| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Growth Hormone Deficiency in Children |
|
| E.1.1.1 | Medical condition in easily understood language |
| Treatment of children with growth failure due to growth hormone deficiency |
|
| E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
| MedDRA Classification |
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 20.0 |
| E.1.2 | Level | PT |
| E.1.2 | Classification code | 10056438 |
| E.1.2 | Term | Growth hormone deficiency |
| E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
|
| E.1.3 | Condition being studied is a rare disease | Yes |
| E.2 Objective of the trial |
| E.2.1 | Main objective of the trial |
| To evaluate the treatment burden of a weekly somatrogon injection schedule and a daily Genotropin® injection schedule. |
|
| E.2.2 | Secondary objectives of the trial |
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. |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
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 |
|
| E.4 | Principal 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). |
|
| E.5 End points |
| E.5.1 | 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 |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
| At baseline and after each treatment schedule experience |
|
| E.5.2 | 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). |
|
| E.5.2.1 | 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 |
|
| E.6 and E.7 Scope of the trial |
| E.6 | Scope of the trial |
| E.6.1 | Diagnosis | No |
| E.6.2 | Prophylaxis | No |
| E.6.3 | Therapy | Yes |
| E.6.4 | Safety | Yes |
| E.6.5 | Efficacy | No |
| E.6.6 | Pharmacokinetic | Yes |
| E.6.7 | Pharmacodynamic | No |
| E.6.8 | Bioequivalence | No |
| E.6.9 | Dose response | No |
| E.6.10 | Pharmacogenetic | No |
| E.6.11 | Pharmacogenomic | No |
| E.6.12 | Pharmacoeconomic | No |
| E.6.13 | Others | Yes |
| E.6.13.1 | Other scope of the trial description |
Treatment Burden
Treatment Preference
Immunogenicity |
|
| E.7 | Trial type and phase |
| E.7.1 | Human pharmacology (Phase I) | No |
| E.7.1.1 | First administration to humans | No |
| E.7.1.2 | Bioequivalence study | No |
| E.7.1.3 | Other | No |
| E.7.1.3.1 | Other trial type description | |
| E.7.2 | Therapeutic exploratory (Phase II) | No |
| E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
| E.7.4 | Therapeutic use (Phase IV) | No |
| E.8 Design of the trial |
| E.8.1 | Controlled | Yes |
| E.8.1.1 | Randomised | Yes |
| E.8.1.2 | Open | Yes |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | No |
| E.8.1.5 | Parallel group | No |
| E.8.1.6 | Cross over | Yes |
| E.8.1.7 | Other | No |
| E.8.2 | Comparator of controlled trial |
| E.8.2.1 | Other medicinal product(s) | Yes |
| E.8.2.2 | Placebo | No |
| E.8.2.3 | Other | No |
| E.8.2.4 | Number of treatment arms in the trial | 2 |
| E.8.3 |
The trial involves single site in the Member State concerned
| Information not present in EudraCT |
| E.8.4 | The trial involves multiple sites in the Member State concerned | Information not present in EudraCT |
| E.8.5 | The trial involves multiple Member States | Yes |
| E.8.5.1 | Number of sites anticipated in the EEA | 10 |
| E.8.6 Trial involving sites outside the EEA |
| E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
| E.8.6.2 | Trial being conducted completely outside of the EEA | No |
| E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
| Bulgaria |
| Czech Republic |
| Slovakia |
| United Kingdom |
| United States |
|
| E.8.7 | Trial has a data monitoring committee | No |
| E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
| End of trial in a Member State of the European Union is defined as the time at which it is deemed that a sufficient number of subjects have been recruited and completed the study as stated in the regulatory application and ethics application in the Member State. Poor recruitment (recruiting less than the anticipated number in the CTA) by a Member State is not a reason for premature termination but is considered a normal conclusion to the study in that Member State. |
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | |
| E.8.9.1 | In the Member State concerned months | 11 |
| E.8.9.1 | In the Member State concerned days | 19 |
| E.8.9.2 | In all countries concerned by the trial years | 1 |
| E.8.9.2 | In all countries concerned by the trial months | 1 |
| E.8.9.2 | In all countries concerned by the trial days | 1 |
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