Growth Hormone Deficiency in Children MedDRA version: 20.0 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
Conditions
Interventions
Product Name: Somatrogon
Product Code: PF-06836922
Pharmaceutical Form: Solution for injection
INN or Proposed INN: somatrogon
CAS Number: 1663481-09-1
Concentration unit: mg/ml milligram(s)/millilitr
Sponsors
Pfizer Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Children aged =3 years old and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the treatment burden of a weekly somatrogon injection schedule and a daily Genotropin® injection schedule.;Secondary Objective: 1. To evaluate aspects of the treatment experience as determined by subject and caregiver self-assessments (dyadic approach) of weekly somatrogon therapy and daily Genotropin® therapy. 2. To use a patient global impression scale – impact on daily activities (PGIS-IDA) at baseline and at the end of each period (Week 12 and Week 24) to support the interpretation of scores from the Dyad Clinical Outcome Assessment (DCOA) 1 and DCOA 2 Questionnaires. 3. To confirm the psychometric properties and sensitivity of the DCOA Questionnaires in patients who have experienced both a weekly injection schedule and a daily injection schedule. 4. To describe the safety and tolerability of somatrogon.;Primary end point(s): Treatment burden assessed as the difference in mean Overall Life Interference total scores between the weekly injection schedule and daily injection schedule as assessed by the Patient Life Interference Questionnaire (as part of DCOA 1) completed by the Subject/Caregiver Dyad ;Timepoint(s) of evaluation of this end point: At baseline and after each treatment schedule experience | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Treatment experience assessed as the difference in mean scores between the weekly injection schedule experience and daily injection schedule experience in each of the following variables within DCOA 1 questionnaires : -Pen ease of use. -Ease of the injection schedule. -Convenience of the injection schedule. -Satisfaction with overall treatment experience. -Willingness to continue injection schedule. -Injection signs and symptoms (from the patient). -Assessment of Signs (from the Caregiver). -Caregiver Life Interference, including Family Life Interference. -Missed injections. 2. Proportion of subjects that select the weekly injection schedule compared to the daily injection schedule in each of the outcome domains below as assessed by the DCOA 2 Questionnaires: -Choice of injection pen. -Preferred injection schedule. -Convenience of injection schedule. -Easier to follow. -Ease of the injection schedule. -Patient life interference. -Caregiver Life Interference, including Family Life Interference. -Benefit relating to the injection schedule. -Intention to comply. 3. The Patient Global Impression 4. Safety -Frequency, severity, and relationship of adverse events to somatrogon. -Serious adverse events. -Discontinuations due to adverse events. -Frequency and severity of abnormal lab values. -Detection of anti-somatrogon antibodies (and neutralizing antibodies).;Timepoint(s) of evaluation of this end point: 1. at baseline and after subjects have experienced both treatment schedules 2. at Week 24 3. at baseline and at the end of each period (Week 12 and Week 24) 4. continuous | — |
Countries
Bulgaria, Czech Republic, Slovakia, United Kingdom, United States
Contacts
Public ContactClinical Trials.gov Call Centre
Pfizer Inc
Outcome results
None listed