STUDY ASSESSING SUBJECT PERCEPTION OF TREATMENT BURDEN WITH USE OF WEEKLY GROWTH HORMONE (SOMATROGON) VERSUS DAILY GROWTH HORMONE (GENOTROPIN®) INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY
- Conditions
- Growth Hormone Deficiency in ChildrenMedDRA version: 20.0Level: PTClassification code 10056438Term: Growth hormone deficiencySystem Organ Class: 10014698 - Endocrine disordersTherapeutic area: Diseases [C] - Hormonal diseases [C19]
- Registration Number
- EUCTR2018-000918-38-CZ
- Lead Sponsor
- Pfizer Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 90
1. Children aged =3 years old and <18 years (17 years and 364 days) on the date of ICF signature with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiencies.
2. Currently on treatment with either Genotropin Pen® or Genotropin GoQuick Pen® =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.
5. Women of childbearing potential and fertile men must agree to use a highly effective method of contraception as outlined in this protocol (see Section 4.4.1) during the study until at least 28 days after the last dose of investigational product. Fertile men must agree to a barrier contraceptive (condom). Vasectomy older than 6 months is also acceptable.
a. Female subjects of non-childbearing potential must meet at least 1 of the following criteria:
i. Premenarchal;
ii. Have undergone a documented hysterectomy and/or bilateral oophorectomy;1
iii. Have medically confirmed ovarian failure.
b. Male subjects Tanner stage 3 and above are to be considered fertile.
All other female subjects (including female subjects with tubal ligations) are considered to be of childbearing potential.
6. Evidence of a personally signed and dated informed consent document (and written assent where applicable based on age and country regulation) indicating that the subject or a legally acceptable representative/parent(s)/legal guardian has been informed of all pertinent aspects of the study.
7. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. Subjects and/or caregiver must express the ability to understand, read, and write in the language native to the country in which the study is being conducted. In the U.S., subjects and/or caregiver must express the ability to understand, read, and write English
Are the trial subjects under 18? yes
Number of subjects for this age range: 90
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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 or Genotropin GoQuick®
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).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method