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

NCT ID: NCT06113952

Last Updated: 2024-02-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2024-01-31

Study Completion Date

2025-10-17

Brief Summary

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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

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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).

Conditions

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Pediatric Growth Hormone Deficiency

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

Sharps bin to collect used needles/injections

Intervention Type DEVICE

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

Sharps bin to collect used needles/injections

Intervention Type DEVICE

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.

Interventions

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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.

Intervention Type DEVICE

Eligibility Criteria

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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
Minimum Eligible Age

3 Years

Maximum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=C0311024

To obtain contact information for a study center near you, click here.

Other Identifiers

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C0311024

Identifier Type: -

Identifier Source: org_study_id

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