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

NCT06113952 | Withdrawn

• 1 other identifier: C0311024
• Study Type: observational
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Pediatric Growth Hormone Deficiency

Keywords

adherence; pediatric; Ngenla; growth hormone; growth hormone deficiency; daily growth hormone; smart bin; treatment experience

Outcome Measures

Primary Outcomes (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™ .

  12 months

Secondary Outcomes (13)

• Percentage of scheduled injections taken per participant

  12 months

• Change from baseline in Life Interference Questionnaire-Growth Hormone Deficiency I (LIQ-GHD I) questionnaire

  12 months

• Change from baseline in Quality of Life in Short Stature Youth (QoLISSY) questionnaire for patients and for caregivers

  12 months

• Healthcare practitioner (HCP) preference survey

  12 months

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

  12 month

Study Arms (2)

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

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

Interventions

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.

Patients treated with daily growth hormone; Patients treated with weekly growth hormone

Eligibility Criteria

• Age: 3 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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 \[as per normal standard of care (SoC)\].

You may not qualify if:

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

Sponsors & Collaborators

• Pfizer: lead

Related Links

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

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Injections

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 5, 2023
• First Posted: November 2, 2023
• Study Start: January 1, 2024
• Primary Completion: October 17, 2025
• Study Completion: October 17, 2025
• Last Updated: February 9, 2024

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will not share