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

February 7, 2024 updated by: Pfizer

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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).

Study Type

Observational

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients treated with weekly growth hormone
Approximately 200 children with a diagnosis of pGHD who are treatment naïve or currently receiving once weekly Ngenla will be enrolled.
Device: Sharps bin to collect used needles/injections
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.
Patients treated with daily growth hormone
Approximately 200 children with a diagnosis of pGHD who are treatment naive or currently receiving daily GH injections will be enrolled
Device: Sharps bin to collect used needles/injections
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean Adherence To Ngenla and daily growth hormone
Time Frame: 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™ .
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of scheduled injections taken per participant
Time Frame: 12 months
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.
12 months
Change from baseline in Life Interference Questionnaire-Growth Hormone Deficiency I (LIQ-GHD I) questionnaire
Time Frame: 12 months

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
12 months
Change from baseline in Quality of Life in Short Stature Youth (QoLISSY) questionnaire for patients and for caregivers
Time Frame: 12 months

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
12 months
Healthcare practitioner (HCP) preference survey
Time Frame: 12 months
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.
12 months
Change from baseline in effectiveness evaluations for Ngenla and daily growth hormone
Time Frame: 12 month
Height in cm, weight in kg, and BMI in kg/m2 at diagnosis and follow-up visits and annualized height velocity in cm.
12 month
Presence or absence of factors influencing adherence
Time Frame: 12 months

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
12 months
Mean Adherence To Ngenla and daily growth hormone for age subgroups.
Time Frame: 12 months
-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™ .
12 months
Mean Adherence To Ngenla and daily growth hormone for switch participants
Time Frame: 12 months
- 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™ .
12 months
Mean Adherence To Ngenla and daily growth hormone for naive/non-naive subgroups.
Time Frame: 12 months
- 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™ .
12 months
Number of participants who discontinued Ngenla or daily growth hormone
Time Frame: 12 months
Persistency/discontinuation is evaluated at specific time windows, after patients stop utilizing the sharps bin.
12 months
Percent of participants who discontinued Ngenla or daily growth hormone
Time Frame: 12 months
Persistency/discontinuation is evaluated at specific time windows, after patients stop utilizing the sharps bin.
12 months
Number of participants who switch growth hormone treatment
Time Frame: 12 months
Number of participants who switch from daily to weekly GH injections or from weekly to daily GH injections is reported.
12 months
Percentage of participants who switch growth hormone treatment
Time Frame: 12 months
Percentage of participants who switch from daily to weekly GH injections or from weekly to daily GH injections is reported.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Investigators

  • Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

January 1, 2024

Primary Completion (Estimated)

October 17, 2025

Study Completion (Estimated)

October 17, 2025

Study Registration Dates

First Submitted

October 5, 2023

First Submitted That Met QC Criteria

November 1, 2023

First Posted (Actual)

November 2, 2023

Study Record Updates

Last Update Posted (Estimated)

February 9, 2024

Last Update Submitted That Met QC Criteria

February 7, 2024

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C0311024

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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