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Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD

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ClinicalTrials.gov Identifier: NCT03831880
Recruitment Status : Completed
First Posted : February 6, 2019
Last Update Posted : December 9, 2020
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE December 21, 2018
First Posted Date  ICMJE February 6, 2019
Last Update Posted Date December 9, 2020
Actual Study Start Date  ICMJE February 7, 2019
Actual Primary Completion Date August 28, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 1, 2019)
Treatment burden assessed as the difference in mean overall 'Life Interference' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience. [ Time Frame: Change from Week 12 to Week 24 ]
Comparative assessment of 7 questions using a 5 point scale (never [1], rarely [2], sometimes [3], often [4] and always [5]) using the dyad questionnaire (Patient Life Interference Section I) and completed by the subject/caregiver. The total score can range from 7 to 35; a lower score for Life Interference is considered to be a better outcome.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 1, 2019)
  • Pen ease of use - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 5 questions using a 5 point scale (Very easy [1], Somewhat easy [2], Neither easy nor difficult [3], Somewhat difficult [4], and Very difficult [5]) using the dyad questionnaire (Pen Ease of Use Section I)) and completed by the subject/caregiver. The total score can range from 5 to 25; a lower score for Pen Ease of Use is considered to be a better outcome. The difference in mean overall 'Pen Ease of Use' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience will be assessed.
  • Ease of the injection schedule - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 1 question using a 5 point scale (Very easy [1], Somewhat easy [2], Neither easy nor difficult [3], Somewhat difficult [4] and Very difficult [5]) using the dyad questionnaire (Ease of Injection Schedule, Section I) and completed by the subject/caregiver. The total score can range from 1 to 5; a lower score for Ease of Injection Schedule is considered to be a better outcome. The difference in mean overall 'Ease of Injection Schedule' total score between the weekly injection schedule and daily injection schedule after each treatment schedule experience will be assessed.
  • Convenience of the injection schedule - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 1 question using a 7 point scale (Extremely convenient [1], Very convenient [2], Convenient [3], Neither convenient nor inconvenient [4], Inconvenient [5], Very inconvenient [6], and Extremely inconvenient [7]) using the dyad questionnaire (Ease of Injection Schedule, Section I) and completed by the subject/caregiver. The total score can range from 1 to 7; a lower score for Convenience of the Injection Schedule is considered to be a better outcome. The difference in mean overall 'Convenience of the Injection Schedule' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience will be assessed.
  • Satisfaction with overall treatment experience- Comparative assessment of 1 question, using a 5 point scale (Very satisfied to Very dissatisfied) comparing the weekly treatment experience to the daily treatment experience- Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 1 question, using a 5 point scale (Very satisfied [1], Satisfied [2], Neither satisfied nor dissatisfied [3], Dissatisfied [4], and Very dissatisfied [5]) using the dyad questionnaire (Patient Satisfaction and Willingness to Continue, Section I) and completed by the subject/caregiver. The total score can range from 1 to 5; a lower score for Satisfaction with Overall Treatment Experience is considered to be a better outcome. The difference in mean overall 'Patient Satisfaction' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience will be assessed.
  • Willingness to continue injection schedule - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 1 question, using a 5 point scale (Extremely [1], Very [2], Moderately [3], Slightly [4], and Not at all [5)] using the dyad questionnaire (Patient Satisfaction and Willingness to Continue, Section I) and completed by the subject/caregiver. The total score can range from 1 to 5; a lower score for Willingness to Continue Injection Schedule is considered to be a better outcome. The difference in mean overall 'Patient Willingness to Continue' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience will be assessed.
  • Injection signs and symptoms (from the patient; completed by subjects 8 years and older) - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 4 questions, each using an 11 point scale (No pain [0] to Worst possible pain [10]; No stinging [0] to Worst possible stinging [10]; No bruising [0] to Worst possible bruising [10]; and No bleeding [0] to Worst possible bleeding [10]) using the dyad questionnaire (Patient Injection Signs and Symptoms) and completed by the subjects 8-17 years old. The total score can range from 0 to 40; a lower score for Patient Injection Signs and Symptoms is considered to be a better outcome. The difference in mean overall 'Patient Injection Signs and Symptoms' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience in subjects 8 years and older will be assessed.
  • Assessment of signs (from the caregiver; completed by caregiver for subjects under 8 years of age) - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 4 questions, each using an 11 point scale (No bruising [0] to Worst possible bruising [10]; and No bleeding [0] to Worst possible bleeding [10]) using the dyad questionnaire (Patient Injection Signs and Symptoms) and completed by the caregiver for subjects less than 8 years old. The total score can range from 0 to 20; a lower score for Patient Injection Signs and Symptoms is considered to be a better outcome. The difference in mean overall 'Patient Injection Signs and Symptoms' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience in subjects under 8 years will be assessed.
  • Caregiver life interference, including family life interference - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 13 questions using a 5 point scale (Never [1], Rarely [2], Sometimes [3], Often [4] and Always [5]) using the dyad questionnaire (Caregiver Life Interference Section I) and completed by the caregiver. The total score can range from 13 to 65; a lower score for Caregiver Life Interference, Including Family Life Interference is considered to be a better outcome. The difference in mean overall 'Caregiver Life Interference, Including Family Life Interference' total scores between the weekly injection schedule and daily injection schedule after each treatment schedule experience will be assessed.
  • Missed Injections - Assessment 1 [ Time Frame: Change from Week 12 to Week 24 ]
    Comparative assessment of 2 questions, 1 question assessing number of missed injections during past 4 weeks (filled in by subject/caregiver) and the other question assessing the reason for missing injection(s) using a list of multiple potential reasons of which the subject/caregiver can check all that apply, using the dyad questionnaire (Missed Injections [Daily or Weekly Administration], Section I). The difference in percent injections missed and reasons for missing injections between the weekly injection schedule and daily injection schedule after each treatment schedule experience will be assessed.
  • Choice of injection pen - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 1 question in which the patient/caregiver is asked to choose 1 option (Choose the Daily pen or Choose the Weekly pen) using the dyad questionnaire (The Injection Pen Assessment Questionnaire, Section II). The proportion of subjects selecting each option will be assessed.
  • Preferred injection schedule - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 1 question in which the patient/caregiver is asked to choose 1 option (Prefer the Daily injection schedule or Prefer the Weekly injection schedule or No preference) using the dyad questionnaire (The Injection Pen Assessment Questionnaire, Section II). The proportion of subjects selecting each option will be assessed.
  • Convenience of injection schedule - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 1 question in which the patient/caregiver is asked to choose 1 option (Daily injection schedule was more convenient or Weekly injection schedule was more convenient or No difference) using the dyad questionnaire (The Injection Pen Assessment Questionnaire, Section II). The proportion of subjects selecting each option will be assessed.
  • Ease of the injection schedule - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 1 question in which the patient/caregiver is asked to choose 1 option (Easier to follow Daily injection schedule or Easier to follow Weekly injection schedule or No difference) using the dyad questionnaire (The Injection Pen Assessment Questionnaire, Section II). The proportion of subjects selecting each option will be assessed.
  • Patient Life Interference - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 5 activities in which the subject/caregiver is asked to choose 1 option (Daily injection schedule interfered less or Weekly injection schedule interfered less or No difference) for each activity using the dyad questionnaire (Patient Life Interference, Section II). The proportion of subjects selecting each option will be assessed.
  • Caregiver Life Interference, including family life interference - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 2 questions, one related to the caregiver's activities (5 activities) and the other related to other family members' activities (5 activities) in which the caregiver is asked to choose 1 option (Daily injection schedule interfered less or Weekly injection schedule interfered less or No difference) for each activity using the dyad questionnaire (Patient Life Interference, Section II). The proportion of subjects selecting each option will be assessed.
  • Benefit relating to the injection schedule - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 1 question using a 5 point scale (Extremely [1], Very [2], Moderately [3], Slightly [4], and Not at all [5]) answered by subject/caregiver of how beneficial it is to take injections less often, using the dyad questionnaire (Patient Satisfaction and Willingness to Continue, Section II). The total score can range from 1 to 5; a lower score for Patient Satisfaction and Willingness to Continue is considered to be a better outcome.
  • Intention to Comply - Assessment 2 [ Time Frame: 24 Weeks ]
    Assessment of 4 questions in which subject/caregiver must choose 1 option for each question (More likely to follow Daily injection schedule or More likely to follow Weekly injection schedule or No Difference); (Better able to follow Daily injection schedule or Better able to follow Weekly injection schedule or No difference); (More likely to follow Daily injection schedule for a longer time or More likely to follow Weekly injection schedule for a longer time or No difference); (Better able to follow Daily injection schedule for a longer time or Better able to follow Weekly injection schedule for a longer time or No difference) using the dyad questionnaire (Patient Intention to Comply with Treatment, Section II). The proportion of subjects selecting each option will be assessed.
  • The Patient Global Impression Severity - Impact on Daily Activities [ Time Frame: Change from baseline at week 12 and week 24. ]
    Comparative assessment of 1 question using a 7 point scale (Not present [1], Very mild [2], Mild [3], Moderate [4], Moderately severe [5], Severe [6], Extremely severe [7]) using the Patient Global Impression Severity - Impact on Daily Activities and completed by the subject/caregiver. The total score can range from 1 to 7; a score of 1 is considered to be a better outcome.
  • Frequency, severity, and relationship of adverse events (including serious adverse events and discontinuations due to adverse events) by treatment group [ Time Frame: 24 Weeks ]
    Frequency, severity, and relationship of adverse events (including serious adverse events and discontinuations due to adverse events) by treatment group
  • Frequency and severity of abnormal lab values by treatment group. [ Time Frame: 24 Weeks ]
    Frequency and severity of abnormal lab values by treatment group.
  • Frequency of anti-rhGH antibodies (and neutralizing antibodies) by treatment group. [ Time Frame: 24 Weeks ]
    Frequency of anti-rhGH antibodies (and neutralizing antibodies) by treatment group.
  • Frequency of anti-somatrogon antibodies (and neutralizing antibodies) by treatment group. [ Time Frame: 24 Weeks ]
    Frequency of anti-somatrogon antibodies (and neutralizing antibodies) by treatment group. Note that anti-somatrogon antibodies are tested at the 12 week and 24 week time points for subjects who have been on somatrogon for the previous 12 weeks of treatment.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD
Official Title  ICMJE A PHASE 3, RANDOMIZED, MULTICENTER, OPEN-LABEL, CROSSOVER STUDY ASSESSING SUBJECT PERCEPTION OF TREATMENT BURDEN WITH USE OF WEEKLY GROWTH HORMONE (SOMATROGON) VERSUS DAILY GROWTH HORMONE (GENOTROPIN (REGISTERED)) INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY
Brief Summary This is an open label randomized 24 week crossover trial assessing the treatment burden of a weekly growth hormone injection regimen (somatrogon) compared to a daily growth hormone injection regimen (Genotropin). Approximately 90 children with growth hormone deficiency who have been stable on treatment with daily Genotropin will be enrolled.
Detailed Description Subjects will be randomized to one of two sequences, either 12 weeks of continued treatment with daily Genotropin followed by 12 weeks of treatment with weekly somatrogon, or 12 weeks of treatment with weekly somatrogon followed by 12 weeks of treatment with daily Genotropin. Subjects will have study visits at Baseline, Weeks 6, 12, 18, and 24. Subjects will also be followed up by phone 8 to 12 days after each treatment period begins (Week 1 and Week 13). Subjects and caregivers (as a Dyad) will complete questionnaires assessing treatment burden at baseline and at the end of each 12 week treatment period. All subjects/caregivers will receive a follow up phone call at Week 28.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Growth Hormone Deficiency
Intervention  ICMJE
  • Drug: Genotropin
    Genotropin (dose [mg] at time of enrollment) given subcutaneously once daily
  • Drug: somatrogon
    0.66 mg/kg/week given subcutaneously once weekly
Study Arms  ICMJE
  • Daily to Weekly
    Genotropin to somatrogon
    Interventions:
    • Drug: Genotropin
    • Drug: somatrogon
  • Weekly to Daily
    somatrogon to Genotropin
    Interventions:
    • Drug: Genotropin
    • Drug: somatrogon
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 15, 2020)
87
Original Estimated Enrollment  ICMJE
 (submitted: February 1, 2019)
90
Actual Study Completion Date  ICMJE August 28, 2020
Actual Primary Completion Date August 28, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Children aged 3 years old and <18 years with either isolated GHD, or GH insufficiency.
  2. Currently on treatment with either Genotropin Pen®, Genotropin GoQuick Pen®, HumatroPen® (United States of America [USA] only), or Omnitrope® Pen (USA only) ≥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.

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, Genotropin GoQuick®, HumatroPen® (USA only), or Omnitrope® Pen (USA only).
  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).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Bulgaria,   Czechia,   Slovakia,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03831880
Other Study ID Numbers  ICMJE C0311002
2018-000918-38 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
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.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP