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
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 87
- 试验地点
- 32
- 主要终点
- Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 <
- •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).
排除标准
- 未提供
研究组 & 干预措施
Weekly to Daily
somatrogon to Genotropin
干预措施: Genotropin (Drug)
Daily to Weekly
Genotropin to somatrogon
干预措施: Genotropin (Drug)
Daily to Weekly
Genotropin to somatrogon
干预措施: somatrogon (Drug)
Weekly to Daily
somatrogon to Genotropin
干预措施: somatrogon (Drug)
结局指标
主要结局
Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire
时间窗: 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 by Treatment in Overall Study, Using DCOA 1 Questionnaire
时间窗: 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).
Total Score Related to Overall Life Interference Assessed at Week 24, Using DCOA 1 Questionnaire
时间窗: 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 Assessed at Week 12, Using DCOA 1 Questionnaire
时间窗: 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).
次要结局
- 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(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(Baseline up to Week 24)
- Total Score Related to Pen Ease of Use by Treatment in Overall Study, Using DCOA 1 Questionnaire(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(Baseline, Week 12, Week 24)
- Total Score Related to Pen Ease of Use Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire(Baseline, Week 12, Week 24)
- Total Score Related to Convenience of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire(Baseline, Week 12, Week 24)
- Total Score Related to Satisfaction With Overall Treatment Experience by Treatment in Overall Study, Using DCOA 1 Questionnaire(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(Baseline, Week 12, Week 24)
- Total Scores Related to Willingness to Continue Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire(Baseline up to Week 24)
- Total Score Related to Ease of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire(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(Baseline, Week 12, Week 24)
- Total Score Related to Convenience of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire(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(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(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(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(Baseline up to Week 24)
- Total Scores Related to Missed Injections Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire(Baseline, Week 12, Week 24)
- Total Scores Related to Missed Injections by Treatment in Overall Study, Using DCOA 1 Questionnaire(Baseline up to Week 24)
- Number of Participants as Per Responses to Choice of Injection Pen Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Preferred Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Convenience of the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Ease of Following Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Pen Ease of Use Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Participant Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Caregiver Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Family Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Response to Benefit Relating to the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Number of Participants as Per Responses to Intention to Comply Assessed at Week 24, Using DCOA 2 Questionnaire(Week 24)
- Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score Assessed at Baseline, Week 12 and Week 24(Baseline, Week 12, Week 24)
- Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score by Treatment in Overall Study(Baseline up to Week 24)