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company business-strategic decision
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The purpose of this study is to learn about:
This study is seeking participants who:
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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
| |
| 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sharps bin to collect used needles/injections | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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™ . | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 12 months |
| Change from baseline in Life Interference Questionnaire-Growth Hormone Deficiency I (LIQ-GHD I) questionnaire |
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Inclusion Criteria:
Exclusion Criteria:
7. Children with other acute medical or psychiatric condition
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Patients diagnosed with pGHD who are currently receiving growth hormone treatment or are about to start it.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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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.
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| ID | Term |
|---|---|
| D004393 | Dwarfism, Pituitary |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D007267 | Injections |
| ID | Term |
|---|---|
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |
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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:
|
| 12 months |
| 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:
| 12 months |
| 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. | 12 months |
| 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. | 12 month |
| Presence or absence of factors influencing adherence | Lifestyle factors are assessed:
| 12 months |
| 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™ . | 12 months |
| 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™ . | 12 months |
| 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™ . | 12 months |
| 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. | 12 months |
| 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. | 12 months |
| 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. | 12 months |
| 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. | 12 months |
| D001849 |
| Bone Diseases, Endocrine |
| D007018 | Hypopituitarism |
| D010900 | Pituitary Diseases |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D004700 | Endocrine System Diseases |