A Prospective Non-Interventional Study Protocol With Primary Data Collection - Assessment Of The Long Term Treatment Outcomes Of Genotropin Treatment In Growth Hormone Deficiency (GHD) Patients

Study Overview
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Brief Summary
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The purpose of this study is to assess the long term treatment outcomes of Growth Hormone treatment in patients who are prescribed and treated with Genotropin. Also, plan to determine the relationships between clinical status, dosage schedule and response to Genotropin treatment. This study will also contribute to our knowledge of adult Growth Hormone Deficiency, including transition period in Childhood Onset Growth Hormone Deficiency and its treatment.

Detailed Description
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Patients within inclusion criteria are asked to participate in the study.

Official Title
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SWEGHO - A PROSPECTIVE NON INTERVENTIONAL STUDY PROTOCOL WITH PRIMARY DATA COLLECTION - ASSESSMENT OF THE LONG TERM TREATMENT OUTCOMES OF GENOTROPIN TREATMENT IN GHD PATIENTS IN SWEDEN

Conditions
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Growth Hormone Deficiency

Intervention / Treatment
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* Other: Non Interventional Study


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult patients of 18 years of age and above and fulfilling one of the three alternatives a-c below; Newly diagnosed with GHD according to the current medical standard. Diagnosed with GHD before 2013 and previously treated with Genotropin and followed in KIMS®. Transition patients diagnosed with CO-GHD before 2013. Prescribed Genotropin at the time of inclusion. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study. Exclusion Criteria: Patients who participate in any concurrent clinical interventional trial where a non-authorized or authorized study medication is used, during their participation in Swedish KIMS® Xtended. Concurrent studies which do not include any study interventional items (whether medications or devices) are allowed.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Adult Growth Hormone deficient Patients<br>Patients with GHD on Genotropin® replacement therapy. | Other: Non Interventional Study<br>* Non Interventional Study<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Classified According to Insulin-like Growth Factor (IGF-I) Assessments | IGF-I along with growth hormone helps promote normal bone and tissue growth and development. Categories for assessment for participant's post-baseline IGF-I values: (1) IGF-I LLN = if any of assessments of IGF-I post-baseline visit was lower than lower limit of normal (LLN); (2) IGF-I ULN = If any of assessments of IGF-I post-baseline visit was greater than upper level of normal (ULN); (3) IGF-I unknown = no IGF-I reported; (4) Within reference range = IGF-I levels within normal range. Following is normal reference range of IGF-I in nanogram per milliliter. 18 Years of age (Y): Male =162-541, Female =170-640; 19 Y: Male =138-442, Female =147-527; 20 Y: Male =122-384,Female =132-457; 21-25 Y=116-341; 26-30 Y=117-321; 31-35 Y=113-297; 36-40 Y=106-277; 41-45 Y =98-261; 46-50 Y=91-246; 51-55 Y=84-233; 56-60 Y=78-220; 61-65 Y=72-207; 66-70 Y=67-195; 71-75 Y=62-184; 76-80 Y=57-172; >80 Y=53-162. There was no differentiation for male and female in normal range of IGF-I after 20 years of age. | Up to 5 years (after baseline visit) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any untoward medical occurrence in a participant who received Genotropin without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A treatment emergent AE was defined as an event that emerged during the treatment period that was absent before treatment, or worsened during the treatment period relative to the pretreatment state. AEs included both serious and non-serious AEs. | Baseline up to 5 years | 
| Number of Treatment Related Adverse Events | Treatment-related AEs refer to AEs that have a causal relationship with the treatment or usage. If there was any relationship between AE and Genotropin treatment,that was judged by investigator. | Baseline up to 5 years | 
| Number of Adverse Events Leading to Withdrawal of Genotropin Treatment | An AE is any untoward medical occurrence in a participant administered a medicinal product that need not necessarily have a causal relationship with the product treatment or usage. An SAE is any untoward medical occurrence in a participant administered a medicinal or nutritional product at any dose that resulted to death, life-threatening, hospitalization or prolongation of hospitalization, persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); and congenital anomaly/birth defect. | Baseline up to 5 years | 
| Number of Participants Who Discontinued Study Due to Adverse Events | An AE is any untoward medical occurrence in a participant administered a medicinal product that need not necessarily have a causal relationship with the product treatment or usage. An SAE is any untoward medical occurrence in a participant administered a medicinal or nutritional product at any dose that resulted to death, life-threatening, hospitalization or prolongation of hospitalization, persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); and congenital anomaly/birth defect. Participants who discontinued study due to AEs were reported. | Baseline up to 5 years | 
| Weight of Participants at Baseline, Years 1, 2, 3, 4 and 5 | Weight of participants was measured in kilograms (kg). | Baseline, Year 1, 2, 3, 4, 5 | 
| Change From Baseline in Weight of Participants at Years 1, 2, 3, 4 and 5 | Weight of participants was measured in kg. | Baseline, Year 1, 2, 3, 4, 5 | 
| Height of Participants at Baseline, Years 1, 2, 3, 4 and 5 | Height of participants was measured in centimeters. | Baseline, Year 1, 2, 3, 4, 5 | 
| Change From Baseline in Height of Participants at Years 1, 2, 3, 4 and 5 | Height of participants was measured in centimeters. | Baseline, Year 1, 2, 3, 4, 5 | 
| Body Mass Index (BMI) of Participants at Baseline, Years 1, 2, 3, 4 and 5 | BMI was defined as an index for assessing overweight and underweight and was obtained by dividing body weight in kilograms (kg) by height in meters squared (m^2). | Baseline, Year 1, 2, 3, 4, 5 | 
| Change From Baseline in Body Mass Index of Participants at Years 1, 2, 3, 4 and 5 | BMI was defined as an index for assessing overweight and underweight and was obtained by dividing body weight in kilograms (kg) by height in m^2. | Baseline, Year 1, 2, 3, 4, 5 | 
| Blood Pressure (BP) of Participants at Baseline, Years 1, 2, 3, 4 and 5 | Measurement of BP included supine systolic blood pressure (SBP) and diastolic blood pressure (DBP). | Baseline, Year 1, 2, 3, 4, 5 | 
| Change From Baseline in Blood Pressure of Participants at Years 1, 2, 3, 4 and 5 | Measurement of BP included supine SBP and DBP. | Baseline, Year 1, 2, 3, 4, 5 | 
| Heart Rate of Participants at Baseline, Years 1, 2, 3, 4 and 5 | Heart rate was measured in supine position. | Baseline, Year 1, 2, 3, 4, 5 | 
| Change From Baseline in Heart Rate of Participants at Years 1, 2, 3, 4 and 5 | Heart rate was measured in supine position. | Baseline, Year 1, 2, 3, 4, 5 | 
| Percentage of Participants With Body Composition Assessments at Baseline, Years 1, 2, 3 and 4 | Body composition included parameters fat mass and muscle mass. | Baseline, Year 1, 2, 3, 4 | 
| Percentage of Participants With Computed Tomography (CT) or Magnetic Resonance Imaging (MRI) Investigation at Baseline, Years 1, 2, 3, 4 and 5 | CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue and blood vessels. MRI investigation uses strong magnetic field and radio waves to create detailed images of the organs and tissues within the body. | Baseline, Year 1, 2, 3, 4, 5 | 
| Percentage of Participants With Any Change From Baseline in Hormone Abnormalities at Years 1, 2, 3, and 4 | Hormones that were evaluated were thyroid stimulating hormone, adrenocorticotropic hormone, luteinizing hormone, follicle-stimulating hormone, antidiuretic hormone and prolactin hormone. Abnormalities were judged by the investigator. | Baseline, Year 1, 2, 3, 4 | 
| Percentage of Participants With Any Concomitant Medication at Baseline and During Follow-up | Percentage of participants taking any medications other than Genotropin (concomitant medication) are reported. | Baseline, Follow-up (during 28 days after last dose of Genotropin treatment) |