A MULTICENTER LOW-INTERVENTIONAL STUDY TO EVALUATE AND MONITOR TREATMENT EXPERIENCE WITH WEEKLY GROWTH HORMONE (NGENLA) VERSUS DAILY GROWTH HORMONE INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- Pfizer
- 主要终点
- Mean Adherence To Ngenla and daily growth hormone
研究概览
简要总结
The purpose of this study is to learn about:
- how children stick to taking their injections
- their experience with the study medicines (Ngenla and daily growth hormone) prescribed to children with low levels of growth hormone.
This study is seeking participants who:
- are being treated or are ready to start treatment with daily growth hormone or Ngenla.
- use a sharps bin to collect used needles.
The study medicines will be given as per regular care agreed with the doctor. The study will compare participant experiences to help us see:
- the difference in how the participants stick to taking their daily growth hormone injections compared to participants using once weekly Ngenla.
Participants will take part in this study for up to 14 months. During this time, they will have 3 study visits at the study clinic. The participants will use the HealthBeacon™ smart sharps bin for collecting the used needles or injections.
详细描述
This multicenter, international, longitudinal, prospective, site-based, low-interventional study aims to evaluate and monitor treatment experience, defined by treatment adherence and persistence, in children aged 3-16 years old with a diagnosis of pediatric growth hormone deficiency (pGHD), receiving either daily growth hormone (dGH) treatment or once weekly Ngenla. The study is planned to be conducted in a total of 50 participating sites distributed across Belgium, Canada, France, Italy, Luxembourg, Spain, UK, and US.
Data on treatment adherence and persistence will be collected in real-time over a period of 12 months using the HealthBeacon™ Smart Sharps Bin™ as a treatment monitoring device.
Additional data, including demographic and clinical characteristics, will be collected at three timepoints, in line with the routine care in each participating site and/or country, over a maximum period of 14 months.
Approximately 400 participants will be enrolled in the study (approximately 200 participants prescribed dGH injections and approximately 200 prescribed Ngenla).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 3 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children of any sex aged 3-16 years at baseline
- •Children that have received a diagnosis of pGHD requiring GH injections.
- •Children that are currently being treated (dGH/Ngenla) or are treatment naïve but ready to start treatment.
- •Those who currently use a sharps bin to collect used needles [as per normal standard of care (SoC)].
排除标准
- •Children with psychosocial dwarfism.
- •Children born small for gestational age (SGA)
- •Children with chromosomal abnormalities
- •Children with other causes of short stature, such as uncontrolled primary hypothyroidism or rickets.
- •Children with a history of cancer.
- •Children with other acute medical or psychiatric condition
结局指标
主要结局
Mean Adherence To Ngenla and daily growth hormone
时间窗: 12 months
The adherence for Ngenla and daily growth hormone followed the medication possession ratio definition(MPR). The MPR is calculated from the total number of injections used and collected in the Smart Sharps Bin™ .
次要结局
- Percentage of scheduled injections taken per participant(12 months)
- Change from baseline in Life Interference Questionnaire-Growth Hormone Deficiency I (LIQ-GHD I) questionnaire(12 months)
- Mean Adherence To Ngenla and daily growth hormone for switch participants(12 months)
- Mean Adherence To Ngenla and daily growth hormone for naive/non-naive subgroups.(12 months)
- Percent of participants who discontinued Ngenla or daily growth hormone(12 months)
- Number of participants who switch growth hormone treatment(12 months)
- Percentage of participants who switch growth hormone treatment(12 months)
- Change from baseline in Quality of Life in Short Stature Youth (QoLISSY) questionnaire for patients and for caregivers(12 months)
- Change from baseline in effectiveness evaluations for Ngenla and daily growth hormone(12 month)
- Healthcare practitioner (HCP) preference survey(12 months)
- Presence or absence of factors influencing adherence(12 months)
- Mean Adherence To Ngenla and daily growth hormone for age subgroups.(12 months)
- Number of participants who discontinued Ngenla or daily growth hormone(12 months)