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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03831880
Recruitment Status : Completed
First Posted : February 6, 2019
Results First Posted : October 14, 2021
Last Update Posted : October 14, 2021
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Description
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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.

Condition or disease Intervention/treatment Phase
Growth Hormone Deficiency Drug: Genotropin Drug: somatrogon Phase 3

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 Design
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Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 87 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: 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
Actual Study Start Date : February 7, 2019
Actual Primary Completion Date : August 28, 2020
Actual Study Completion Date : August 28, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hormones

Arms and Interventions
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Arm Intervention/treatment
Daily to Weekly
Genotropin to somatrogon
 Drug: Genotropin
Genotropin (dose [mg] at time of enrollment) given subcutaneously once daily

Drug: somatrogon
0.66 mg/kg/week given subcutaneously once weekly
Weekly to Daily
somatrogon to Genotropin
 Drug: Genotropin
Genotropin (dose [mg] at time of enrollment) given subcutaneously once daily

Drug: somatrogon
0.66 mg/kg/week given subcutaneously once weekly


Outcome Measures
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Primary Outcome Measures :
  1. Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire [ Time Frame: Baseline ]
    Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference [5 questions]: a measure of life interference [daily activities/social activities/leisure/night away from home/travel]; life interference-changes to life routine [1 question]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone [GH] injections [1 question]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

  2. Total Score Related to Overall Life Interference Assessed at Week 12, Using DCOA 1 Questionnaire [ Time Frame: Week 12 ]
    Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference [5 questions]: a measure of life interference [daily activities/social activities/leisure/night away from home/travel]; life interference-changes to life routine [1 question]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone [GH] injections [1 question]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

  3. Total Score Related to Overall Life Interference Assessed at Week 24, Using DCOA 1 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference [5 questions]: a measure of life interference [daily activities/social activities/leisure/night away from home/travel]; life interference-changes to life routine [1 question]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone [GH] injections [1 question]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

  4. Total Score Related to Overall Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference [5 questions]: a measure of life interference [daily activities/social activities/leisure/night away from home/travel]; life interference-changes to life routine [1 question]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone [GH] injections [1 question]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).


Secondary Outcome Measures :
  1. Total Score Related to Pen Ease of Use Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked 5 questions from Section I of the Injection Pen Assessment Questionnaire (IPAQ) patient-reported outcome (PRO) tool related to pen ease of use and used a 5-point scale: 1= very easy, 2= somewhat easy, 3= neither easy nor difficult, 4= somewhat difficult, 5= very difficult. The total score related to pen ease of use was sum of all 5 questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  2. Total Score Related to Pen Ease of Use by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked 5 questions from Section I of the IPAQ PRO tool related to pen ease of use and used a 5-point scale: 1= very easy, 2= somewhat easy, 3= neither easy nor difficult, 4= somewhat difficult, 5= very difficult. The total score related to pen ease of use was sum of all 5 questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  3. Total Score Related to Ease of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to ease of injection schedule and used a 5-point scale: 1= very easy, 2= somewhat easy, 3= neither easy nor difficult, 4= somewhat difficult, 5= very difficult. The total score related to ease of the injection schedule ranged from 1 to 5; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  4. Total Score Related to Ease of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to ease of injection schedule and used a 5-point scale: 1= very easy, 2= somewhat easy, 3= neither easy nor difficult, 4= somewhat difficult, 5= very difficult. The total score related to ease of the injection schedule ranged from 1 to 5; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  5. Total Score Related to Convenience of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to ease of injection schedule and used a 7-point scale: 1=extremely convenient to 7=extremely inconvenient. The total score related to convenience of injection schedule ranged from 1 to 7; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  6. Total Score Related to Convenience of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to ease of injection schedule and used a 7-point scale: 1=extremely convenient to 7=extremely inconvenient. The total score related to convenience of injection schedule ranged from 1 to 7; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  7. Total Score Related to Satisfaction With Overall Treatment Experience Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to participant satisfaction with treatment and used a 5-point scale: 1=very satisfied to 5=very dissatisfied. The total score related to satisfaction with overall treatment ranged from 1 to 5; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  8. Total Score Related to Satisfaction With Overall Treatment Experience by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to participant satisfaction with treatment and used a 5-point scale: 1=very satisfied to 5=very dissatisfied. The total score related to satisfaction with overall treatment ranged from 1 to 5; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  9. Total Scores Related to Willingness to Continue Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to participant willingness to continue treatment and used a 5-point scale: 1=extremely willing to 5=not at all willing. The total score related to willingness to continue injection schedule ranged from 1 to 5; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  10. Total Scores Related to Willingness to Continue Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to participant willingness to continue treatment and used a 5-point scale: 1=extremely willing to 5=not at all willing. The total score related to willingness to continue injection schedule ranged from 1 to 5; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant a better outcome.

  11. Total Scores Related to Injection Signs and Symptoms for Participants Aged 8 Years and Above Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participants (8-17 years old). Participants were asked 4 questions from Section I of the IPAQ PRO tool related to participant's injection signs and symptoms and used a 11-point scale: 0=no pain to 10=worst possible pain; 0=no stinging to 10=worst possible stinging; 0=no bruising to 10=worst possible bruising; and 0=no bleeding to 10=worst possible bleeding, respectively. The total score was sum of all questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for injection signs and symptoms meant a better outcome.

  12. Total Scores Related to Injection Signs and Symptoms for Participants Aged 8 Years and Above by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participants (8-17 years old). Participants were asked 4 questions from Section I of the IPAQ PRO tool related to participant's injection signs and symptoms and used a 11-point scale: 0=no pain to 10=worst possible pain; 0=no stinging to 10=worst possible stinging; 0=no bruising to 10=worst possible bruising; and 0=no bleeding to 10=worst possible bleeding, respectively. The total score was sum of all questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for injection signs and symptoms meant a better outcome.

  13. Total Scores Related to Assessment of Signs, Completed by Caregiver for Children Aged <8 Years Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by caregiver for children under 8 years. Participants were asked 2 questions from Section I of the IPAQ PRO tool related to participant's assessment of signs and used a 11-point scale: 0=no bruising to 10=worst possible bruising and 0=no bleeding to 10=worst possible bleeding, respectively. The total score was sum of all questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for assessment of signs meant a better outcome.

  14. Total Scores Related to Assessment of Signs, Completed by Caregiver for Children Aged <8 Years by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by caregiver for children under 8 years. Participants were asked 2 questions from Section I of the IPAQ PRO tool related to participant's assessment of signs and used a 11-point scale: 0=no bruising to 10=worst possible bruising and 0=no bleeding to 10=worst possible bleeding, respectively. The total score was sum of all questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for assessment of signs meant a better outcome.

  15. Total Scores Related to Caregiver Life Interference, Including Family Life Interference Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by caregiver. Participants were asked 13 questions from Section I of the IPAQ PRO tool related to caregiver life interference and used a 5-point scale: 1= never to 5= always. The total score ranged was sum of scores from all questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for caregiver and family life interference meant less life interference (a better outcome).

  16. Total Scores Related to Caregiver Life Interference, Including Family Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by caregiver. Participants were asked 13 questions from Section I of the IPAQ PRO tool related to caregiver life interference and used a 5-point scale: 1= never to 5= always. The total score ranged was sum of scores from all questions; scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for caregiver and family life interference meant less life interference (a better outcome).

  17. Total Scores Related to Missed Injections Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire [ Time Frame: Baseline, Week 12, Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to number of missed injections (daily or weekly administration) during past 4 weeks. The total scores ranged from 0 to 31 for daily administration (Genotropin) and from 0 to 5 for weekly administration (Somatrogon). All scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for missed injections meant a better outcome.

  18. Total Scores Related to Missed Injections by Treatment in Overall Study, Using DCOA 1 Questionnaire [ Time Frame: Baseline up to Week 24 ]
    Participants were assessed for their treatment experience using DCOA 1 questionnaire completed by participant/caregiver dyads. Participants were asked a question from Section I of the IPAQ PRO tool related to number of missed injections (daily or weekly administration) during past 4 weeks. The total scores ranged from 0 to 31 for daily administration (Genotropin) and from 0 to 5 for weekly administration (Somatrogon). All scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score for missed injections meant a better outcome.

  19. Number of Participants as Per Responses to Choice of Injection Pen Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/caregivers responded to question from Section II of the IPAQ PRO tool "If you were given the choice between the daily growth hormone injection pen and the weekly growth hormone injection pen, which pen would you choose?" Response was: 1) the daily injection pen (Genotropin) or 2) the weekly injection pen (Somatrogon).

  20. Number of Participants as Per Responses to Preferred Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/caregivers responded to question from Section II of the IPAQ PRO tool "Which growth hormone injection schedule do you prefer overall?" by choosing from any 1 option from: 1) prefer the weekly injection schedule (Somatrogon); 2) prefer the daily injection schedule (Genotropin); 3) no preference.

  21. Number of Participants as Per Responses to Convenience of the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/caregivers responded to question from Section II of the IPAQ PRO tool "Which growth hormone injection schedule was more convenient overall?" by choosing from any 1 option from: 1) weekly injection schedule was more convenient (Somatrogon); 2) daily injection schedule was more convenient (Genotropin); 3) no difference.

  22. Number of Participants as Per Responses to Ease of Following Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/caregivers responded to question from Section II of the IPAQ PRO tool "Which growth hormone injection schedule was easier to follow overall?" by choosing from any 1 option from: 1) easier to follow weekly injection schedule (Somatrogon); 2) easier to follow daily injection schedule (Genotropin); 3) no difference.

  23. Number of Participants as Per Responses to Pen Ease of Use Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/caregiver were asked a question "Which pen was easier to use?" from Section II of the IPAQ PRO tool. Question had 4 parts: preparing the injection pen (Part I), setting the dose (Part II), injecting the medicine (Part III) and storing the pen (Part IV). Participants/caregiver expressed their preference by choosing from any 1 option for each activity from: 1) weekly pen easier to use (Somatrogon); 2) daily pen easier to use (Genotropin); 3) no difference.

  24. Number of Participants as Per Responses to Participant Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/caregiver were asked a question "Which injection schedule interfered less?" from Section II of the IPAQ PRO tool related to participant life interference. Participants were assessed for 5 activities: daily activities (Activity 1), social activities (Activity 2), recreation/leisure activities (Activity 3), spending night away from home (Activity 4) and travel (Activity 5). The participants expressed their preference by choosing from any 1 option for each activity from: 1) weekly injection schedule interfered less (Somatrogon); 2) daily injection schedule interfered less (Genotropin); 3) no difference.

  25. Number of Participants as Per Responses to Caregiver Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Caregivers of participants were asked a question "Which injection schedule interfered less?" from Section II of the IPAQ PRO tool related to caregiver life interference and were assessed for 5 activities: daily activities (Activity 1), social activities (Activity 2), recreation/leisure activities (Activity 3), spending night away from home (Activity 4) and travel (Activity 5). Preference was expressed by choosing from any 1 option for each activity from: 1) weekly injection schedule interfered less (Somatrogon); 2) daily injection schedule interfered less (Genotropin); 3) no difference. The caregivers responded for the participants, and in actual they respond to the number of participants only but per caregiver responses.

  26. Number of Participants as Per Responses to Family Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/ caregiver were asked a question "Which injection schedule interfered less?" from Section II of the IPAQ PRO tool related to family life interference and assessed for 5 activities: daily activities (Activity 1), social activities (Activity 2), recreation/leisure activities (Activity 3), spending night away from home (Activity 4) and travel (Activity 5). Preference was expressed by choosing from any 1 option for each activity from: 1) weekly injection schedule interfered less (Somatrogon); 2) daily injection schedule interfered less (Genotropin); 3) no difference.

  27. Number of Participants as Per Response to Benefit Relating to the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants were assessed for their treatment experience using DCOA 2 questionnaire completed by participant/caregiver dyads. Participants/caregiver were asked a question "How beneficial was to take injections less often?" from Section II of the IPAQ PRO tool pertaining to benefit relating to the Injection schedule and used a 5-point scale: 1= extremely beneficial, 2= very beneficial, 3= moderately beneficial, 4= slightly beneficial and 5= not at all beneficial. Lower score of benefit relating to injection schedule meant a better outcome.

  28. Number of Participants as Per Responses to Intention to Comply Assessed at Week 24, Using DCOA 2 Questionnaire [ Time Frame: Week 24 ]
    Participants/caregiver dyads were asked 4 questions "Which schedule would be better able to follow?" (Question 1), "Which schedule would be more likely to follow for a longer time?" (Question 2), "Which schedule would be better able to follow for a longer time?" (Question 3) and "Which schedule would be more likely to follow?" (Question 4) from Section II of the IPAQ PRO tool related to participant intention to comply with treatment. Options for each question were: 1) weekly injection (Somatrogon) 2) daily injection (Genotropin), or 3) no difference.

  29. Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score Assessed at Baseline, Week 12 and Week 24 [ Time Frame: Baseline, Week 12, Week 24 ]
    The PGIS-IDA rated the severity of the impact on daily activities due to the treatment administration during the past 4 weeks on a 7-point scale (1= not present to 7= extremely severe). Scores were transformed from raw scores to a 0 to 100 scale. Lower scores meant less impact on daily activities (better outcome).

  30. Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score by Treatment in Overall Study [ Time Frame: Baseline up to Week 24 ]
    The PGIS-IDA rated the severity of the impact on daily activities due to the treatment administration during the past 4 weeks on a 7-point scale (1= not present to 7= extremely severe). Scores were transformed from raw scores to a 0 to 100 scale. Lower scores meant less impact on daily activities (better outcome).


Other Outcome Measures:
  1. Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), Treatment-Emergent Treatment Related AEs and SAEs [ Time Frame: Baseline up to 35 days after last dose of study drug (up to 29 Weeks) ]
    An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. SAE was any untoward medical occurrence at any dose that: resulted in death, was life threatening (immediate risk of death), required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions), resulted in congenital anomaly/birth defect. Treatment-emergent AEs (TEAEs) were defined as events that occurred between first dose of study drug up to 35 days after last dose of study drug. Related TEAEs were those AEs who had relation to the study treatment and was judged by investigator.

  2. Number of Participants With Adverse Events According to Severity [ Time Frame: Baseline up to 35 days after last dose of study drug (up to 29 Weeks) ]
    AEs were assessed and categorized according to the severity as mild (did not interfered with participant's usual function), moderate (interfered to some extent with participant's usual function) and severe (interfered significantly with participant's usual function).

  3. Number of Participants With Discontinuation Due to Adverse Events (AEs) [ Time Frame: Baseline up to 35 days after last dose of study drug (up to 29 Weeks) ]
    An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. The discontinuations due to adverse events was defined for participants and reported in this outcome measure.

  4. Number of Participants With Laboratory Abnormalities [ Time Frame: Week 1 to Week 12, Week 13 to Week 24 ]
    The laboratory abnormality parameters included Hematology: erythrocyte (Er.) mean corpuscular volume, Er. mean corpuscular hemoglobin:<0.9*lower limit normal (LLN), leukocytes:<0.6*LLN, lymphocytes:<0.8*LLN, neutrophils:<0.8*LLN, greater than (>) 1.2*upper limit normal (ULN), eosinophils, monocytes:>1.2*ULN. Clinical chemistry: bilirubin, direct bilirubin, indirect bilirubin:>1.5*ULN, gamma glutamyl transferase:>3.0*ULN, albumin:>1.2*ULN, blood urea nitrogen:>1.3*ULN, urate:>1.2*ULN, high-density lipoprotein (HDL) cholesterol:<0.8*LLN, potassium, magnesium:>1.1*ULN, phosphate:>1.2*ULN, bicarbonate:<0.9*LLN, creatine kinase:>2.0*ULN. Urinalysis: specific gravity:>1.030, ketones, urine protein, urine hemoglobin, nitrite, leukocyte esterase:>=1.

  5. Number of Participants With Positive Anti-Recombinant Human Growth Hormone (rhGH) Antibodies and Neutralizing Antibodies (NAb) [ Time Frame: Baseline, Week 12, Week 24 ]
    Blood samples were collected for determination of rhGH and NAb. The participants who tested positive for antibodies were reported.

  6. Number of Participants With Positive Anti-Somatrogon Antibodies and Neutralizing Antibodies (NAb) [ Time Frame: Baseline, Week 12, Week 24 ]
    Blood samples were collected for determination of anti-somatrogon antibodies and NAb. The participants who tested positive for antibodies were reported.


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   3 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03831880


Locations
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Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
  Study Documents (Full-Text)

Documents provided by Pfizer:
Study Protocol  [PDF] August 26, 2019
Statistical Analysis Plan  [PDF] September 1, 2020

More Information
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Additional Information:

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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03831880    
Other Study ID Numbers: C0311002
2018-000918-38 ( EudraCT Number )
First Posted: February 6, 2019    Key Record Dates
Results First Posted: October 14, 2021
Last Update Posted: October 14, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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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