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

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

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).

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