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

Growth Hormone Deficiency in Children Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06109935
• Other study ID #: NN8640-5005
• Secondary ID: U1111-1274-4223
• Status: Recruiting
• Start date: October 4, 2023
• Est. completion date: July 31, 2026

Study information

• Verified date: October 2023
• Source: Novo Nordisk A/S
• Contact: Novo Nordisk
• Phone: (+1) 866-867-7178
• Email: clinicaltrials[@]novonordisk.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: July 31, 2026
• Est. primary completion date: July 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Dwarfism
• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency in Children

Intervention

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

Locations

Country	Name	City	State
Japan	Novo Nordisk Investigational Site	Tokyo

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse reactions (AR)	Measured as count of reactions.	From baseline (week 0) to end of study (up to 156 weeks)
Secondary	Number of adverse events (AEs)	Measured as count of events.	From baseline (week 0) to end of study (up to 156 weeks)
Secondary	Number of serious adverse events (SAEs)	Measured as count of events.	From baseline (week 0) to end of study (up to 156 weeks)
Secondary	Number of serious adverse reactions (SARs)	Measured as count of reactions.	From baseline (week 0) to end of study (up to 156 weeks)
Secondary	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)
Secondary	Change in bone age	Measured in years.	Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Secondary	Change in ratio of bone age/chronological age		Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Secondary	Change in height standard deviation score (HSDS)		Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Secondary	Change in height velocity standard deviation score (HVSDS)		Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
Secondary	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)
Secondary	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
Secondary	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