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

• ClinicalTrials.gov Identifier: NCT01947894
• Recruitment Status: Completed
• First Posted: September 23, 2013
• Results First Posted: November 20, 2019
• Last Update Posted: November 20, 2019
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment
Growth Hormone Deficiency	Other: Non Interventional Study

Detailed Description:

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

Study Design

• Study Type: Observational
• Actual Enrollment: 377 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: 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
• Actual Study Start Date: November 20, 2013
• Actual Primary Completion Date: October 31, 2018
• Actual Study Completion Date: October 31, 2018

Groups and Cohorts

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

Outcome Measures

Primary Outcome Measures :

1. Number of Participants Classified According to Insulin-like Growth Factor (IGF-I) Assessments [ Time Frame: Up to 5 years (after baseline visit) ]
   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.

Secondary Outcome Measures :

1. Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to 5 years ]
   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.
2. Number of Treatment Related Adverse Events [ Time Frame: Baseline up to 5 years ]
   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.
3. Number of Adverse Events Leading to Withdrawal of Genotropin Treatment [ Time Frame: Baseline up to 5 years ]
   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.
4. Number of Participants Who Discontinued Study Due to Adverse Events [ Time Frame: Baseline up to 5 years ]
   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.
5. Weight of Participants at Baseline, Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
   Weight of participants was measured in kilograms (kg).
6. Change From Baseline in Weight of Participants at Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
   Weight of participants was measured in kg.
7. Height of Participants at Baseline, Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
   Height of participants was measured in centimeters.
8. Change From Baseline in Height of Participants at Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
   Height of participants was measured in centimeters.
9. Body Mass Index (BMI) of Participants at Baseline, Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 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).
10. Change From Baseline in Body Mass Index of Participants at Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 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.
11. Blood Pressure (BP) of Participants at Baseline, Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
    Measurement of BP included supine systolic blood pressure (SBP) and diastolic blood pressure (DBP).
12. Change From Baseline in Blood Pressure of Participants at Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
    Measurement of BP included supine SBP and DBP.
13. Heart Rate of Participants at Baseline, Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
    Heart rate was measured in supine position.
14. Change From Baseline in Heart Rate of Participants at Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 5 ]
    Heart rate was measured in supine position.
15. Percentage of Participants With Body Composition Assessments at Baseline, Years 1, 2, 3 and 4 [ Time Frame: Baseline, Year 1, 2, 3, 4 ]
    Body composition included parameters fat mass and muscle mass.
16. Percentage of Participants With Computed Tomography (CT) or Magnetic Resonance Imaging (MRI) Investigation at Baseline, Years 1, 2, 3, 4 and 5 [ Time Frame: Baseline, Year 1, 2, 3, 4, 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.
17. Percentage of Participants With Any Change From Baseline in Hormone Abnormalities at Years 1, 2, 3, and 4 [ Time Frame: Baseline, Year 1, 2, 3, 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.
18. Percentage of Participants With Any Concomitant Medication at Baseline and During Follow-up [ Time Frame: Baseline, Follow-up (during 28 days after last dose of Genotropin treatment) ]
    Percentage of participants taking any medications other than Genotropin (concomitant medication) are reported.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

Patients wiht Growth Hormone Deficiency

Criteria

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.

Contacts and Locations

Locations

Sweden

Vastra Gotalands Regionen

Skovde, Skaraborg, Sweden, 541 85

Landstinget Dalarna

Falun, Sweden, 791 82

Sahlgrenska University hospital

Goteborg, Sweden, 413 45

Central Hospital/ Department of Medicine

Kristianstad, Sweden, 291 85

Universitetssjukhuset, EM-kliniken

Linköping, Sweden, 581 85

Ljungby Lasarettet

Ljungby, Sweden, 341 82

University Hospital SUS

Malmo, Sweden, 205 02

Landstinget i Stockholms Lan

Stockholm, Sweden, 118 83

Karolinska Universitetssjukhuset, Kliniken for Endokrinologi

Stockholm, Sweden, 171 76

Akademiska sjukhuset / Medicincentrum, Diabetes- och Endokrinsektionen

Uppsala, Sweden, 751 85

Landstinget i Jonkopings Lan

Varnamo, Sweden, 331 85

Medicinkliniken, Centrallasarettet Vaxjo

Vaxjo, Sweden, 351 85

Sponsors and Collaborators

Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

  Study Documents (Full-Text)

Documents provided by Pfizer:

Study Protocol  [PDF] October 23, 2015

Statistical Analysis Plan  [PDF] June 14, 2019

More Information

Additional Information:

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

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT01947894
• Other Study ID Numbers: A6281313; SWEGHO ( Other Identifier: Alias Study Number )
• First Posted: September 23, 2013
• Results First Posted: November 20, 2019
• Last Update Posted: November 20, 2019
• Last Verified: October 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No
• 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.

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Dwarfism, Pituitary; Endocrine System Diseases; Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases