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

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

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.

Study Overview

• Status: Completed
• Conditions: Growth Hormone Deficiency
• Intervention / Treatment: Other: Non Interventional Study

Detailed Description

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

• Study Type: Observational
• Enrollment (Actual): 377

Contacts and Locations

Study Locations

• Sweden
  • Falun, Sweden, 791 82
    • Landstinget Dalarna
  • Goteborg, Sweden, 413 45
    • Sahlgrenska University Hospital
  • Kristianstad, Sweden, 291 85
    • Central Hospital/ Department of Medicine
  • Linköping, Sweden, 581 85
    • Universitetssjukhuset, EM-kliniken
  • Ljungby, Sweden, 341 82
    • Ljungby Lasarettet
  • Malmo, Sweden, 205 02
    • University Hospital SUS
  • Stockholm, Sweden, 118 83
    • Landstinget i Stockholms Lan
  • Stockholm, Sweden, 171 76
    • Karolinska Universitetssjukhuset, Kliniken for Endokrinologi
  • Uppsala, Sweden, 751 85
    • Akademiska sjukhuset / Medicincentrum, Diabetes- och Endokrinsektionen
  • Varnamo, Sweden, 331 85
    • Landstinget i Jonkopings Lan
  • Vaxjo, Sweden, 351 85
    • Medicinkliniken, Centrallasarettet Vaxjo
  • Skaraborg
    • Skovde, Skaraborg, Sweden, 541 85
      • Vastra Gotalands Regionen

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients wiht Growth Hormone Deficiency

Description

Inclusion Criteria:

• Adult patients of 18 years of age and above and fulfilling one of the three alternatives a-c below;

  1. Newly diagnosed with GHD according to the current medical standard.
  2. Diagnosed with GHD before 2013 and previously treated with Genotropin and followed in KIMS®.
  3. 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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Adult Growth Hormone deficient Patients; Patients with GHD on Genotropin® replacement therapy.	Other: Non Interventional Study; Non Interventional Study

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): November 20, 2013
• Primary Completion (Actual): October 31, 2018
• Study Completion (Actual): October 31, 2018

Study Registration Dates

• First Submitted: June 18, 2013
• First Submitted That Met QC Criteria: September 9, 2013
• First Posted (Estimate): September 23, 2013

Study Record Updates

• Last Update Posted (Actual): November 20, 2019
• Last Update Submitted That Met QC Criteria: October 29, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases; Musculoskeletal Diseases; Hypothalamic Diseases; Bone Diseases; Bone Diseases, Endocrine; Pituitary Diseases; Dwarfism; Bone Diseases, Developmental; Hypopituitarism; Dwarfism, Pituitary
• Other Study ID Numbers: A6281313; SWEGHO (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No