Growth Hormone DeficiencyPhase 3NCT03831880
Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD
A Phase 3 study testing Genotropin and somatrogon in people with Growth Hormone Deficiency, run by Pfizer at 32 sites in 5 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Growth Hormone Deficiency
- What is being tested?
- Genotropin and somatrogon
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 87 participants
- Where
- 32 sitesin 5 countries
- Study dates
- 7 Feb 2019to 28 Aug 2020
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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).
Trial locations and contacts
32 sites in 5 countries.
32 sites in 5 countries
United States18 sites
Emory Children's Center
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Rocky Mountain Pediatric Endocrinology
Centennial, Colorado, 80112
Pediatric Endocrine Associates, PC
Greenwood Village, Colorado, 80111
Hackensack University Medical Center
Hackensack, New Jersey, 07601
IU Health Pharmacy
Indianapolis, Indiana, 46202
Riley Hospital for Children
Indianapolis, Indiana, 46202
Nemours Children's Health System
Jacksonville, Florida, 32207
Nemours Children's Specialty Care
Jacksonville, Florida, 32207
Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64111
Nemours Biomedical Research
Orlando, Florida, 32827
Nemours Children's Hospital
Orlando, Florida, 32827
Nemours Children's Clinic
Pensacola, Florida, 32514
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Center of Excellence in Diabetes and Endocrinology
Sacramento, California, 95821
MultiCare Health System - Mary Bridge Children's Health Center
Tacoma, Washington, 98405
MultiCare Institute for Research & Innovation
Tacoma, Washington, 98405
Shriners Hospitals for Children
Tampa, Florida, 33612
United Kingdom5 sites
St. Georges University Hospitals NHS Foundation Trust
London, SW17 0QT
St. Georges University Hospital NHS Foundation Trust
London, SW17 0RE
The Institute of Child Health, University College London
London, WC1N 1EH
Great Ormond Street Hospital
London, WC1N 3JH
NIHR Clinical Research Facility, Great Ormond Street Hospital for Children NHS Trust
London, WC1N 3JH
Czechia4 sites
Fakultni nemocnice Brno, Pediatricka Klinika
Brno, 61300
Nemocnicni lekarna FN Brno
Brno, 61300
Fakultni nemocnice v Motole, Pediatricka klinika 2.LF UK a FN Motol
Prague, 150 06
Nemocnicni lekarna FN Motol
Prague, 150 06
Slovakia4 sites
Nemocnicna Lekaren Nudch
Bratislava, 833 40
Národný ústav detských chorôb, Detská klinika
Bratislava, 833 40
Detská fakultná nemocnica Košice Klinika detí a dorastu LF UPJŠ a DFN
Košice, 040 11
Nemocnica lekaren DFN Kosice, Klinika deti a dorastu
Košice, 040 11
About this study
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
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.
Read the 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.
- Conditions
- Growth Hormone Deficiency
- Being tested
- Genotropin, somatrogon
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GenotropinDrug
Genotropin (dose [mg] at time of enrollment) given subcutaneously once daily
somatrogonDrug
0.66 mg/kg/week given subcutaneously once weekly
| Study group | Role | What they receive |
|---|---|---|
| Daily to Weekly | Other | Genotropin to somatrogon Receives: Drug: Genotropin; Drug: somatrogon |
| Weekly to Daily | Other | somatrogon to Genotropin Receives: Drug: Genotropin; Drug: somatrogon |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire
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).
Measured: Baseline
Total Score Related to Overall Life Interference Assessed at Week 12, Using DCOA 1 Questionnaire
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).
Measured: Week 12
Total Score Related to Overall Life Interference Assessed at Week 24, Using DCOA 1 Questionnaire
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).
Measured: Week 24
Total Score Related to Overall Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire
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).
Measured: Baseline up to Week 24
Secondary outcomes (30)
Total Score Related to Pen Ease of Use Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
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.
Measured: Baseline, Week 12, Week 24
Total Score Related to Pen Ease of Use by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
Total Score Related to Ease of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
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.
Measured: Baseline, Week 12, Week 24
Total Score Related to Ease of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
Total Score Related to Convenience of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
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.
Measured: Baseline, Week 12, Week 24
Total Score Related to Convenience of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
Total Score Related to Satisfaction With Overall Treatment Experience Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
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.
Measured: Baseline, Week 12, Week 24
Total Score Related to Satisfaction With Overall Treatment Experience by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
Total Scores Related to Willingness to Continue Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
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.
Measured: Baseline, Week 12, Week 24
Total Scores Related to Willingness to Continue Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
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
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.
Measured: Baseline, Week 12, Week 24
Total Scores Related to Injection Signs and Symptoms for Participants Aged 8 Years and Above by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
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
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.
Measured: Baseline, Week 12, Week 24
Total Scores Related to Assessment of Signs, Completed by Caregiver for Children Aged <8 Years by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
Total Scores Related to Caregiver Life Interference, Including Family Life Interference Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
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).
Measured: Baseline, Week 12, Week 24
Total Scores Related to Caregiver Life Interference, Including Family Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire
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).
Measured: Baseline up to Week 24
Total Scores Related to Missed Injections Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
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.
Measured: Baseline, Week 12, Week 24
Total Scores Related to Missed Injections by Treatment in Overall Study, Using DCOA 1 Questionnaire
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.
Measured: Baseline up to Week 24
Number of Participants as Per Responses to Choice of Injection Pen Assessed at Week 24, Using DCOA 2 Questionnaire
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).
Measured: Week 24
Number of Participants as Per Responses to Preferred Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Responses to Convenience of the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Responses to Ease of Following Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Responses to Pen Ease of Use Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Responses to Participant Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Responses to Caregiver Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Responses to Family Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Response to Benefit Relating to the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Number of Participants as Per Responses to Intention to Comply Assessed at Week 24, Using DCOA 2 Questionnaire
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.
Measured: Week 24
Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score Assessed at Baseline, Week 12 and 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).
Measured: Baseline, Week 12, Week 24
Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score by Treatment in Overall Study
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).
Measured: Baseline up to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2019
- Study start7 Feb 2019
- Primary completion28 Aug 2020
- Study completion28 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 87 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03831880
- Sponsor's protocol ID
- C0311002
- Other IDs
- 2018-000918-38, 2018
- Status last verified
- Oct 2021
- First submitted
- 21 Dec 2018
- Last updated
- 14 Oct 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03831880
- Acronym / sponsor ID
- C0311002
- Conditions
- Growth Hormone Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 87 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 7 Feb 2019 (actual)
- Primary completion
- 28 Aug 2020 (actual)
- Study completion
- 28 Aug 2020 (actual)
- First posted
- 6 Feb 2019 (actual)
- Results
- Posted 14 Oct 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Daily to Weekly | Other | Drug: Genotropin, Drug: somatrogonGenotropin to somatrogon |
| Weekly to Daily | Other | Drug: Genotropin, Drug: somatrogonsomatrogon to Genotropin |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (20 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GenotropinGenotropin (dose [mg] at time of enrollment) given subcutaneously once daily | Drug | Daily to Weekly, Weekly to Daily |
| somatrogon0.66 mg/kg/week given subcutaneously once weekly | Drug | Daily to Weekly, Weekly to Daily |
Primary outcome
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).
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).
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).
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).
30 secondary outcomes
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
Total Score Related to Pen Ease of Use by Treatment in Overall Study, Using DCOA 1 Questionnaire
Time frame: Baseline up to Week 24
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Total Scores Related to Missed Injections by Treatment in Overall Study, Using DCOA 1 Questionnaire
Time frame: Baseline up to Week 24
Number of Participants as Per Responses to Choice of Injection Pen Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
Number of Participants as Per Responses to Preferred Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
Number of Participants as Per Responses to Convenience of the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
Number of Participants as Per Responses to Ease of Following Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
Number of Participants as Per Responses to Pen Ease of Use Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
Number of Participants as Per Responses to Participant Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
Number of Participants as Per Responses to Caregiver Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
Number of Participants as Per Responses to Family Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
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
Number of Participants as Per Responses to Intention to Comply Assessed at Week 24, Using DCOA 2 Questionnaire
Time frame: Week 24
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
Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score by Treatment in Overall Study
Time frame: Baseline up to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
32 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Genotropin and somatrogon in people with Growth Hormone Deficiency, run by Pfizer at 32 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, United Kingdom, Czechia, Slovakia, Bulgaria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Oct 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire (time frame: Baseline). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2021.