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Pediatric Growth Hormone DeficiencyNCT06113952

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

An observational study of Pediatric Growth Hormone Deficiency, run by Pfizer.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 3 to 16 years
Conditions the study is for
Pediatric Growth Hormone Deficiency
What is being tested?
Sharps bin to collect used needles/injections
How many people
0 participants
Where
No sites listed yet
Study dates
Jan 2024to 17 Oct 2025
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 16 years
  • All sexes
  • No healthy volunteers
  • Child

Study population: Patients diagnosed with pGHD who are currently receiving growth hormone treatment or are about to start it.

Who may be able to join

  • 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)].

Who cannot join

  • 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.
  • 7. Children with other acute medical or psychiatric condition

About this study

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

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.
Read more

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.

Read the 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).

Being tested
Sharps bin to collect used needles/injections
Keywords
adherence, pediatric, Ngenla, growth hormone, growth hormone deficiency, daily growth hormone, smart bin, treatment experience
Medical subject headings
Dwarfism, Pituitary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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.

Study groups in NCT06113952
Study groupRoleWhat they receive
Patients treated with weekly growth hormoneApproximately 200 children with a diagnosis of pGHD who are treatment naïve or currently receiving once weekly Ngenla will be enrolled.
Receives: Device: Sharps bin to collect used needles/injections
Patients treated with daily growth hormoneApproximately 200 children with a diagnosis of pGHD who are treatment naive or currently receiving daily GH injections will be enrolled
Receives: Device: Sharps bin to collect used needles/injections

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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™ .

    Measured: 12 months

Secondary outcomes (13)

  1. 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.

    Measured: 12 months

  2. 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

    Measured: 12 months

  3. 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

    Measured: 12 months

  4. 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.

    Measured: 12 months

  5. Change from baseline in effectiveness evaluations for Ngenla and daily growth hormone

    Height in cm, weight in kg, and BMI in kg/m2 at diagnosis and follow-up visits and annualized height velocity in cm.

    Measured: 12 month

  6. Presence or absence of factors influencing adherence

    Lifestyle factors are assessed:

    • travel, illness, overnight sleep
    • injection administration by caregiver/nurse or self-administration Number of concomitant medications and route of administration are collected

    Measured: 12 months

  7. Mean Adherence To Ngenla and daily growth hormone for age subgroups.

    -Different age groups: 3-7 years/8-11 years/12-16 years The adherence for Ngenla and daily growth hormone follows the medication possession ratio definition(MPR). The MPR is calculated from the total number of injections used and collected in the Smart Sharps Bin™ .

    Measured: 12 months

  8. Mean Adherence To Ngenla and daily growth hormone for switch participants

    • Switch participants: dGH to Ngenla and vice versa The adherence for Ngenla and daily growth hormone follows the medication possession ratio definition(MPR). The MPR is calculated from the total number of injections used and collected in the Smart Sharps Bin™ .

    Measured: 12 months

  9. Mean Adherence To Ngenla and daily growth hormone for naive/non-naive subgroups.

    • Treatment naïve vs non-naïve (either dGH or Ngenla) The adherence for Ngenla and daily growth hormone follows the medication possession ratio definition(MPR). The MPR is calculated from the total number of injections used and collected in the Smart Sharps Bin™ .

    Measured: 12 months

  10. Number of participants who discontinued Ngenla or daily growth hormone

    Persistency/discontinuation is evaluated at specific time windows, after patients stop utilizing the sharps bin.

    Measured: 12 months

  11. Percent of participants who discontinued Ngenla or daily growth hormone

    Persistency/discontinuation is evaluated at specific time windows, after patients stop utilizing the sharps bin.

    Measured: 12 months

  12. Number of participants who switch growth hormone treatment

    Number of participants who switch from daily to weekly GH injections or from weekly to daily GH injections is reported.

    Measured: 12 months

  13. Percentage of participants who switch growth hormone treatment

    Percentage of participants who switch from daily to weekly GH injections or from weekly to daily GH injections is reported.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Nov 2023
  2. Study startJan 2024 (estimated)
  3. Primary completion17 Oct 2025 (estimated)
  4. Study completion17 Oct 2025 (estimated)

Why it stopped: company business-strategic decision

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06113952
Sponsor's protocol ID
C0311024
Status last verified
Oct 2023
First submitted
5 Oct 2023
Last updated
9 Feb 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Questions about this trial

What is this clinical trial studying?

An observational study of Pediatric Growth Hormone Deficiency, run by Pfizer.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 9 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mean Adherence To Ngenla and daily growth hormone (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06113952.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2024.

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