Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed

A Multi-centre, Prospective, Open Label, Single-arm, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Clinical Parameters of Sogroya® Treatment in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed Under Normal Clinical Practice Conditions in Japan Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency (GHD) Where Epiphysial Discs Are Not Closed

The purpose of the study is to investigate the safety and effectiveness of Sogroya® in children with short stature due to growth hormone deficiency where epiphysial discs are not closed under real-world clinical practice in Japan. The study will last for about 1 year (at shortest) to 3 years (at longest) depending on when the participant takes part in the study. The participant will be asked to answer questionnaire(s) about how they feel about the growth hormone (GH) product treatment once during the study (at about 3 months after starting the Sogroya® treatment) and about 3 months after starting the Sogroya® treatment.

Study Overview

• Status: Recruiting
• Conditions: Growth Hormone Deficiency in Children
• Intervention / Treatment: Drug: Somapacitan

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Novo Nordisk; Phone Number: (+1) 866-867-7178; Email: clinicaltrials@novonordisk.com

Study Locations

• Japan
  • Tokyo, Japan, 158-0097
    • Recruiting
    • Novo Nordisk Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Children with short stature due to Growth Hormone Deficiency (GHD) where epiphysial discs are not closed under normal clinical practice conditions in Japan.

Description

Inclusion Criteria:

1. Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
2. The decision to initiate treatment with commercially available Sogroya® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study. Both GH treatment naïve and non-naïve children are eligible.
3. Male or female age 0 to 18 years (exclusive) at the time of signing informed consent.
4. Diagnosis with short stature due to GHD where epiphysial discs are not closed according to local normal clinical practice.

Exclusion Criteria:

1. Previous participation in this study. Participation is defined as having given informed consent in this study.
2. Treatment with any investigational drug within 30 days prior to baseline (the starting date of Sogroya® treatment).
3. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

4. Contraindication described in approved product labelling in Japan.

   1. Patients with hypersensitivity to the active substance or to any of the excipients
   2. Patients with malignant tumour
   3. Female patients who are either pregnant or likely to be pregnant

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Children with GHD; Participants will be treated with commercially available Sogroya® according to routine clinical practice at the discretion of the treating physician. Administration will be according to the approved product labelling. The decision to treat a participant with Sogroya® is made at the treating physician's discretion before and independently from the decision to include the patient in this study.

Drug: Somapacitan; Sogroya® treatment regimen will be in accordance with the approved product labelling in Japan.; Other Names: Sogroya®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse reactions (AR)	Measured as count of reactions.	From baseline (week 0) to end of study (up to 156 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse events (AEs)	Measured as count of events.	From baseline (week 0) to end of study (up to 156 weeks)
Number of serious adverse events (SAEs)	Measured as count of events.	From baseline (week 0) to end of study (up to 156 weeks)
Number of serious adverse reactions (SARs)	Measured as count of reactions.	From baseline (week 0) to end of study (up to 156 weeks)
Change in height velocity (HV)	Measured in centimeter (cm)/year.	Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Change in bone age	Measured in years.	Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Change in ratio of bone age/chronological age		Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Change in height standard deviation score (HSDS)		Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Change in height velocity standard deviation score (HVSDS)		Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Change in insulin-like growth factor-I standard deviation score (IGF-I SDS)	Measured as score ranging from -10 to +10. Negative scores indicated a IGF-I below the mean IGF-I for a child with the same age and gender, whereas positive scores indicated a IGF-I above the mean IGF-I for a child with the same age and gender. For participants with low IGF-I SDS at baseline, a positive change from baseline in IGF-I SDS indicated a better outcome.	Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Growth hormone device assessment tool (G-DAT)	Measured as count of patients choosing the individual response category. G-DAT is a questionnaire to gather information on how they feel about the GH product device assessed as "very easy", "easy", "neither difficult or easy", "difficult" or "very difficult" where "very easy" is best and "very difficult" is worst.	At 12 weeks
Growth hormone patient preference questionnaire (GH-PPQ)	Measured as count of patients choosing the individual response category. GH-PPQ is a disease specific questionnaire which measures the patient's growth hormone treatment preference.	At 12 weeks

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Transparency dept. 2834, Novo Nordisk A/S

Study record dates

Study Major Dates

• Study Start (Actual): October 4, 2023
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): July 31, 2026

Study Registration Dates

• First Submitted: October 25, 2023
• First Submitted That Met QC Criteria: October 25, 2023
• First Posted (Actual): October 31, 2023

Study Record Updates

• Last Update Posted (Actual): October 31, 2023
• Last Update Submitted That Met QC Criteria: October 25, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Hypothalamic Diseases; Bone Diseases; Bone Diseases, Endocrine; Pituitary Diseases; Bone Diseases, Developmental; Hypopituitarism; Dwarfism, Pituitary; Endocrine System Diseases; Dwarfism
• Other Study ID Numbers: NN8640-5005; U1111-1274-4223 (Other Identifier: World Health Organization (WHO))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com