A Study to Compare the Experiences of Taking Daily Growth Hormone Injections to Weekly Ngenla in Children With Low Levels of Growth Hormone

Pfizer

Overview

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.

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).

Study Type

OBSERVATIONAL

Conditions

Pediatric Growth Hormone Deficiency

Interventions

Sharps bin to collect used needles/injectionsDEVICE

Enrolled participants will be provided with a Smart Sharps Bin. The bin will collect used needles over a period of 12 months starting from enrollment.

Eligibility

Sex: ALLMin age: 3 YearsMax age: 16 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Children of any sex aged 3-16 years at baseline 2. Children that have received a diagnosis of pGHD requiring GH injections. 3. Children that are currently being treated (dGH/Ngenla) or are treatment naïve but ready to start treatment. 4. Those who currently use a sharps bin to collect used needles \[as per normal standard of care (SoC)\]. Exclusion Criteria: 1. Children with psychosocial dwarfism. 2. Children born small for gestational age (SGA) 3. Children with chromosomal abnormalities 4. Children with other causes of short stature, such as uncontrolled primary hypothyroidism or rickets. 5. Children with a history of cancer. 7\. Children with other acute medical or psychiatric condition

Outcomes

Primary Outcomes

Mean Adherence To Ngenla and daily growth hormone

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™ .

Time frame: 12 months

Secondary Outcomes

Percentage of scheduled injections taken per participant

The percentage of scheduled injections taken per participant is calculated as the proportion of actual number of injections with Ngenla or daily growth hormone used from the total number of expected injections.

Time frame: 12 months

Change from baseline in Life Interference Questionnaire-Growth Hormone Deficiency I (LIQ-GHD I) questionnaire

The LIQ-GHD I instrument uses either a 5 point scale from never to always or very easy to very difficult or a 7 point scale from extremely convenient to extremely inconvenient. It contains the following subscales: * Pen ease of use * Ease of the injection schedule * Satisfaction with overall treatment experience * Willingness to continue injection schedule * Patient life interference * Number of missed injections during the past 4 weeks(two questions asking for number of missed injections and reasons) * Caregiver life interference

Time frame: 12 months

Change from baseline in Quality of Life in Short Stature Youth (QoLISSY) questionnaire for patients and for caregivers

The QoLISSY questionnaire is a 5 point scale from not at all/never to extremely/always. It evaluates the following health related quality of life domains for participants and their caregivers: * Physical impact of height * Social impact of height * Emotional impact of height

Time frame: 12 months

Healthcare practitioner (HCP) preference survey

HCP preference and satisfaction survey for treatment regimen and treatment experience (daily or weekly injections or no preference) and satisfaction rated from very satisfied to very dissatisfied.

Data from ClinicalTrials.gov

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