Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD

A PHASE 3, RANDOMIZED, MULTICENTER, OPEN-LABEL, CROSSOVER STUDY ASSESSING SUBJECT PERCEPTION OF TREATMENT BURDEN WITH USE OF WEEKLY GROWTH HORMONE (SOMATROGON) VERSUS DAILY GROWTH HORMONE (GENOTROPIN (REGISTERED)) INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY

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.

Subjects will be randomized to one of two sequences, either 12 weeks of continued treatment with daily Genotropin followed by 12 weeks of treatment with weekly somatrogon, or 12 weeks of treatment with weekly somatrogon followed by 12 weeks of treatment with daily Genotropin. Subjects will have study visits at Baseline, Weeks 6, 12, 18, and 24. Subjects will also be followed up by phone 8 to 12 days after each treatment period begins (Week 1 and Week 13). Subjects and caregivers (as a Dyad) will complete questionnaires assessing treatment burden at baseline and at the end of each 12 week treatment period. All subjects/caregivers will receive a follow up phone call at Week 28.

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).