Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)

A Non-interventional, Prospective Study in Germany to Investigate the Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in a Patient Population With Isolated Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)

Participants are free to decide if they want to take part in this study or not. The study will be conducted to collect information about the influence of adherence to growth hormone therapy with Norditropin® in children and teenagers in daily practice in Germany. This study will look mainly at the difference in near final height between children and teenagers who adhere to their therapy plan with Norditropin® to non-adherent patients. Participants will get Norditropin® as prescribed to them by their doctor. The study will last as long as the therapy with growth hormone is seen necessary by the participants' doctors and the participants, up to a maximum of 10 years. During the visits at the participants' doctors participants will be asked to fill in a questionnaire.

Study Overview

• Status: Enrolling by invitation
• Conditions: Growth Hormone Deficiency in Children; Born Small for Gestational Age
• Intervention / Treatment: Drug: Norditropin® FlexPro®

• Study Type: Observational
• Enrollment (Estimated): 750

Contacts and Locations

Study Locations

• Germany
  • Ulm, Germany, 89075
    • Universitätsklinikum Ulm für Kinder- und Jugendmedizin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 13 years (Child)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Paediatric patients with iGHD or SGA.

Description

Inclusion Criteria:

• Informed consent of parent or legally acceptable representative of subject and child assent, as age-appropriate must be obtained before any study-related activities.

  1. The parent or legally acceptable representative of the child must sign and date the Informed Consent Form (according to local requirements) and
  2. The child must sign and date the Child Assent Form or provide oral assent (if required according to local requirements).
• The decision to initiate treatment with commercially available Norditropin® FlexPro® has been made by the treating physician and the patient's parents/legal guardian before and independently of the decision to include the patient in this study.
• Male or female, all age groups equal to or below 15 years with more than 2 years expected remaining treatment time until reaching NFH. Patients who self-inject should be above 8 years of age in order to be able to fill in the questionnaire.

• Children being GH naïve at baseline with one of the following confirmed diagnoses

  1. Isolated growth hormone deficiency (iGHD)
  2. Small for gestational age (SGA)

Exclusion Criteria:

• Previous participation in this study. Participation is defined as having given informed consent in this study.
• Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
• Patients who have an expected future duration of therapy of less than 2 years are not eligible for the study.

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

Paediatric patients with iGHD or SGA; Children with one of the following confirmed diagnoses: isolated growth hormone deficiency (iGHD) or small for gestational age (SGA)

Drug: Norditropin® FlexPro®; Patients will be treated with commercially available Norditropin® FlexPro® according to routine clinical practice at the discretion of the treating physician. The decision to initiate treatment with commercially available Norditropin® FlexPro® has been made by the treating physician and the patient's parents/legal guardian before and independently of the decision to include the patient in this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Near final height (NFH) standard deviation score (SDS) measured in scores	SDS, -3 to +3	At the end of the study (up to 10 years)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in growth rate (height velocity standard deviation score, HV SDS)	Score, range -10 to +10	From baseline (month 0) to end of study (up to 10 years)
Change in growth rate (HV SDS) between visits (usually patients are seen every 3 to 6 months)	Score, range -10 to +10	From baseline (month 0) to end of study (up to 10 years)
Adherence response as indicated in question at the bottom of newly developed Patient Reported Outcome (PRO) questionnaire each time questionnaire is filled out (preferably at each visit)	The following adherence levels will be used: High adherence: above 85.7% of doses administered Medium adherence: 57.1 - 85.7% of doses administered Low adherence: below 57.1% of doses administered	From baseline (month 0) to end of study (up to 10 years)
Adherence measured as number of medication devices returned versus number of prescribed devices for the period between visits	Count	From baseline (month 0) to end of study (up to 10 years)
Change of self-rated adherence level throughout the study measured in newly developed PRO questionnaire each time questionnaire is filled out (preferably at each visit)	The following adherence levels will be used: High adherence: above 85.7% of doses administered Medium adherence: 57.1 - 85.7% of doses administered Low adherence: below 57.1% of doses administered	From baseline (month 0) to end of study (up to 10 years)

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): June 21, 2019
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): June 30, 2031

Study Registration Dates

• First Submitted: May 31, 2019
• First Submitted That Met QC Criteria: May 31, 2019
• First Posted (Actual): June 3, 2019

Study Record Updates

• Last Update Posted (Actual): March 12, 2024
• Last Update Submitted That Met QC Criteria: March 11, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Musculoskeletal Diseases; Hypothalamic Diseases; Bone Diseases; Bone Diseases, Endocrine; Pituitary Diseases; Dwarfism; Bone Diseases, Developmental; Hypopituitarism; Dwarfism, Pituitary; Endocrine System Diseases
• Other Study ID Numbers: GH-4488; U1111-1217-5835 (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

Drug and device information, study documents

• Studies a U.S. FDA-regulated device product: No