A Study to Follow Paediatric Participants With Growth Hormone Deficiency Treated With Somapacitan for Long Term Safety Information and Clinical Parameters

NCT06709040 | Enrolling By Invitation

• 2 other identifiers: NN8640-4787U1111-1294-5941
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study is to look at the long-term safety and clinical parameters of somapacitan in paediatric participants with growth hormone deficiency under routine clinical practice conditions. The study population will include 400 paediatric growth hormone deficient participants from the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (GloBE-Reg) treated with once-weekly somapacitan and fulfilling the eligibility criteria of the study. The total duration of the study is planned to 10 years consisting of a 5-year recruitment period in the GLoBE-Reg followed by a 5-year follow-up period.

Conditions

Growth Hormone Deficiency; Neoplasms; Diabetes Mellitus Type 2

Outcome Measures

Primary Outcomes (1)

• Number of adverse drug reactions

  Measured as count of events.

  From baseline (week 0) to end of study (up to 10 years)

Secondary Outcomes (11)

• Number of medication errors (incorrect dose administration)

  From baseline (week 0) to end of study (up to 10 years)

• Number of participants with incident neoplasm

  From baseline (week 0) to end of study (up to 10 years)

• Number of participants with incident diabetes mellitus type 2

  From baseline (week 0) to end of study (up to 10 years)

• Height velocity

  From baseline (week 0) to end of study (up to 10 years)

• Change in height velocity standard deviation score (HVSDS)

  From baseline (week 0) to end of study (up to 10 years)

Study Arms (1)

Participants with Growth Hormone Deficieny

The study is non-interventional as there are no interventions involved and decision to treat participants with commerically available somapacitan will be made at the treating physician's discretion prior to, and independently from, the decision to include the participants in the GLoBE-Reg registry. Novo Nordisk will not provide any products for included participants during the conduct of the study.

Drug: Somapacitan

Interventions

Somapacitan DRUG

Participants will be treated with commercially available somapacitan according to routine clinical practice at the discretion of the treating physician.

Participants with Growth Hormone Deficieny

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Paediatric participants with growth hormone deficiency.

You may qualify if:

• Treated with commercially available somapacitan according to local practice at the discretion of the physician.
• Primary confirmed diagnosis of growth hormone deficiency as per local practice.
• Male or female below 18 years of age at the time of signing informed consent in the GLoBE-Reg.

You may not qualify if:

• Participants with active malignancy or in treatment for active pre-existing malignancy.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (1)

University of Glasgow

Glasgow, Scotland, United Kingdom

Location

MeSH Terms

Conditions

Dwarfism, Pituitary; Neoplasms; Diabetes Mellitus, Type 2

Interventions

somapacitan

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; Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Clinical Transparency (dept. 2834)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: November 26, 2024
• First Posted: November 29, 2024
• Study Start: December 13, 2024
• Primary Completion (Estimated): April 14, 2034
• Study Completion (Estimated): April 14, 2034
• Last Updated: February 5, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share

Locations

GB (1)