Korean Post-marketing Surveillance Ngenla® Pre-filled Pen Injection for the Treatment of Pediatric Patients With Growth Disturbance Due to Insufficient Secretion of Growth Hormone

Post Marketing Surveillance (PMS) Study for Ngenla Prefilled Pen in Pediatric Patients Who Have Endogenous Growth Failure Due to an Inadequate Secretion of Endogenous Growth Hormone in Korea

This non-interventional study will be performed by design of post-marketing surveillance (PMS) as an additional pharmacovigilance activity of the Risk Management Plan (RMP) for Ngenla® pre-filled pen injection, which is required by the Ministry of Food and Drug Safety (MFDS) according to the local regulation. This post-marketing surveillance will investigate the safety and effectiveness of Ngenla® pre-filled pen injection as the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone during 6 years under the setting of routine practice in Korea.

Study Overview

• Status: Active, not recruiting
• Conditions: Pediatric Growth Hormone Deficiency
• Intervention / Treatment: Drug: Ngenla

• Study Type: Observational
• Enrollment (Estimated): 565

Contacts and Locations

Study Locations

• South Korea
  • Seoul, South Korea
    • Pfizer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 19 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study will be performed in patients who have received at least 1 dose of Ngenla® pre-filled pen injection for the following indications as per local product label. (Treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone)

Description

Inclusion Criteria:

- 1. Children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone who have received treatment with somotarogon or have been determined to start treatment with somatrogon according to the approved indications of the medicinal product;

2. Evidence of a personally signed and dated informed consent document indicating that the patient or their parent(s)/legal guardian, if applicable, have been informed of all pertinent aspects of the study.

Exclusion Criteria:

1. Patients concurrently participating in other studies involving therapeutic interventions and/or investigational products;
2. Patients who have contraindications to somatrogon as specified in the approved LPD.
3. Patients with hypersensitivity or case history to somatrogon or to any of the excipients in the product

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Participants receiving Ngenla; Participants receiving Ngenla according to label	Drug: Ngenla; As provided in real world practice

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events (AEs)	up to 28 days after last dose

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in Annualized Height Velocity in cm/year	baseline, up to 12 months
Change from baseline in Height Standard Deviation Score	baseline, up to 12 months
Change from baseline in Bone Maturation (BM)	baseline, up to 12 months

Collaborators and Investigators

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

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): June 20, 2024
• Primary Completion (Estimated): September 23, 2026
• Study Completion (Estimated): September 23, 2026

Study Registration Dates

• First Submitted: August 19, 2022
• First Submitted That Met QC Criteria: August 19, 2022
• First Posted (Actual): August 22, 2022

Study Record Updates

• Last Update Posted (Actual): January 27, 2026
• Last Update Submitted That Met QC Criteria: January 26, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric Growth Hormone Deficiency; Somatrogon
• Other Study ID Numbers: C0311016

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