Subjects Developing Binding Antibodies (BAbs) to Saizen® [ Time Frame: Baseline up to Week 39 ]

Current Secondary Outcome:

• Percentage of Subjects With Binding Antibodies (BAbs) Who Became Positive for Neutralizing Antibodies (NAbs) [ Time Frame: Baseline up to Week 39 ]
  Percentage of subjects with BAbs who become positive for NAbs = (Number of NAb positive subjects / Number of BAbs positive subjects) x 100
• Insulin-like Growth Factor-I (IGF-I) Levels [ Time Frame: Baseline, Week 2, 8, 16, 29, 39 and 41 ]
  Growth Hormone (GH) biomarker levels were summarized by GH treatment status at study entry (that is subjects were classified as GH treatment-naïve subjects or subjects with prior GH treatment for adult growth hormone deficiency [AGHD]).
• Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) Levels [ Time Frame: Baseline, Week 2, 8, 16, 29, 39 and 41 ]
  Growth Hormone (GH) biomarker levels were summarized by GH treatment status at study entry (that is subjects were classified as GH treatment-naïve subjects or subjects with prior GH treatment for adult growth hormone deficiency [AGHD])
• Insulin-like Growth Factor-I Standard Deviation Score (IGF-I SDS) [ Time Frame: Baseline, Week 2, 8, 16, 29, 39 and 41 ]
  Insulin-like Growth Factor-1 SDS was calculated based on the actual value of IGF-1 minus reference value of IGF-1 divided by reference standard deviation of IGF-1. SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a subject's value was relative to the mean of the reference population. The scores were centered around zero. Negative score indicated that the IGF-I value was lower compared to the reference population.
• Treatment Adherence Rate as Documented Using EasypodTM Connect [ Time Frame: Week 2, 8, 16, 29 and 39 ]
  Treatment adherence rate was measured by: (total dose received divided by total dose prescribed) multiplied by 100. Saizen solution for injection was administered using the easypod device and treatment adherence information was obtained from the device using the easypod connect software.

Original Secondary Outcome:

• Percentage of subjects with BAbs who become positive for Neutralizing Antibody (NAbs) [ Time Frame: Baseline up to 2 weeks after last dose of study treatment ]
• Insulin-like Growth Factor-I (IGF-I) Levels [ Time Frame: Baseline, and Week 2, 8, 16, 29, 39 and 41 ]
• Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) Levels [ Time Frame: Baseline, and Week 2, 8, 16, 29, 39 and 41 ]
• Insulin-like Growth Factor-I Standard Deviation Score (IGF-I SDS) levels [ Time Frame: Baseline, and Week 2, 8, 16, 29, 39 and 41 ]
• Treatment adherence rate for each subject as documented using EasypodTM connect [ Time Frame: Week 2, 8, 16, 29 and 39 ]
• Number of subjects with treatment-emergent adverse events [ Time Frame: Week 2, 8, 16, 29 and 39 ]

Dates:
Date Received: March 5, 2013
Date Started: June 2013
Date Completion:
Last Updated: February 15, 2017
Last Verified: February 2017