Clinical trial NCT06113952: A Study to Compare the Experiences of Taking Daily Growth Hormone Injections to Weekly Ngenla in Children With Low Levels of Growth Hormone
Official title: 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
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Pediatric Growth Hormone Deficiency
Keywords: adherence
pediatric
Ngenla
growth hormone
growth hormone deficiency
daily growth hormone
smart bin
treatment experience
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2023-11-02
Enrollment: 0
Sex: ALL
Minimum age (years): 3
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients diagnosed with pGHD who are currently receiving growth hormone treatment or are about to start it.

Brief summary: 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.

Detailed description: 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).

Eligibility criteria: 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

Primary outcomes:
measure: Mean Adherence To Ngenla and daily growth hormone
timeFrame: 12 months
Secondary outcomes:
measure: Percentage of scheduled injections taken per participant
timeFrame: 12 months
measure: Change from baseline in Life Interference Questionnaire-Growth Hormone Deficiency I (LIQ-GHD I) questionnaire
timeFrame: 12 months
measure: Change from baseline in Quality of Life in Short Stature Youth (QoLISSY) questionnaire for patients and for caregivers
timeFrame: 12 months
measure: Healthcare practitioner (HCP) preference survey
timeFrame: 12 months
measure: Change from baseline in effectiveness evaluations for Ngenla and daily growth hormone
timeFrame: 12 month
measure: Presence or absence of factors influencing adherence
timeFrame: 12 months
measure: Mean Adherence To Ngenla and daily growth hormone for age subgroups.
timeFrame: 12 months
measure: Mean Adherence To Ngenla and daily growth hormone for switch participants
timeFrame: 12 months
measure: Mean Adherence To Ngenla and daily growth hormone for naive/non-naive subgroups.
timeFrame: 12 months
measure: Number of participants who discontinued Ngenla or daily growth hormone
timeFrame: 12 months
measure: Percent of participants who discontinued Ngenla or daily growth hormone
timeFrame: 12 months
measure: Number of participants who switch growth hormone treatment
timeFrame: 12 months
measure: Percentage of participants who switch growth hormone treatment
timeFrame: 12 months
Other outcomes:
Locations:
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: