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Completed

NCT Number: NCT03831880

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

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.

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Key information

Conditions

Growth Hormone Deficiency Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases Dwarfism Dwarfism, Pituitary Endocrine System Diseases Hypopituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Pituitary Diseases

Age range

3 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

First Pediatric Clinic UMHAT "St. Marina" EAD, Varna, Bulgaria

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Children aged 3 years old and <18 years with either isolated GHD, or GH insufficiency.
  • 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.
  • IGF I SDS < 2.
  • 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

  • History of leukemia, lymphoma, sarcoma or any other cancer.
  • History of radiation therapy or chemotherapy.
  • Children with psychosocial dwarfism.
  • Children born small for gestational age (SGA) - birth weight and/or birth length < 2 SDS for gestational age.
  • Other causes of short stature such as uncontrolled primary hypothyroidism and rickets.
  • 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.
  • Treatment with regularly scheduled daily or weekly injectable medications other than Genotropin® Pen, Genotropin GoQuick®, HumatroPen® (USA only), or Omnitrope® Pen (USA only).
  • Diabetes Mellitus.
  • Current treatment with Genotropin MiniQuick.
  • History of any exposure to a long acting hGH preparation.
  • Known or suspected human immunodeficiency virus (HIV) positive patient, or patient with advanced diseases such as acquired immunodeficiency syndrome (AIDS) or tuberculosis.
  • Drug, substance, or alcohol abuse.
  • Known hypersensitivity to the components of the medication.
  • 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.
  • 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.
  • 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.
  • Participation in other studies involving investigational drug(s) within 30 days prior to study entry and/or during study participation.
  • Patient and/or the parent/legal guardian are likely to be non-compliant with respect to study conduct.
  • 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.
  • Children with closed epiphyses (this determination can be based on available existing clinical data).

Treatment and study plan

Genotropin

Drug

Genotropin (dose [mg] at time of enrollment) given subcutaneously once daily

Somatrogon

Drug

0.66 mg/kg/week given subcutaneously once weekly

Primary outcomes

  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 outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study 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

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Feb 6, 2019
Registry last updated
Oct 14, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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