Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD
Condition: Growth Hormone Deficiency
MeSH terms: Dwarfism, Pituitary
Intervention: Genotropin; somatrogon
Intervention description: Genotropin (dose [mg] at time of enrollment) given subcutaneously once daily; 0.66 mg/kg/week given subcutaneously once weekly
Phase: Phase 3
Study type: Interventional
Primary purpose: Other
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Enrollment: 87.0
Sex: All
Minimum age: 3 Years
Maximum age: 17 Years
Healthy volunteers: No

Summary:

      This is an open label randomized 24 week crossover trial assessing the treatment burden of a
      weekly growth hormone injection regimen (somatrogon) compared to a daily growth hormone
      injection regimen (Genotropin). Approximately 90 children with growth hormone deficiency who
      have been stable on treatment with daily Genotropin will be enrolled.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Children aged 3 years old and <18 years with either isolated GHD, or GH insufficiency.

          2. Currently on treatment with either Genotropin Pen®, Genotropin GoQuick Pen®,
             HumatroPen® (United States of America [USA] only), or Omnitrope® Pen (USA only) ≥3
             months and have been compliant on a stable dose (±10%) for at least 3 months prior to
             screening.

          3. IGF I SDS < 2.

          4. Subjects on hormonal replacement therapy for other hypothalamic pituitary axis (HPA)
             hormonal deficiencies and/or diabetes insipidus must be on an optimized and stable
             treatment regimen, as determined by the Investigator, for at least 3 months prior to
             screening.

        Exclusion Criteria

          1. History of leukemia, lymphoma, sarcoma or any other cancer.

          2. History of radiation therapy or chemotherapy.

          3. Children with psychosocial dwarfism.

          4. Children born small for gestational age (SGA) - birth weight and/or birth length < 2
             SDS for gestational age.

          5. Other causes of short stature such as uncontrolled primary hypothyroidism and rickets.

          6. Chromosomal abnormalities including Turner's syndrome, Laron syndrome, Noonan
             syndrome, Prader Willi syndrome, Russell Silver syndrome, short stature homeobox
             (SHOX) mutations/deletions or skeletal dysplasias.

          7. Treatment with regularly scheduled daily or weekly injectable medications other than
             Genotropin® Pen, Genotropin GoQuick®, HumatroPen® (USA only), or Omnitrope® Pen (USA
             only).

          8. Diabetes Mellitus.

          9. Current treatment with Genotropin MiniQuick.

         10. History of any exposure to a long acting hGH preparation.

         11. Known or suspected human immunodeficiency virus (HIV) positive patient, or patient
             with advanced diseases such as acquired immunodeficiency syndrome (AIDS) or
             tuberculosis.

         12. Drug, substance, or alcohol abuse.

         13. Known hypersensitivity to the components of the medication.

         14. Pregnant female subjects; breastfeeding female subjects; fertile male subjects and
             female subjects of childbearing potential who are unwilling or unable to use a highly
             effective method of contraception as outlined in this protocol for the duration of the
             study and for at least 28 days after the last dose of investigational product.

         15. Other acute or chronic medical or psychiatric condition including recent (within the
             past year) or active suicidal ideation or behavior or laboratory abnormality that may
             increase the risk associated with study participation or investigational product
             administration or may interfere with the interpretation of study results and, in the
             judgment of the investigator, would make the subject inappropriate for entry into this
             study.

         16. Investigator site staff members directly involved in the conduct of the study and
             their family members, site staff members otherwise supervised by the investigator, or
             subjects who are Pfizer employees, including their family members, directly involved
             in the conduct of the study.

         17. Participation in other studies involving investigational drug(s) within 30 days prior
             to study entry and/or during study participation.

         18. Patient and/or the parent/legal guardian are likely to be non-compliant with respect
             to study conduct.

         19. Subject and/or the parent/legal guardian are unable to understand written and/or
             verbal instructions on the proper use of growth hormone injection devices.

         20. Children with closed epiphyses (this determination can be based on available existing
             clinical data).